Here are the studies that match your search criteria. If you are interested in participating, please reach out to the contact listed for the study. If no contact is listed, contact us and we'll help you find the right person.
DExterous Hand Control Through Fascicular Targeting (DEFT) - (Human Subjects)
Our goal is to temporarily implant the following groups for 180 +/- 30 days:
1. Five human partial hand amputees (amputated at the level of the hand) with 2 FAST-LIFE
electrodes, one inserted into the motor fascicle of the ulnar nerve and the other into
the sensory fascicle.
2. Five human hand and forearm amputees (amputated at the level of the forearm) with 2
FAST-LIFE electrodes in the ulnar nerve (one in the motor fascicle, one in the sensory
fascicle) and 2-5 FAST-LIFE electrodes in the median nerve (one in the motor fascicle,
one to four in the remaining sensory fascicles).
Call 214-648-5005 studyfinder@utsouthwestern.edu
Jonathan Cheng
98715
All
18 Years to 95 Years old
N/A
This study is NOT accepting healthy volunteers
NCT02994160
STU 092014-061
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Criteria for Inclusion of Subjects:
Hand and forearm amputees:
1. Male or female, age 18 and older, of any race or ethnicity
2. Able and willing to sign Consent
3. Able and willing to participate in all study activities including implantation,
testing and explantation of the study device.
4. Able to communicate effectively in English without an interpreter
After preliminary screening subjects will be assessed for the following inclusion criteria:
1. Patient has an existing myoelectric hand prosthesis and demonstrates proficiency
during daily use
2. Overall and phantom pain are well-controlled and not incapacitating
Criteria for Exclusion of Subjects:
1. If MR neurogram and EMG/NCS study show nerve or muscle dysfunction/injury at a higher
level than anticipated based on the appearance of the physical amputation stump, the
subject may be excluded from the study due to adverse neuromuscular anatomy which
would preclude use of the proposed experimental electrode implants. The radiographs
will be used to confirm suitability of the amputation stump configuration. If the bony
anatomy of the amputation stump is found to be unsuitable, the patient may be excluded
from the study.
2. Subjects who have a history of cardiac arrhythmia will be excluded from the study.
Other: FastLIFE electrode
Amputation, Traumatic, Hand, Brain and Nervous System
peripheral nerve, intraneural electrode, hand amputation, forearm amputation
Phase 1 Dose-escalating Study of MM-398 (Irinotecan Sucrosofate Liposome Injection) Plus Intravenous Cyclophosphamide in Recurrent or Refractory Pediatric Solid Tumors
This is a Phase 1 study of the combination of two drugs: MM-398 and Cyclophosphamide. The
goal is to find the highest dose of MM-398 that can be given safely when it is used together
with the chemotherapy drug Cyclophosphamide.
• Histologically or cytologically-confirmed Ewing sarcoma, rhabdomyosarcoma,
neuroblastoma, or osteosarcoma
• Disease progression after prior therapy in locally advanced or metastatic setting
• Measurable or evaluable disease based on the Response Evaluation Criteria in Solid
Tumors (RECIST v1.1) criteria
• Age 12 months to <21 years
• Adequate bone marrow reserves, hepatic function, and renal function
• Recovered from effects of any prior surgery or cancer therapy
• Patients 18 years or older will provide written consent. A parent or legal guardian of
a patient <18 years of age will provide informed consent and patients 11 to 18 years
of age will provide written assent or as per participating institutional policy.
Exclusion Criteria:
• Clinically significant gastrointestinal disorders
• NYHA Class III or IV congestive heart failure, ventricular arrhythmias or uncontrolled
blood pressure
• Active infection or unexplained fever
• Known hypersensitivity to any of the components of MM-398 or other liposomal products
• Recent Investigational therapy
• Pregnant or breast feeding; females of child-bearing potential must test negative for
pregnancy at the time of enrollment
Drug: MM-398 (Irinotecan Sucrosofate Liposome Injection) plus cyclophosphamide
Neuroblastoma, Sarcoma, Ewing Sarcoma, Mycosis Fungoides, Rhabdomyosarcoma, Osteosarcoma, Recurrent or Refractory Solid Tumors, Brain and Nervous System, Eye and Orbit, Anus, Bones and Joints, Breast - Female, Breast - Male, Cervix, Colon, Corpus Uteri, Ear, Esophagus, Gall Bladder, Head and Neck, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung/Thoracic, Melanoma, skin, Nose, Other Digestive Organ, Other Endocrine System, Other Female Genital, Other Male Genital, Other Respiratory and Intrathoracic Organs, Other Skin, Other Urinary, Ovary, Pancreas, Prostate, Rectum, Stomach, Throat, Thyroid, Urinary Bladder, Uterine (Endometrial), Vulva, Hodgkins Lymphoma, Kaposis sarcoma, Other Hematopoietic, Small Intestine, Soft Tissue
Main Study Up to 100 subjects, both non-CF volunteers and Cystic Fibrosis (CF) patients, will
participate in a single study visit that will include a DEXA scan, micro CT, and blood
collection.
Denosumab (Prolia) Sub study Approximately 10 adult subjects with CF who participated in the
main study and have results indicating bone disease will receive treatment with Denosumab for
up to 5 years. They will be asked to return annually for repeat DEXA scans, micro CT, and
blood collection.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Raksha Jain
19733
All
18 Years to 64 Years old
Phase 4
This study is also accepting healthy volunteers
NCT03921060
STU 052018-007
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Cystic Fibrosis Main Study
Inclusion Criteria:
• Must have CF diagnosis confirmed by sweat test or genotype analysis
• Subjects (and parents/legal guardians as applicable) must have the ability to read and
write in English
Sub-study
Exclusion Criteria:
• No CF diagnosis
• Men or women without osteoporosis
• Less than 18 years of age
• Unwilling to return annually for study visits for up to 5 years
• Unwilling and/or medically unable to take denosumab
• Stratum I
• Patients must be less than 18 years of age at the time of diagnosis.
• Patients must have histological verification of the diagnosis of Langerhans cell
histiocytosis according to the criteria described in Section 6.1
• Signed informed consent form
• Stratum II
• Patients of Stratum I who have:
• Progressive disease (AD worse) in non-risk organs after 6 weeks (Initial Course
• AD intermediate or worse in non-risk organs or AD better in risk organs after 12
weeks (Initial Course 2)
• Disease progression (AD worse) in non-risk organs at any time during continuation
treatment
• Active disease at the end of Stratum I treatment
• Disease reactivation in non-risk organs at any time after completion of Stratum I
treatment
• Stratum III
• Patients from Stratum I who fulfill the following criteria:
• AD worse in risk organs after week 6 (after Initial Course 1), or AD worse or AD
intermediate in risk organs after week 12 (after Initial Course 2).
• Presence of unequivocally severe organ dysfunction at the above mentioned
evaluation points (hematological dysfunction, liver dysfunction, or both of them)
as
• Hb <70 g/L (<7.0 g/dl) and/or transfusion dependency
• PLT <20 x109/L (20,000/μL) and/or transfusion dependency (both criteria have
to be fulfilled) AND/OR
• Liver dysfunction (or digestive involvement with protein loss)
• Total protein <55 g/L or substitution dependency
• Albumin <25 g/L or substitution dependency (at least one of the two criteria
to be fulfilled)
• Stratum IV
• Patients from Stratum I or Stratum III who fulfill the following criteria:
• AD worse in risk organs after week 6 (after Initial Course 1), or AD worse or AD
intermediate in risk organs after week 12 (after Initial Course 2) of Stratum I
OR
• AD worse after the 2nd and 3rd 2-CdA/Ara-C course, and those AD worse or AD
intermediate after the 4th 2-CdA/Ara-C course of Stratum III AND
• Presence of unequivocally severe organ dysfunction at the above mentioned
evaluation points (hematological dysfunction, liver dysfunction, or both of them)
as defined in Table XI (see Section 10.3.1).
• Informed consent: All patients or their legal guardians (if the patient is <18
years of age) must sign an Ethics or institutional Review Board approved consent
form indicating their awareness of the investigational nature and the risks of
this study. When appropriate, younger patients will be included in all
discussions in order to obtain assent.
• Adequate organ function: Patients should have adequate hepatic, renal, cardiac
and pulmonary function to undergo reduced intensity HCT based upon local
institutional guidelines, or at a minimum meet requirements noted in eligibility
checklist Appendix A-VIII_1. However, significant hepatic and pulmonary
dysfunction, if secondary to underlying LCH disease activity, will not exclude
patients from protocol enrollment and should be discussed with the National PI
Coordinator and the Coordinating Principal Investigator.
• Stratum V
• All patients with verified diagnosis of LCH and MRI findings consistent with
ND-CNSLCH irrespective of previous treatments (also those not registered to other
Strata ofLCH-IV).
• Patients with isolated tumorous CNS-LCH (including isolated DI with mass lesion
in the hypothalamus-pituitary axis). In patients with already established
diagnosis of LCH and radiologic finding of CNS lesions compatible with LCH, a
biopsy of the lesion is not obligatory. In all other cases a biopsy of the lesion
is needed for inclusion into the study
• Stratum VI
-- Patients with newly diagnosed SS-LCH and localization other than "multifocal
bone",isolated tumorous CNS lesion, or isolated "CNS-risk" lesion.
• Stratum VII -- All patients registered in LCH IV (regardless of treatment) as long as
consent for longterm follow-up has not been withheld.
Exclusion Criteria:
• Stratum I
• Pregnancy (patients of child-bearing age must be appropriately tested before
chemotherapy)
• LCH-related permanent consequences (e.g. vertebra plana, sclerosing cholangitis,
lung fibrosis, etc.) in the absence of active disease
• Prior systemic therapy
• Stratum II
• Patients with progressive disease in risk organs
• Permanent consequences (e.g. sclerosing cholangitis, lung fibrosis, etc.) without
evidence of active LCH in the same organ or in any other locations
• No written consent of the patient or his/her parents or legal guardian
• Stratum III
• The presence of any of the following criteria will exclude the patient from the
study:
• Isolated sclerosing cholangitis without evidence of active hepatic LCH as the
only evidence of risk organ involvement.
• Inadequate renal function as defined by serum creatinine > 3x normal for age
• Stratum IV
• Pulmonary failure (requiring mechanical ventilation) not due to active LCH.
• Isolated liver sclerosis or pulmonary fibrosis, without active LCH.
• Uncontrolled active life-threatening infection.
• Decreased renal function with a GFR of less than 50ml/1.73m2/min.
• Pregnancy or active breast feeding
• Failure to provide signed informed consent
• Stratum VI
• Patients with SS-LCH who have an isolated tumorous CNS lesion (they are eligible
for Stratum V),
• Patients with isolated "CNS-risk" or multifocal bone lesions (they are eligible
for Stratum I, Group 2)
Neuromuscular Blockade in Patients With Severe Renal Impairment
This study is intended to be a single-site, prospective, randomized, double-blinded study
that intends to enroll a total of 60 patients with severe renal impairment undergoing surgery
with general endotracheal anesthesia at Parkland Hospital. Patients will be randomized to
receive either neostigmine (for reversal of cisatracurium) or sugammadex (for reversal of
rocuronium). A standardized anesthetic protocol that is usual and customary for the type of
operation the patient is having will be provided to the anesthesia teams of enrolled
subjects. The remainder of the anesthetic care of the subject will not deviate from the
standard of care. All patients will be monitored with continuous pulse oximetry
postoperatively for 24 hours.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Tiffany Moon
66760
All
18 Years to 80 Years old
Phase 2
This study is also accepting healthy volunteers
NCT03904550
STU-2018-0411
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Inclusion Criteria:
• 18-80 years old
• Severe renal impairment (CrCl < 30 mL/min)
• Undergoing non-emergent surgery that requires neuromuscular blockade
• Planned extubation in the operating room immediately after surgery
• American Society of Anesthesiologists (ASA) physical status classification 3 to 4
• Willing and able to consent in English or Spanish
• No personal history of neuromuscular disease
Exclusion Criteria:
• Age less than 18 or older than 80
• Patient does not speak English or Spanish
• Planned postoperative intubation/ventilation
• Allergy to sugammadex, neostigmine, glycopyrrolate, cisatracurium, or rocuronium
• Family or personal history of malignant hyperthermia
• Patient refusal
• Pregnant or nursing women
• "Stat" (emergent) cases
• Pre-existing muscle weakness of any etiology
• Patients on toremifene (a selective estrogen receptor modulator)
• Women on oral contraceptives who do not wish to use a non-hormonal method of
contraception for 7 days following surgery
Best Available Therapy Versus Autologous Hematopoetic Stem Cell Transplant for Multiple Sclerosis (BEAT-MS) (BEAT-MS)
This is a multi-center prospective rater-masked (blinded) randomized controlled trial of 156
participants, comparing the treatment strategy of Autologous Hematopoietic Stem Cell
Transplantation (AHSCT) to the treatment strategy of Best Available Therapy (BAT) for
treatment-resistant relapsing multiple sclerosis (MS). Participants will be randomized at a 1
to 1 (1:1) ratio.
All participants will be followed for 72 months after randomization (Day 0, Visit 0).
Call 214-648-5005 studyfinder@utsouthwestern.edu
Benjamin Greenberg
105091
All
18 Years to 55 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT04047628
STU-2020-0855
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Inclusion Criteria:
Participant(s) must meet all of the following criteria to be eligible for this study:
1. Diagnosis of Multiple Sclerosis (MS) according to the 2017 McDonald Criteria
2. (Kurtzke) Expanded Disability Status Scale (EDSS) ≥ 2.0 and ≤ 5.5 at the time of
randomization (Day 0)
3. T2 abnormalities on brain Magnetic Resonance Imaging (MRI) that fulfill the 2017
McDonald MRI criteria for dissemination in space
--A detailed MRI report or MRI images must be available for review by the site
neurology investigator.
4. Highly active treatment-resistant relapsing MS, defined as ≥ 2 episodes of treatment
failure in the 36 months prior to the screening visit (Visit -2). The two treatment
failure events need not occur during treatment with different Disease- modifying
Therapy (DMT), but must meet all the criteria as described below:
1. Each episode of treatment failure must occur following ≥ 3 months of treatment
with an FDA-approved DMT for relapsing forms of MS, or with rituximab or
ofatumumab, and
2. At least one episode of treatment failure must occur with an oral agent or a
monoclonal antibody, specifically: dimethyl fumarate (Tecfidera®), diroximel
fumarate, teriflunomide (Aubagio®), cladribine (Mavenclad®), daclizumab
(Zinbryta®), siponimod (Mayzent®), ozanimod, fingolimod (Gilenya®), rituximab
(Rituxan®), ocrelizumab (Ocrevus®), natalizumab (Tysabri®), alemtuzumab
(Campath®, Lemtrada®), or ofatumumab (Arzerra®), and
3. At least one episode of treatment failure must have occurred within the 12 months
prior to the screening visit (Visit -2), and
4. At least one episode of treatment failure must be a clinical MS relapse (see item
d.i. below). The other episode(s) must occur at least one month before or after
the onset of the clinical MS relapse, and must be either another clinical
MS relapse or MRI evidence of disease activity (see item d.ii. below):
i. Clinical MS relapse must be confirmed by a neurologist's assessment and documented
contemporaneously in the medical record. If the clinical MS relapse is not documented
in the medical record, it must be approved by the study adjudication committee, and
ii. MRI evidence of disease activity must include ≥ 2 unique active lesions on a brain
or spinal cord MRI. A detailed MRI report or MRI images must be available for review
by the site neurology investigator. A unique active lesion is defined as either of the
following:
• A gadolinium-enhancing lesion, or
• A new non-enhancing T2 lesion compared to a reference scan obtained not more than
24 months prior to the screening visit (Visit -2).
5. Candidacy for treatment with at least one of the following high efficacy DMTs:
Cladribine, natalizumab, alemtuzumab, ocrelizumab, rituximab, and ofatumumab (after
approval by the FDA for relapsing MS).
--Note: Rituximab, ofatumumab, and ocrelizumab are considered equivalent for
candidacy. Candidacy for treatment for each DMT is defined as meeting all of the
following:
• No prior treatment failure with the candidate DMT, and
• No contraindication to the candidate DMT, and
• No treatment with the candidate DMT in the 12 months prior to screening.
6. Insurance or public funding approval for MS treatment with at least one candidate DMT,
and
7. Ability to comply with study procedures and provide informed consent, in the opinion
of the investigator.
Exclusion Criteria:
Subject(s) who meet any of the following criteria will not be eligible for this study:
1. Diagnosis of primary progressive Multiple Sclerosis (MS) according to the 2017
McDonald criteria
2. History of neuromyelitis optica or anti-myelin oligodendrocyte glycoprotein (anti-MOG)
antibodies associated encephalomyelitis
3. Prior treatment with an investigational agent within 3 months or 5 half-lives,
whichever is longer
4. Either of the following within one month prior to randomization (Day 0):
1. Onset of acute MS relapse, or
2. Treatment with intravenous methylprednisolone 1000 mg/day for 3 days or
equivalent.
5. Initiation of natalizumab, alemtuzumab, ocrelizumab, or rituximab between screening
visit (Visit -2) and randomization (Day 0)
6. Brain MRI or Cerebrospinal fluid (CSF) examination indicating a diagnosis of
progressive multifocal leukoencephalopathy (PML)
7. History of cytopenia consistent with the diagnosis of myelodysplastic syndrome (MDS)
8. Presence of unexplained cytopenia, polycythemia, thrombocythemia or leukocytosis
9. History of sickle cell anemia or other hemoglobinopathy
10. Evidence of past or current hepatitis B or hepatitis C infection, including treated
hepatitis B or hepatitis C
-Note: Hepatitis B surface antibody following hepatitis B immunization is not
considered to be evidence of past infection.
11. Presence or history of mild to severe cirrhosis
12. Hepatic disease with the presence of either of the following:
1. Total bilirubin ≥ 1.5 times the upper limit of normal (ULN) or total bilirubin
• 3.0 times the ULN in the presence of Gilbert's syndrome, or
2. Alanine Aminotransferase (ALT) or Aspartate Aminotransferase (AST) ≥ 2.0 times
the ULN.
13. Evidence of HIV infection
14. Positive QuantiFERON •TB Gold or TB Gold Plus test results (e.g., blood test results
that detect infection with Mycobacterium tuberculosis) Note: A Purified Protein
Derivative (PPD) tuberculin test may be substituted for QuantiFERON •TB Gold or TB
Gold Plus test.
15. Active viral, bacterial, endoparasitic, or opportunistic infections
16. Active invasive fungal infection
17. Hospitalization for treatment of infections or parenteral (IV or IM) antibacterials,
antivirals, antifungals, or antiparasitic agents within the 30 days prior to
randomization (Day 0) unless clearance is obtained from an Infectious Disease
specialist
18. Receipt of live or live-attenuated vaccines within 6 weeks of randomization (Day 0)
19. Presence or history of clinically significant cardiac disease including:
1. Arrhythmia requiring treatment with any antiarrhythmia therapy, with the
exception of low dose beta blocker for intermittent premature ventricular
contractions
2. Coronary artery disease with a documented diagnosis of either:
• Chronic exertional angina, or
• Signs or symptoms of congestive heart failure.
3. Evidence of heart valve disease, including any of the following:
• Moderate to severe valve stenosis or insufficiency,
• Symptomatic mitral valve prolapse, or
• Presence of prosthetic mitral or aortic valve.
20. Left ventricular ejection fraction (LVEF) < 50%
21. Impaired renal function defined as Estimated glomerular filtration rate (eGFR) < 60
mL/min/1.73 m^2 according to the Chronic Kidney Disease Epidemiology Collaboration
(CKD-EPI) formula
22. Forced expiratory volume in one second (FEV1) <70% predicted (no bronchodilator)
23. Diffusing capacity of the lungs for carbon monoxide (DLCO) (corrected for Hgb) < 70%
predicted
24. Poorly controlled diabetes mellitus, defined as HbA1c >8%
25. History of malignancy, with the exception of adequately treated localized basal cell
or squamous skin cancer, or carcinoma in situ of the cervix.
-Note:Malignancies for which the participant is judged to be cured by therapy
completed at least 5 years prior to randomization (Day 0) will be considered on an
individual basis by the study adjudication committee.
26. Presence or history of any moderate to severe rheumatologic autoimmune disease
requiring treatment, including but not limited to the following:
• systemic lupus erythematous
• systemic sclerosis
• rheumatoid arthritis
• Sjögren's syndrome
• polymyositis
• dermatomyositis
• mixed connective tissue disease
• polymyalgia rheumatica
• polychondritis
• sarcoidosis
• vasculitis syndromes, or
• unspecified collagen vascular disease.
27. Presence of active peptic ulcer disease, defined as endoscopic or radiologic diagnosis
of gastric or duodenal ulcer
28. Prior history of AHSCT
29. Prior history of solid organ transplantation
30. Positive pregnancy test or breast-feeding
31. Inability or unwillingness to use effective means of birth control
32. Failure to willingly accept or comprehend irreversible sterility as a side effect of
therapy
33. Psychiatric illness, mental deficiency, or cognitive dysfunction severe enough to
interfere with compliance or informed consent
34. History of hypersensitivity to mouse, rabbit, or Escherichia coli-derived proteins
35. Any metallic material or electronic device in the body, or condition that precludes
the participant from undergoing MRI with gadolinium administration
36. Presence or history of ischemic cerebrovascular disorders, including but not limited
to transient ischemic attack, subarachnoid hemorrhage, cerebral thrombosis, cerebral
embolism, or cerebral hemorrhage
37. Presence or history of other neurological disorders, including but not limited to:
• central nervous system (CNS) or spinal cord tumor
• metabolic or infectious cause of myelopathy
• genetically-inherited progressive CNS disorder
• CNS sarcoidosis, or
• systemic autoimmune disorders potentially causing progressive neurologic disease
or affecting ability to perform the study assessments.
38. Presence of any medical comorbidity that the investigator determines will
significantly increase the risk of treatment mortality, or
39. Presence of any other concomitant medical condition that the investigator deems
incompatible with trial participation.
Procedure: Autologous Hematopoietic Stem Cell Transplantation, Biological: Best Available Therapy (BAT)
31P-MRS Imaging to Assess the Effects of CNM-Au8 on Impaired Neuronal Redox State in Multiple Sclerosis (REPAIR-MS)
REPAIR-MS is a single-center open label, sequential group, investigator and patient blinded
study to assess the CNS metabolic effects, safety, pharmacokinetics, and pharmacodynamics of
CNM-Au8 in patients who have been diagnosed with Multiple Sclerosis (MS) within fifteen (15)
years of Screening. The primary endpoint is the ratio of the oxidized to reduced form of
nicotinamide adenine dinucleotide (NAD+:NADH) measured non-invasively by 31phosphorous
magnetic resonance spectroscopy (31P-MRS).
Call 214-648-5005 studyfinder@utsouthwestern.edu
Benjamin Greenberg
105091
All
18 Years to 55 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT03993171
STU-2019-0992
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Inclusion Criteria:
1. At least 18 years of age and up to 55 years (inclusive) of age at Baseline.
2. Clinical diagnosis of Multiple Sclerosis (MS) who have had Relapsing MS (RMS) no
longer than 15 years from diagnosis.
3. Stable treatment with natalizumab for at least the prior six (6) months.
4. Stable disease activity over the prior six (6) months.
5. All hematological parameters and biochemical parameters deemed stable or transient in
nature.
6. Able to understand and give written informed consent.
Exclusion Criteria:
1. Patients with a clinical relapse requiring systemic steroid treatment within the prior
six (6) months.
2. Patients treated with any other MS therapy other than natalizumab; or treated with
clemastine fumarate.
3. Patients with a history of significant other major medical condition that may
interfere with the conduct of the study or interpretation of the study results.
4. Patients who may have difficulty complying with the protocol and/or study procedures.
5. Patient with clinically significant abnormalities in hematology, blood chemistry, ECG,
or physical examination not resolved by the Baseline visit which according to
Investigator can interfere with study participation.
6. Patients with clinically significant hepatic or renal dysfunction or clinical
laboratory findings that would limit the interpretability of change in liver or kidney
function, or those with low platelet counts (< 150 x 109 per liter) or eosinophilia
(absolute eosinophil count of ≥500 eosinophils per microliter).
7. Patients with a prior history of, or positive serological assay for the presence of
HIV infection, or laboratory evidence of active or chronic infection with hepatitis C
(HCV) or hepatitis B (HBV).
8. Patients participating in any other investigational drug trial or using an
investigational drug (within 12 weeks prior to screening and thereafter)
9. Positive screen for drugs of abuse or known alcohol abuse.
10. Females who are pregnant, have a positive pregnancy test, are nursing, or who plan to
get pregnant during the course of this clinical trial or within 6 months of the end of
this trial.
11. Women of child-bearing potential, or men, who are unwilling or unable to use accepted
methods of birth control during the study and for 6 months following completion of
study participation.
12. Patients with implanted metal objects in their body that may be affected by an MRI
procedure.
13. Patients who are claustrophobic or otherwise unlikely to be able to complete the MRI
scanning procedures.
14. Patients with a history of gold allergy.
15. Patient is considered a suicide risk in the opinion of the Investigator, has
previously made a suicide attempt, or is currently demonstrating active suicidal
ideation.
16. Any active ophthalmological cause for retinal damage other than MS or based on the
Investigator's judgment any other ophthalmic diseases that would confound the study
results or optical coherence tomography assessment.
17. PRN use of stimulant medications including: amphetamine, dextroamphetamine,
lisdexamfetamine, methylphenidate, or modafinil.
Drug: gold nanocrystals
Relapsing Remitting Multiple Sclerosis, Brain and Nervous System
Efficacy and Safety Study of MYOBLOC® in the Treatment of Adult Lower Limb Spasticity
Phase 2/3, randomized, double-blind, placebo-controlled, single-treatment, multicenter trial
assessing the efficacy and safety of MYOBLOC for the treatment of lower limb spasticity, in
adults followed by an open-label extension safety trial.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Fatma Gul
12837
All
18 Years to 80 Years old
Phase 2/Phase 3
This study is NOT accepting healthy volunteers
NCT04099667
STU-2020-0097
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Inclusion Criteria:
1. Able to understand the potential risks and benefits, the study requirements, and
provide written informed consent before enrollment into the study; or if unable, the
subject's Legally Authorized Representative (LAR) may provide written informed
consent.
2. Male or female ≥18 to maximum of 80 years of age, inclusive.
3. Lower limb spasticity due to stroke, traumatic brain injury, or spinal cord injury
that occurred ≥6 months prior to randomization. Eligible subjects may have lower limb
monoplegia or hemiplegia. Subjects with cerebral palsy are eligible for study
enrollment.
4. Ambulatory (with or without the use of a walking assistive device).
5. Modified Ashworth Scale (MAS) score ≥2 in the ankle plantar flexors of the affected
lower limb at screening and at baseline.
6. In the Investigator's opinion, the subject will be available and able to comply with
the study requirements for at least 1 year, based on the subject's overall health and
disease prognosis.
7. In the Investigator's opinion, the subject will be willing and able to comply with all
requirements of the protocol, including completion of study questionnaires. A
caregiver may be designated to assist with the physical completion of
questionnaires/scales.
Exclusion Criteria:
1. Quadriplegia/tetraplegia, lower limb diplegia or triplegia.
2. Uncontrolled epilepsy or any type of seizure disorder with a seizure(s) within the
previous year.
3. Neuromuscular disorders including, but not limited to, amyotrophic lateral sclerosis
(ALS), primary lateral sclerosis (PLS), multiple sclerosis (MS), myasthenia gravis, or
muscular dystrophy.
4. History of major joint contracture(s), in which, based on the Investigator's
assessment, the contracture(s) significantly contribute(s) to joint immobility in the
affected lower limb.
5. Unresolved fracture(s) in the affected lower limb.
6. Severe atrophy in the affected lower limb.
7. Known hypersensitivity to botulinum toxins type A or B or to any MYOBLOC solution
components.
8. Concomitant use or exposure within 5 half-lives of randomization of the following:
aminoglycoside antibiotics, curare-like agents, or other agents that may interfere
with neuromuscular function.
9. Treatment with a neurolytic agent (e.g., phenol, alcohol blocks) to the affected lower
limb within 1 year before randomization.
10. Presence of a spinal stimulator or intrathecal baclofen pump that has not been turned
off within 30 days before screening.
11. Changes to treatment regimen or any new treatment with oral antispasmodics and/or
muscle relaxants within 30 days before randomization.
12. Initiation of physical and/or occupational therapy <30 days before randomization.
Subjects receiving physical and/or occupational therapy ≥30 days before randomization
must be willing to maintain their therapy regimen through Week 4 of the DBP.
13. Application of an ankle-foot orthosis (AFO) <30 days before randomization. Subjects
regularly using an AFO ≥30 days before randomization must be willing to maintain use
of the AFO through Week 4 of the Double-Blind Period.
14. Prior botulinum toxin type A (BoNT/A) or B (BoNT/B) treatment in the affected lower
limb within 24 weeks before screening. Prior BoNT/A or BoNT/B treatment in areas other
than the affected lower limb is not exclusionary but must have occurred at least 12
weeks before screening. Prior toxin exposure must have been well tolerated and without
any significant long-term side effects in the case of repeated prior exposure.
15. Subjects should not receive nor have any plans to receive any botulinum toxin
treatment, other than the study drug (MYOBLOC), from the point informed consent is
obtained until participation in the study is complete.
16. Severe dysphagia (i.e., inability to swallow liquids, solids or both without choking
or medical intervention), or dysphagia with a history of aspiration pneumonia, within
6 months before screening.
17. Prior surgery to treat spasticity in the affected lower limb (i.e., tendon lengthening
or tendon transfer).
18. Any anticipated or scheduled surgery during the study period, with the exception of
dermatological procedures performed under local anesthesia for the purposes of
removing precancerous and cancerous lesions.
19. Major surgery within 3 months before screening.
20. Pregnancy or breastfeeding.
21. Females of childbearing potential must agree to practice a medically acceptable method
of contraception (e.g., intrauterine device, hormonal contraception started at least
one full cycle before study enrollment or barrier method in conjunction with
spermicide) for the duration of the study (including 2 months after study completion).
For the purposes of this study, all females are considered to be of childbearing
potential unless they are confirmed by the Investigator to be post-menopausal (at
least 1 year since last menses and laboratory test confirmation), biologically
sterile, or surgically sterile (e.g., hysterectomy with bilateral oophorectomy, tubal
ligation).
22. History of drug or alcohol abuse within 6 months before screening.
23. Obstructive pulmonary disease with forced expiratory volume in 1 second (FEV1)/forced
vital capacity (FVC) <70%.
24. Slow vital capacity (SVC) <60% of predicted.
25. Chronic or current use of inhaled corticosteroids.
26. Ventilator dependence (i.e., 24-hour ventilator dependence when intubated, or due to a
failure to wean the subject from the ventilator while hospitalized in the intensive
care unit or respiratory care center). Subjects who use oxygen on an as-needed basis
or during sleeping hours only via a nasal cannula are eligible for the study.
27. Infection at the planned sites of injection.
28. Treatment with an investigational drug, device, or biological agent within 30 days
before screening or while participating in this study.
29. Malignancy diagnosed 3 months before screening.
30. Has one or more screening clinical laboratory test values outside the reference range
that, in the opinion of the Investigator, are clinically significant, or any of the
following :
• Serum creatinine >1.5 times the upper limit of normal (ULN);
• Serum total bilirubin > 1.5 times ULN;
• Serum alanine aminotransferase or aspartate aminotransferase >2 times ULN.
31. Has any of the following cardiology findings at screening:
• Abnormal ECG that is, in the Investigator's opinion/evaluation, clinically
significant;
• PR interval >220 ms;
• QRS interval >130 ms;
• QTcF interval >450 ms (for men), or >470 ms (for women) (QT corrected using
Fridericia's method);
• Second-or third-degree atrioventricular block;
• Any rhythm, other than sinus rhythm, that is interpreted or assessed by the
Investigator to be clinically significant.
32. Any other medical illness, condition, or clinical finding that, in the opinion of the
Investigator and/or the Sponsor, would put the subject at undue risk.
Extension Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Who Previously Participated in a Study With Nusinersen (ONWARD)
The primary objective of this study is to evaluate the long-term safety and tolerability of
nusinersen administered intrathecally at higher doses to participants with spinal muscular
atrophy (SMA) who previously participated in study 232SM203 (NCT04089566).
The secondary objective of this study is to evaluate the long-term efficacy of nusinersen
administered intrathecally at higher doses to participants with SMA who previously
participated in study 232SM203 (NCT04089566).
Call 214-648-5005 studyfinder@utsouthwestern.edu
Susan Iannaccone
13463
All
Not specified
Phase 3
This study is NOT accepting healthy volunteers
NCT04729907
STU-2021-0777
Show full eligibility criteria
Hide eligibility criteria
Key
Inclusion Criteria:
• Completed the Day 302 visit in study 232SM203 (NCT04089566) in accordance with the
study protocol.
Key
Exclusion Criteria:
• Treatment with another investigational therapy or enrollment in another interventional
clinical study.
• Treatment with an approved therapy for SMA that is inconsistent with protocol
requirements for allowed or disallowed concomitant therapies
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
Drug: Nusinersen
Muscular Atrophy, Spinal, Brain and Nervous System
Testicular Tissue Cryopreservation for Fertility Preservation
Testicular tissue cryopreservation is an experimental procedure where a young boy's
testicular tissue is retrieved and frozen. This technique is reserved for young male patients
who are not yet producing mature sperm, with the ultimate goal that their tissue may be used
in the future to restore fertility when experimental techniques emerge from the research
pipeline.
• Be male at any age.
• Be scheduled to undergo surgery, chemotherapy, drug treatment and/or radiation for the
treatment or prevention of a medical condition or malignancy with risk of causing
permanent and complete loss of subsequent testicular function.
• Or, have a medical condition or malignancy that requires removal of all or part of one
or both testicles.
• Have newly diagnosed or recurrent disease. Those who were not enrolled at the time of
initial diagnosis (i.e., patients with recurrent disease) are eligible if they have
not previously received therapy that is viewed as likely to result in complete and
permanent loss of testicular function.
• Have two testicles if undergoing elective removal of all or part of a testicle for
fertility preservation only. Note: removal of both testicles will limit fertility
preservation options.
• Sign an approved informed consent and authorization permitting the release of personal
health information. The patient and/or the patient's legally authorized guardian must
acknowledge in writing that consent for specimen collection has been obtained, in
accordance with institutional policies approved by the U.S. Department of Health and
• Consent for serum screening tests for infectious diseases [HIV-1, HIV-2, Hepatitis B,
Hepatitis C], to be performed at the time of testicular tissue harvesting.
• Undergo a full history and physical examination and obtain standard pre-operative
clearance (based on the most recent ACC/AHA Guideline for Perioperative Cardiovascular
Evaluation for Noncardiac Surgery) as determined by their primary surgeon.
• Participating in long term follow-up is a requirement of the protocol.
Exclusion Criteria:
• Diagnosed with psychological, psychiatric, or other conditions which prevent giving
fully informed consent.
• Diagnosed with an underlying medical condition that significantly increases their risk
of complications from anesthesia and surgery.
Procedure: Testicular biopsy
Lymphoma, Sarcoma, Cancer, Mycosis Fungoides, Brain and Nervous System, Other, Eye and Orbit, Anklylosing Spondylitis, Anus, Bones and Joints, Breast - Male, Carcinoid Tumor, Cardiovascular, Colon, Esophagus, Gall Bladder, Head and Neck, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung/Thoracic, Nose, Other Digestive Organ, Other Endocrine System, Other Male Genital, Other Respiratory and Intrathoracic Organs, Other Skin, Other Urinary, Pancreas, Prostate, Rectum, Stomach, Throat, Thyroid, Urinary Bladder, Leukemia, Other, Hodgkins Lymphoma, Heart, Leukemia, Not Otherwise Specified, Lymphoid Leukemia, Myeloid and Monocytic Leukemia, Non-Hodgkins Lymphoma, Other Hematopoietic, Small Intestine, Soft Tissue, Unknown Sites, Ill - Defined Sites, Autoimmune Disorders
Comparing Virtual Reality (VR) to Non-VR for Decreasing Preoperative/Procedural Anxiety
The primary objective of the study is to utilize the modified Yale Preoperative Anxiety scale
(mYPAS), a validated preoperative/procedural anxiety score, to measure preoperative anxiety
via distraction in pediatric oncology patients undergoing port access.
The hypothesis is that using Virtual Reality (VR) will objectively decrease anxiety scores
measured by mYPAS by five percent (primary outcome).
The secondary outcome will be the parents or the legally authorized representative (LAR)
subjective reports of anxiety with the use of VR.
The Kind VR device is used in house at Children's Health in the Dallas and Plano campuses.
The VR device used in this study qualifies as exempt from FDA IDE regulations. It is a
non-significant risk, non-invasive, interactive video device the user wears like goggles. The
study carries minimal risks to the subjects and is designed to minimize patient discomfort
from placement or motion sickness. Furthermore, the device has disposable covers for
protection against infection and can be sanitized between uses, once the disposable covers
are removed. Children's Health System of Texas (CHST) and this research group are not
partnering entities with the Kind VR, and the Kind VR device is not being studied. The effect
of virtual reality (VR) on preprocedural anxiety as measured by questionnaires and the
observations of the modified Yale Preoperative Anxiety Scale (mYPAS) is being studied
Most patients coming to the Clinic of Cancer and Blood Disorders (CCBD) are under chronic
care for their ongoing disease and are likely to be coming to the CCBD at least twice in a
6-month period. The CCBD schedule will be reviewed by the researchers for patients age 5-12,
requiring port access at least twice during the next six-month period. Patient families whose
child meets the basic screening criteria, and have no exclusion criteria, will be approached
privately as possible participants in the study. Up to 100 subjects will be enrolled over a
2-year period. Once the subject/parent or LAR agrees to participate, study staff will
randomize the subjects into which standard of care distraction method for anxiety management
they will receive first in this study.
• Any patient of the Children's Medical Center CCBD
• 5-12 years of age
• Patient requiring their port accessed twice or more within a 6 months period
• Ability to understand and the willingness to sign a written informed consent.
Exclusion Criteria
• Subjects younger than 5 and older than 12
• Patients requiring recovery in PICU or sites other than PACU
• If parents or subject is not willing to participate
• Subjects with severe developmental delays and subjects with developmental challenges
preventing them from keeping the VR device on are also excluded
• Patients who will not be in CCBD for port access at least twice in 6 months
Other: Virtual reality
Lymphoma, Sarcoma, Multiple Myeloma, Brain and Nervous System, Eye and Orbit, Anus, Bones and Joints, Breast - Female, Breast - Male, Carcinoid Tumor, Cervix, Colon, Corpus Uteri, Ear, Esophagus, Gall Bladder, Head and Neck, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung/Thoracic, Melanoma, skin, Nose, Ovary, Pancreas, Prostate, Rectum, Stomach, Throat, Thyroid, Urinary Bladder, Uterine (Endometrial), Vulva, Leukemia, Other, Hodgkins Lymphoma, Leukemia, Not Otherwise Specified, Lymphoid Leukemia, Myeloid and Monocytic Leukemia, Non-Hodgkins Lymphoma, Small Intestine, Soft Tissue, Preoperative Anxiety
The iCat2, GAIN (Genomic Assessment Informs Novel Therapy) Consortium Study
This research study is evaluating the use of specialized testing of solid tumors including
sequencing. The process of performing these specialized tests is called tumor profiling. The
tumor profiling may result in identifying changes in genes of the tumor that indicate that a
particular therapy may have activity. This is called an individualized cancer therapy (iCat)
recommendation. The results of the tumor profiling and, if applicable, the iCat
recommendation will be returned.
• Age -- Age ≤ 30 years at time of initial qualifying solid tumor diagnosis
• Diagnosis -- Histologic diagnosis of solid malignancy (excluding brain tumors and
lymphoma) that meets at least one of the following criteria:
• Refractory, defined as tumor progression after initiation of standard first line
therapy without having achieved a prior partial or complete remission OR Biopsy
proven residual disease at the completion of planned standard initial front-line
therapy.
• Recurrent, defined as tumor progression after achieving a prior partial or
complete remission
• Newly diagnosed high risk disease, defined as having an expected event free
survival of < 50% at 2 years.
• Lacks definitive diagnosis or classical genomic findings after histologic review
and standard molecular testing (rare tumor group).
• Examples include (eligibility not limited to these examples):
• Histology typically associated with a fusion in which fusion is not detected.
• Ewing-like sarcoma
• Undifferentiated sarcoma
• Inflammatory myofibroblastic tumor without ALK fusion
• Infantile fibrosarcoma without NTRK fusion
• Specimen Samples
• Sufficient tumor specimen available to meet the minimum requirements for
profiling from diagnosis or progression / recurrence
--- OR
• Surgery / biopsy planned as part of clinical care that is anticipated to yield
sufficient material to meet the minimum requirements for profiling; OR
• Patient has already had molecular profiling and patient has not yet started
matched targeted therapy based on the report .
Exclusion Criteria:
• No Therapy Planned
-- Patients who have declined further anticancer therapy will be excluded.
• Performance Status
-- Patients with Lansky (age < 16 years) or Karnofsky (age ≥16 years) score < 50 will
be excluded.
• Life Expectancy -- Patients with anticipated life expectancy < 3 months will be
excluded.
Genetic: Genetic testing and GAIN report
Sarcoma, Pediatric Solid Tumor, Brain and Nervous System, Other, Eye and Orbit, Anklylosing Spondylitis, Anus, Bones and Joints, Breast - Female, Breast - Male, Carcinoid Tumor, Cervix, Colon, Corpus Uteri, Ear, Esophagus, Gall Bladder, Head and Neck, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung/Thoracic, Melanoma, skin, Nose, Other Digestive Organ, Other Endocrine System, Other Female Genital, Other Male Genital, Other Respiratory and Intrathoracic Organs, Other Skin, Other Urinary, Ovary, Pancreas, Prostate, Rectum, Stomach, Throat, Thyroid, Urinary Bladder, Uterine (Endometrial), Vulva, Heart, Kaposis sarcoma, Small Intestine, Soft Tissue, Ill - Defined Sites
Immunotherapy With Nivolumab and Ipilimumab Followed by Nivolumab or Nivolumab With Cabozantinib for Patients With Advanced Kidney Cancer, The PDIGREE Study
This phase III trial compares the usual treatment (treatment with ipilimumab and nivolumab
followed by nivolumab alone) to treatment with ipilimumab and nivolumab, followed by
nivolumab with cabozantinib in patients with untreated renal cell carcinoma that has spread
to other parts of the body. The addition of cabozantinib to the usual treatment may make it
work better. Immunotherapy with monoclonal antibodies, such as nivolumab and ipilimumab, may
help the body's immune system attack the cancer, and may interfere with the ability of tumor
cells to grow and spread. Cabozantinib may stop the growth of tumor cells by blocking some of
the enzymes needed for cell growth. It is not yet known how well the combination of
cabozantinib and nivolumab after initial treatment with ipilimumab and nivolumab works in
treating patients with renal cell cancer that has spread to other parts of the body.
• STEP I REGISTRATION CRITERIA
• Histologically documented renal cell carcinoma with clear cell component, including
patients who have sarcomatoid features.
• Any metastatic disease, including visceral, lymph node, other soft tissue and bone,
measurable per RECIST 1.1.
• Measurable disease as defined in the protocol.
• Must be intermediate or poor risk patient per International Metastatic Renal Cell
Carcinoma Database (IMDC) criteria (1 or more of the following: Karnofsky performance
status [KPS] < 80, < 1 year from diagnosis [including initial nephrectomy] to systemic
treatment for metastatic disease, hemoglobin less than lower limit of normal [LLN],
corrected calcium concentration greater than upper limit of normal [ULN], absolute
neutrophil count greater than ULN, platelet count > ULN).
• Central nervous system (CNS) disease permitted, if stable and not otherwise causing
symptoms or needing active treatment.
• Karnofsky performance status >= 70%.
• No prior treatment with PD-1, PD-L1, or CTLA-4 targeting agents (including but not
limited to nivolumab, pembrolizumab, pidilizumab, durvalumab, atezolizumab,
tremelimumab, and ipilimumab), or any other drug or antibody specifically targeting
T-cell co-stimulation or checkpoint pathways. The only exception is for prior
treatment with nivolumab or other PD-1/PD-L1/CTLA-4 targeting therapy on pre- or
post-operative trials, as long as > 1 year since completion of systemic therapy.
• No prior previous systemic therapy for renal cell carcinoma (prior HD IL-2 [> 28 days]
and prior adjuvant sunitinib > 180 days since completion and prior immunotherapy as
above are allowed).
• No cancer therapy less than 28 days prior to registration; this includes radiation
therapy, except for bone lesions less than 14 days prior to registration. There must
be a complete recovery and no ongoing complications from radiotherapy.
• Not pregnant and not nursing, because this study involves an agent that has known
genotoxic, mutagenic and teratogenic effects. Therefore, for women of childbearing
potential only, a negative serum or urine pregnancy test done =< 14 days prior to
registration is required.
• Age >= 18 years
• Absolute neutrophil count (ANC) >= 1,500/mm^3.
• Platelet count >= 100,000/mm^3.
• Hemoglobin >= 8 g/dL.
• Calculated (Calc.) creatinine clearance >= 30 mL/min.
• Urine protein =< 1+ or urine protein to creatinine (UPC) ratio < 1.
• Total bilirubin =< 1.5 x upper limit of normal (ULN).
• Aspartate aminotransferase/alanine aminotransferase (AST/ALT) =< 2.5 x upper limit of
normal (ULN) or < 5 x ULN if hepatic metastases present.
• STEP 2 REGISTRATION ELIGIBILITY CRITERIA
• Successful completion of at least 1 cycle of ipilimumab/nivolumab.
• Resolution of any treatment-related adverse events to grade 1 or less per dose
modification section (this criteria does not include any adverse events [AEs] not
attributable to treatment which are present due to disease). Exceptions for this
criteria include patients receiving replacement hormone treatments (such as
levothyroxine for treatment-related hypothyroidism or glucocorticoid replacement for
adrenal insufficiency). Please contact study chair if further discussion is needed.
• No more than 70 days from last dose of ipilimumab/nivolumab.
Exclusion Criteria:
• Active autoimmune disease requiring ongoing therapy.
• Ongoing acute toxicity > grade 2 from previous treatment.
• History of severe allergic, anaphylactic or other hypersensitivity reactions to
chimeric or humanized antibodies.
• History of human immunodeficiency virus (HIV) or active hepatitis B/C, or active
tuberculosis (purified protein derivative [PPD] response without active TB is
allowed).
• Concurrent use of immunosuppressive medication including prednisone above 10 mg daily.
• Uncontrolled adrenal insufficiency.
• Uncontrolled hypertension (systolic blood pressure [BP] >150 mmHg or diastolic BP > 90
mmHg).
• Major surgery less than 28 days prior to registration.
• Any serious non-healing wound, ulcer, or bone fracture within 28 days prior to
registration.
• Any arterial thrombotic events within 180 days prior to registration.
• Clinically significant hematuria, hematemesis, or hemoptysis within 12 weeks prior to
registration.
• Cavitating pulmonary lesions or known endotracheal or endobronchial disease
manifestations.
• Lesions encasing or invading any major blood vessels (this does not include tumor
thrombus extending into/through renal vein/inferior vena cava [IVC]). Patients with
tumor thrombus extending into/through renal vein are considered eligible.
• Moderate of severe hepatic impairment (Child-Pugh B or C).
• Any history of untreated pulmonary embolism or deep venous thrombosis (DVT) in the 180
days prior to registration. (Any asymptomatic, treated pulmonary embolism or
asymptomatic, treated deep venous thrombosis > 30 days prior to registration allowed).
• Corrected QT interval calculated by the Fridericia formula (QTcF) > 500 ms.
• Unstable cardiac arrhythmia within 6 months prior to registration.
• Any gastrointestinal (GI) bleeding =< 180 days, hemoptysis, or other signs of
pulmonary hemorrhage =< 90 days prior to registration.
• History of abdominal fistula, gastrointestinal perforation, intra-abdominal abscess,
bowel obstruction, or gastric outlet obstruction within 180 days prior to
registration.
• Active peptic ulcer disease, inflammatory bowel disease, or malabsorption syndrome
within 28 days prior to registration.
• Untreated hypothyroidism, evidence of pancreatitis, history of organ transplant, or
history of congenital QT syndrome.
• Active treatment with coumarin agents (e.g., warfarin), direct thrombin inhibitors
(e.g., dabigatran), direct Xa inhibitor betrixaban or platelet inhibitors (e.g.,
clopidogrel) within 5 days of registration. Allowed anticoagulants include:
prophylactic use of low-dose aspirin for cardio-protection (per local applicable
guidelines) and low-dose low molecular weight heparins (LMWH), therapeutic doses of
LMWH or anticoagulation with direct factor Xa inhibitors rivaroxaban, edoxaban,
apixaban. Allowed also in patients with known brain metastases who are on a stable
dose of the anticoagulant for at least 1 week prior to registration without clinically
significant hemorrhagic complications from the anticoagulation regimen or the tumor.
• Significant cardiac ischemia events (ST elevation myocardial infarction [STEMI] or
non-ST elevation myocardial infarction [NSTEMI]) within 6 months or active NY Heart
Association class 3-4 heart failure symptoms
Clear Cell Renal Cell Carcinoma, Metastatic Malignant Neoplasm in the Bone, Sarcomatoid Renal Cell Carcinoma, Metastatic Malignant Neoplasm in the Soft Tissues, Stage IV Renal Cell Cancer AJCC v8, Metastatic Malignant Neoplasm in the Viscera, Metastatic Malignant Neoplasm in the Lymph Nodes
• Diagnosis of EBV+ disorder
• Eastern Cooperative Oncology Group performance status <= 3 for participants aged >= 16
years; Lansky score >= 20 for participants from 1 year to < 16 years
• Adequate organ function test results, unless organ dysfunction is considered to be due
to the underlying EBV-associated disease by the investigator
Cohort-specific
Inclusion Criteria:
• For participants with PID LPD:
• Newly diagnosed or relapsed/refractory LPD confirmed by biopsy-proven EBV+ LPD or
positive cerebrospinal fluid (CSF) cytology with or without radiographically
measurable intracranial disease with EBV detected in CSF
• Participant must have systemic measurable disease and/ or CNS measurable disease
• Definitive therapy (eg, allogeneic HCT, gene therapy) for the underlying PID is
planned
• Participants with newly diagnosed disease should be ineligible for standard
first-line therapy for EBV+ LPD, as determined by the investigator
• For participants with AID LPD:
• Newly diagnosed or relapsed/refractory LPD confirmed by biopsy-proven EBV+ LPD or
positive CSF cytology, with or without radiographically measurable intracranial
disease, with EBV detected in CSF
• Participant must have systemic measurable disease and/ or CNS measurable disease
• Participants who are human immunodeficiency virus positive (HIV+) must meet both
of the following criteria: Have an HIV viral load assessed by reverse
transcription-polymerase chain reaction (RT-PCR) below the lower limit of
detection and CD4 >= 50 cells/μL within 6 months prior to the first dose of
tabelecleucel
• Participants with newly diagnosed disease should be ineligible for standard
first-line therapy for EBV+ LPD, as determined by the investigator
• For participants with CNS PTLD:
• Newly diagnosed or relapsed/refractory EBV+ CNS PTLD histologically confirmed by
biopsy-proven EBV+ CNS PTLD or positive CSF cytology with or without
radiographically measurable intracranial disease with EBV detected in CSF
• Participant may have systemic and CNS disease or CNS disease only
• Participants with newly diagnosed disease should be ineligible for standard
first-line therapy for EBV+ LPD, as determined by the investigator
• For participants with EBV+ PTLD, where standard first line therapy (rituximab and/or
chemotherapy) is not appropriate, including CD20-negative disease:
• Newly diagnosed, biopsy-proven EBV+ PTLD
• Ineligible for standard first-line therapy for EBV+ PTLD, as determined by the
investigator
• Participants must have systemic disease measurable per Lugano Classification
criteria, except when contraindicated or mandated by local practice, then MRI may
be used.
• For participants with sarcoma, including LMS:
• Newly diagnosed or failed systemic first-line therapy for EBV+ sarcoma.
Participants with newly diagnosed disease should be ineligible for standard
first-line therapy for EBV+ sarcoma, as determined by the investigator.
• Biopsy-proven EBV+ sarcoma
• Measurable disease using diagnostic PET/CT and/or MRI following RECIST 1.1
criteria
• For participants with CAEBV:
• Newly diagnosed or previously treated CAEBV
• Detectable EBV viremia on at least 2 occasions at a minimum of 90 days apart
• At least 3 active clinical findings (per Kimura H, et al. Front Immunol.
2017;8:1867) as: Fever >= 38.5°C; splenomegaly, lymphadenopathy, and/or
hepatomegaly; cytopenia affecting at least 2 or 3 lineages in the peripheral
blood (hemoglobin < 9 g/dL, platelets < 100 × 10^3/mL, neutrophils < 1 ×
10^3/mL); hypogammaglobulinemia; hemophagocytosis; hepatitis; neuropathy; rash;
and hydroa vacciniforme
• For participants with EBV+ viremia with HLH:
• Newly diagnosed or previously treated EBV+ viremia with HLH
• A molecular diagnosis consistent with HLH-2004 trial (per Henter JI, et al.
Pediatr Blood Cancer. 2007;48:124-31) OR 5 or more of the clinical symptoms (per
Jordan MB, et al. Blood. 2011;118:4041-4052): Fever >= 38.5°C; splenomegaly;
cytopenia affecting at least 2 or 3 lineages in the peripheral blood (hemoglobin
< 9 g/dL, platelets < 100 × 10^3/mL, neutrophils < 1 × 10^3/mL);
hypertriglyceridemia (fasting >= 265 mg/dL) and/or hypofibrinogenemia (<= 150
mg/dL); hemophagocytosis in bone marrow, spleen, lymph nodes, or liver; low or
absent natural killer cell (NK-cell) activity; ferritin >= 500 ng/mL; and
elevated soluble CD25
Exclusion Criteria:
• Burkitt, T-cell (except in the setting of HLH), natural killer/T-cell lymphoma/LPD,
Hodgkin, or transformed lymphoma
• Serious known active infections, defined as ongoing uncontrolled adenovirus infection
or infections requiring systemic therapy at the time of enrollment
• Suspected or confirmed Grade >= 2 acute graft-versus-host disease (GvHD) per the
Center for International Blood and Marrow Transplant Research (CIBMTR) consensus
grading system or extensive chronic GvHD per National Institutes of Health (NIH)
consensus criteria at the time of the enrollment
• Need for vasopressor or ventilatory support
• Prior therapy (in order of increasing washout period) prior to enrollment as:
• Within 4 weeks or 5 half-lives (whichever is shorter) for any investigational
product and/ or any chemotherapy (systemic or intrathecal), targeted small
molecule therapy, or antibody/biologic therapy. Note: prior anti-CD20 antibody
use is permitted within the washout period if a subsequent disease response
assessment indicates disease progression
• Within <= 8 weeks for cellular therapies (EBV-CTLs, chimeric antigen receptor
therapies directed at T cells or T-cell subsets, donor lymphocyte infusion, other
CTLs); and/or therapies which could impact tabelecleucel function (anti-thymocyte
globulin, alemtuzumab)
• Unwilling to use protocol specified contraceptive methods
• Women who are pregnant or breastfeeding
• Ongoing need for daily steroids of > 0.5 mg/kg prednisone or glucocorticoid
equivalent, ongoing methotrexate, or extracorporeal photopheresis (protocol-specified
dexamethasone is permitted and concludes by the time of enrollment)
• For participants with PID LPD or AID LPD: history of prior allogeneic HCT or solid
organ transplant
Biological: Tabelecleucel
Sarcoma, Leiomyosarcoma, Stem Cell Transplant Complications, Lymphoproliferative Disorders, Solid Organ Transplant Complications, Allogeneic Hematopoietic Cell Transplant, Brain and Nervous System, Epstein-Barr Virus (EBV)-Associated Diseases, EBV+ Lymphoproliferative Disease With Primary Immunodeficiency (PID LPD), EBV+ Lymphoproliferative Disease With Acquired (Non-congenital) Immunodeficiency (AID LPD), EBV+ Posttransplant Lymphoproliferative Disease in Central Nervous System (CNS PTLD), EBV+ Post-transplant Lymphoproliferative Disease (EBV+ PTLD), EBV+ Sarcomas, Chronic Active Epstein-Barr Virus (CAEBV), Chronic Active Epstein-Barr Virus With Hemophagocytic Lymphohistiocytosis (HLH), Lymphohistiocytosis, Hemophagocytic
Thoracotomy Versus Thoracoscopic Management of Pulmonary Metastases in Patients With Osteosarcoma
This phase III trial compares the effect of open thoracic surgery (thoracotomy) to
thoracoscopic surgery (video-assisted thoracoscopic surgery or VATS) in treating patients
with osteosarcoma that has spread to the lung (pulmonary metastases). Open thoracic surgery
is a type of surgery done through a single larger incision (like a large cut) that goes
between the ribs, opens up the chest, and removes the cancer. Thoracoscopy is a type of chest
surgery where the doctor makes several small incisions and uses a small camera to help with
removing the cancer. This trial is being done evaluate the two different surgery methods for
patients with osteosarcoma that has spread to the lung to find out which is better.
• Patients must be < 50 years at the time of enrollment.
• Patient must have eligibility confirmed by rapid central imaging review.
• Patients must have =< 4 nodules per lung consistent with or suspicious for
metastases, with at least one of which being >= 3 mm and all of which must be =<
3 cm size.
• Lung nodules must be considered resectable by either open thoracotomy or thoracoscopic
surgery. Determination of resectability is made by the institutional surgeon.
• Patients must have a histological diagnosis of osteosarcoma.
• Patients must have evidence of metastatic lung disease at the time of initial
diagnosis, or at time of 1st recurrence following completion of therapy for initially
localized disease.
• Patients with newly diagnosed disease must have completed successful gross tumor
resection for their primary tumor or surgical local control of primary tumor must be
planned to be performed simultaneously with thoracic surgery.
• Newly diagnosed patients must be receiving systemic therapy considered by the treating
physician as at least equivalent to methotrexate, doxorubicin and cisplatin (MAP) at
the time of enrollment on this study.
• Patients at time of 1st recurrence must have previously completed initial systemic
therapy for their primary tumor, considered by the treating physician as at least
equivalent to MAP.
Exclusion Criteria:
• Patients with unresectable primary tumor.
• Patients with pulmonary metastatic lesions that would require anatomic resection
(lobectomy or pneumonectomy) or lesions that are defined as "central" (i.e., central
lesion involves or is proximal to segmental bronchi and peripheral is lesion distal to
segmental bronchi).
• Patients with pleural or mediastinal based metastatic lesions, or with pleural
effusion.
• Patients with disease progression at either the primary or pulmonary metastatic site
while on initial therapy. Note: Once the patient has been enrolled on the study,
additional computed tomography (CT) scans are not anticipated prior to thoracic
surgery. Note: Some variation in nodule size measurements over the course of
pre-operative therapy is anticipated and does not qualify for exclusion unless deemed
true disease progression by the primary treatment team.
• Patients with evidence of extrapulmonary metastatic disease.
• Patients who received pulmonary surgery for lung metastasis prior to enrollment.
• All patients and/or their parents or legal guardians must sign a written informed
consent.
• All institutional, Food and Drug Administration (FDA), and National Cancer Institute
(NCI) requirements for human studies must be met.
Operative Versus Non-Operative Treatment for Atraumatic Rotator Cuff Tears (ARC)
Rotator cuff tears are one of the most common reasons to seek musculoskeletal care in the
United States and one of the fastest growing ambulatory surgery procedures. However, data on
comparison of operative versus non-operative treatment is lacking and urgently needed.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Nitin Jain
186541
All
50 Years to 84 Years old
N/A
This study is NOT accepting healthy volunteers
NCT03295994
STU 012018-095
Show full eligibility criteria
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Inclusion Criteria:
• Aged =>50 years to <85 years
• Shoulder pain and/or loss of range of active motion, strength or function
• MRI-confirmed partial- or full-thickness supraspinatus and/or infraspinatus tear of
4cm or less in longitudinal dimension
• Medically fit for surgery, defined as Category I-III per American Society of
Anesthesiologists (ASA) Physical Status Classification
• Ability and willingness to provide informed consent
Exclusion Criteria:
• Primary diagnosis is something other than a rotator cuff tear
• History (in last 2 years) of shoulder fracture involving the humeral head on affected
side
• Previous rotator cuff surgery on affected side
• Isolated subscapularis &/or teres minor tear on affected side
• Acute rotator cuff tear caused by a severe trauma
• Shoulder used as a weight-bearing joint
• Contraindication to MRI (claustrophobia, pacemaker, pregnancy, shoulder implant, etc.)
• Glenohumeral osteoarthritis on xrays/MRI
• Grade 4 fatty infiltration of rotator cuff (any tendons)
• Candidate for shoulder arthroplasty at baseline
• Non-English speaking
Early Weight-Bearing After the Lapiplasty Procedure (ALIGN3D)
The objectives of this study are to evaluate the following outcomes of the Lapiplasty®
Procedure for patients in need of hallux valgus surgery:
- The study will determine the radiographic recurrence of hallux valgus and the timing of
failure following hallux valgus correction with the Lapiplasty® Procedure.
- The study will determine whether the Lapiplasty® Procedure effectively corrects
anatomical alignment of the 1st metatarsal and sesamoids in all three planes.
- The study will assess whether early weight-bearing after the Lapiplasty® Procedure
affects the union rates or causes loss of 3-plane correction.
- The study will evaluate the quality of life and pain scores following the Lapiplasty®
Procedure.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Dane Wukich
168693
All
14 Years to 58 Years old
N/A
This study is NOT accepting healthy volunteers
NCT03740282
STU-2018-0389
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Inclusion Criteria:
• Male and females between the ages 14 and 58 years at the time of consent
• Closed physeal plates at the time of consent
• Intermetatarsal angle is between 10.0 ̊ •22.0 ̊
• Hallux valgus angle is between 16.0 ̊ •40.0 ̊
• Willing and able to adhere to early weight-bearing instructions post-operatively
• Capable of completing self •administered questionnaires
• Acceptable surgical candidate, including use of general anesthesia
• Female patients must be of non-child bearing potential or have a negative pregnancy
test within 7 days prior to index procedure
• Willing and able to schedule index procedure within 3 months of consent and able to
return for scheduled follow-up visits
• Willing and able to provide written informed consent
Exclusion Criteria:
• Previous surgery for hallux valgus on operative side
• Previous surgeries on operative foot involving fusion of foot or ankle joints (other
than hammertoe or lesser toes/digits)
• Additional arthrodesis outside the first tarsometatarsal joint (other than:
arthrodesis between the medial cuneiform and intermediate cuneiform and/or base of 2nd
metatarsal; arthrodesis of hammertoe proximal interphalangeal joint or lesser
toes/digits)
• Moderate or Severe osteoarthritis of the MTP joint based on radiographic imaging
(including lack of evident crista) or positive grind test
• Symptomatic flatfoot or asymptomatic flatfoot (defined as calcaneal inclination <5
̊and talonavicular subluxation/uncovering >50%)
• BMI >40 kg/m²
• Current nicotine user, including current use of nicotine patch
• Current clinical diagnosis of diabetes with fasting plasma glucose > 126 mg/dL and/or
HbA 1c ≥7.0
• Current clinical diagnosis of peripheral neuropathy or by assessment on 4 •point
monofilament test
• Current clinical diagnosis of fibromyalgia
• Current clinical diagnosis of Complex Regional Pain Syndrome/Reflex Sympathetic
Dystrophy (CRPS/RSD)
• Current uncontrolled hypothyroidism
• Previously sensitized to titanium
• Currently taking oral steroids or rheumatoid biologics
• Currently taking immunosuppressant drugs
• Insufficient quantity or quality of bone to permit stabilization, conditions that
retard healing (not including pathological fractures) and conditions causing poor
blood supply such as peripheral vascular disease
• Active, suspected or latent infection in the affected area
• Use of synthetic or allogenic bone graft substitutes
• Current diagnosis of metatarsus adductus (defined as MAA ≥ 23 ̊)
• Scheduled to undergo a same •bilateral procedure. Patient agrees to refrain from the
Lapiplasty® Procedure (or other hallux valgus procedures) on contralateral foot for
minimum of 6 months post index procedure
• Patient has previously been enrolled into this study for a contralateral procedure
• Scheduled for any concomitant procedure that would alter patient's ability to early
weight-bear post-procedure
• Patient is actively involved with a workman's compensation case or is currently
involved in litigation
• Patient is currently or has participated in a clinical study in the last 30 days prior
to signing
• Patient has a condition or finding that, in the opinion of the Investigator, may
jeopardize the patient's well-being, the soundness of this clinical study, or could
interfere with provision of informed consent, completion of tests, therapy, or
follow-up
Device: Lapiplasty
Hallux Valgus, Bunion
arthrodesis, bunion, early weight-bearing, lapidus, hallux valgus
POCUS: Hemostatic Potential and Joint Health in Patients With Severe Hemophilia A on Novel Replacement Therapies
This is a prospective, randomized control trial in which each patient will be randomly
assigned to receive either extended half-life factor VIII based replacement therapy or
non-FVIII based replacement therapy, which are both standard of care treatment for persons
with Hemophilia A.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Jessica Garcia
181672
All
up to 17 Years old
Phase 4
This study is NOT accepting healthy volunteers
NCT04690322
STU-2020-1378
Show full eligibility criteria
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Inclusion Criteria:
• Subjects with moderate hemophilia A (baseline factor VIII activity 1-5%) or severe
hemophilia A (baseline factor VIII activity <1%) on prophylactic standard half-life
FVIII infusions OR subjects with moderate or severe hemophilia A who have not started
prophylactic treatment
• Less than 18 years of age
Exclusion Criteria:
• Subjects with documented FVIII inhibitor
• Subjects with a history of ≥ 2 target joints
• Subjects with a history of synovectomy
• Currently using medications known to impact bone and mineral metabolism (e.g.,
bisphosphonates, corticosteroids, estrogen, testosterone, calcitonin, thyroid hormone
therapy);
• Disease states known to affect bone integrity (e.g., primary hyperparathyroidism,
Paget's disease, clinically significant liver disease)
Drug: Eloctate, Drug: Adynovate, Drug: Emicizumab
Hemophilia A, Bones and Joints, Other Hematopoietic, Factor VIII
Elimusertib for the Treatment of Relapsed or Refractory Solid Tumors
This phase I/II trial tests the safety, best dose, and whether elimusertib works in treating
patients with solid tumors that have come back (relapsed) or does not respond to treatment
(refractory). Elimusertib may stop the growth of tumor cells by blocking some of the enzymes
needed for cell growth.
• Part A: Patients between >= 12 months and < 18 years of age
• Part B:
• Patients between >= 12 months and =< 30 years of age for the phase 2 expansion
cohorts for both EWS and PAX3-FOXO1 ARMS.
• Patients between >= 12 months and =< 21 years of age for the phase 2 DDR
expansion cohort
• The Phase 2 cohorts will initially open concurrently with the Phase 1
portion but will only enroll patients at least 18 years of age. Patients <
18 years of age will be included in the Phase 2 cohorts only after the
RP2D/MTD has been estimated in the Phase 1 portion
• All patients for both Parts A and B must have a minimum body surface area (BSA) >=
0.74 m^2
• All patients for both Parts A and B must have the ability to swallow BAY 1895344
(elimusertib) tablets intact
• Patients with recurrent or refractory solid tumors. Patients must have had histologic
verification of malignancy at original diagnosis or relapse
• Part A: Any (non-CNS primary) solid tumor diagnosis including lymphoma which meets one
of the following criteria:
• Any Ewing Sarcoma (histological confirmation alone is adequate) or any EWS-fusion
positive solid tumor (i.e. including related Ewing's family of tumors with EWS
fusions such as EWS-WT1, EWS-ATF1, etc.)
• Alveolar rhabdomyosarcoma (ARMS) with the PAX3-FOXO1 fusion. This does not
include PAX7-FOXO1 or other variant fusion ARMS. Please note that a FISH showing
FOXO1 breakapart is NOT sufficient for eligibility onto this cohort since it
cannot distinguish between FOXO1 partners
• Any (non-CNS primary) solid tumor including lymphoma with inactivating
alterations of any of the DNA Damage Repair (DDR) genes: ATM, ATRX, BRCA1, BRCA2,
CDK12, CHEK1, CHEK2, FANCA, MSH2, MRE11, PALB2, PARP1, POLD1, RAD51, or XRCC2
• Part B: Any (non-CNS primary) solid tumor diagnosis including lymphoma which meets one
of the following criteria:
• B1, EWS Cohort:
• Any Ewing Sarcoma (histological confirmation alone is adequate) or any
EWS-fusion positive solid tumor (i.e. including related Ewing's family of
tumors with EWS fusions such as EWS-WT1, EWS-ATF1, etc.)
• B2, PAX3-FOXO1 ARMS Cohort:
• Alveolar rhabdomyosarcoma (ARMS) with the PAX3-FOXO1 fusion. This does not
include PAX7-FOXO1 or other variant fusion ARMS. Please note that a FISH
showing FOXO1 breakapart is NOT sufficient for eligibility onto this cohort
since it cannot distinguish between FOXO1 partners
• B3, DDR Non-statistical Cohort:
• Any (non-CNS primary) solid tumor including lymphoma with inactivating
alterations of any of the DNA Damage Repair (DDR) genes: ATM, ATRX, BRCA1,
BRCA2, CDK12, CHEK1, CHEK2, FANCA, MSH2, MRE11, PALB2, PARP1, POLD1, RAD51,
or XRCC2
• All the genes on the DDR panel are annotated with OncoKB, a precision oncology
knowledge base which is publicly available here: https://www.oncokb.org/. Alterations
which are categorized either 'Oncogenic' or 'Likely Oncogenic' would be considered
sufficient for eligibility on either the phase 1 or phase 2 portions of this study.
Alterations which are not annotated in OncoKB will need to be reviewed with locally
qualified experts in molecular pathology, such as via an established molecular tumor
board, in order to determine the likely oncogenicity AND will require approval by the
study chair, Dr. Michael Ortiz. If such experts are not available at any institution,
the study chair will review
• In cases where multiple mutations are present or multiple samples are available,
either at different locations or different points in time, the presence of a single
qualifying genomic alteration in any of those samples will is considered sufficient
for eligibility on the phase 2 portions of this study
• Qualifying aberrations must be detected in either DNA or ribonucleic acid (RNA) in any
tumor tissue sample (i.e. detection of a variant on circulating tumor DNA/RNA is not
sufficient to qualify) using a somatic (and/or germline) mutational testing approach
with either a targeted panel or whole exome/genome sequencing in the context of a
Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory setting. Any
CLIA certified laboratory is acceptable to use
• Part A: Patients must have either measurable or evaluable disease
• Part B (1, 2, 3): Patients must have measurable disease
• Patients with a prior history of CNS metastases may enroll on study provided there is
no current evidence of active disease at the time of enrollment
• Patient's current disease state must be one for which there is no known curative
therapy or therapy proven to prolong survival with an acceptable quality of life
• Patients must have a performance status corresponding to Eastern Cooperative Oncology
Group (ECOG) scores of 0, 1 or 2. Use Karnofsky >= 50% for patients > 16 years of age
and Lansky >= 50% for patients =< 16 years of age. Note that neurologic deficits in
patients with tumors previously metastatic to the CNS (or other non-oncologic reasons)
must have been stable for at least 7 days prior to study enrollment. Patients who are
unable to walk because of paralysis, but who are up in a wheelchair, will be
considered ambulatory for the purpose of assessing the performance score
• Patients must have fully recovered from the acute toxic effects of all prior
anti-cancer therapy and must meet the following minimum duration from prior
anti-cancer directed therapy prior to enrollment. If after the required timeframe, the
numerical eligibility criteria are met, e.g., blood count criteria, the patient is
considered to have recovered adequately
• Cytotoxic chemotherapy or other anti-cancer agents known to be myelosuppressive:
>= 21 days after the last dose of myelosuppressive chemotherapy (42 days if prior
nitrosourea)
• Anti-cancer agents not known to be myelosuppressive (e.g., not associated with
reduced platelet or absolute neutrophil count [ANC] counts): >= 7 days after the
last dose of agent
• Antibodies: >= 21 days must have elapsed from infusion of last dose of antibody,
and toxicity related to prior antibody therapy must be recovered to grade =< 1
• Corticosteroids: If used to modify immune adverse events related to prior
therapy, >= 14 days must have elapsed since last dose of corticosteroid
• Hematopoietic growth factors: >= 14 days after the last dose of a long-acting
growth factor (e.g., pegfilgrastim) or 7 days for short acting growth factor. For
agents that have known adverse events occurring beyond 7 days after
administration, this period must be extended beyond the time during which adverse
events are known to occur
• Interleukins, interferons and cytokines (other than hematopoietic growth
factors): >= 21 days after the completion of interleukins, interferon or
cytokines (other than hematopoietic growth factors)
• Stem cell Infusions (with or without total-body irradiation [TBI]):
• Allogeneic (non-autologous) bone marrow or stem cell transplant, or any stem
cell infusion including donor lymphocyte infusions (DLI) or boost infusion:
>= 84 days after infusion and no evidence of graft versus host disease
(GVHD)
• Autologous stem cell infusion including boost infusion: >= 30 days
• Cellular therapy: >= 42 days after the completion of any type of cellular therapy
(e.g., modified T cells, natural killer [NK] cells, dendritic cells, etc.)
• Radiation therapy (XRT)/external beam irradiation including protons: >= 14 days
after local XRT; >= 150 days after TBI, craniospinal XRT or if radiation to >=
50% of the pelvis; >= 42 days if other substantial bone marrow (BM) radiation
• Radiopharmaceutical therapy (e.g., radiolabeled antibody, 131I MIBG): >= 42 days
after systemically administered radiopharmaceutical therapy
• Study specific prior therapy: Patients must not have received prior exposure to
BAY 1895344 (elimusertib) or any other specific ATR inhibitors including
berzosertib (M6620, VX-970), ceralasertib (AZD6738), M4344 (VX-803), M1774, and
RP-3500. Treatment with other DNA damage repair inhibitors which do not
specifically inhibit ATR (e.g. PARP inhibitors, WEE1 inhibitors, CHEK1
inhibitors, etc.) does not exclude them from eligibility on this study
• For patients with solid tumors without known bone marrow involvement
• Peripheral absolute neutrophil count (ANC) >= 1000/uL
• For patients with solid tumors without known bone marrow involvement
• Platelet count >= 100,000/uL (transfusion independent, defined as not receiving
platelet transfusions for at least 7 days prior to enrollment)
• For patients with solid tumors without known bone marrow involvement
• Hemoglobin >= 8.0 g/dL at baseline (may receive red blood cell [RBC]
transfusions)
• Patients with known or possible bone marrow metastatic disease will be eligible for
study provided they meet the blood counts in above inclusion criteria (may receive
transfusions provided they are not known to be refractory to red cell or platelet
transfusions). These patients will not be evaluable for hematologic toxicity. At least
5 of every cohort of 6 patients must be evaluable for hematologic toxicity for the
dose-escalation part of the study. If dose-limiting hematologic toxicity is observed,
all subsequent patients enrolled must be evaluable for hematologic toxicity
• Serum creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70
mL/min/1.73 m^2 or a creatinine based on age/gender as follows:
• Age: 1 to < 2 years; Maximum serum creatinine (mg/dL): 0.6 (male); 0.6 (female)
• Age: 2 to < 6 years; Maximum serum creatinine (mg/dL): 0.8 (male); 0.8 (female)
• Age: 6 to < 10 years; Maximum serum creatinine (mg/dL): 1 (male); 1 (female)
• Age: 10 to < 13 years; Maximum serum creatinine (mg/dL): 1.2 (male); 1.2 (female)
• Age: 13 to < 16 years; Maximum serum creatinine (mg/dL): 1.5 (male); 1.4 (female)
• Age: >= 16 years; Maximum serum creatinine (mg/dL): 1.7 (male); 1.4 (female)
• Bilirubin (sum of conjugated + unconjugated or total) =< 1.5 x upper limit of normal
(ULN) for age
• Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 135
U/L. For the purpose of this study, the ULN for SGPT is 45 U/L
• Patients with seizure disorder may be enrolled if on anticonvulsants and well
controlled as evidenced by no increase in seizure frequency in the prior 7 days. For
patients a history of seizure but not on anticonvulsants, no seizure in the past 3
months
• Nervous system disorders (Common Terminology Criteria for Adverse Events [CTCAE]
version [v]5) resulting from prior therapy must be =< grade 2, with the exception of
decreased tendon reflex (DTR). Any grade of DTR is eligible
Exclusion Criteria:
• Pregnant or breast-feeding women will not be entered on this study due to risks of
fetal and teratogenic adverse events as seen in animal/human studies, OR because there
is yet no available information regarding human fetal or teratogenic toxicities.
Pregnancy tests must be obtained in girls who are post-menarchal. Males or females of
reproductive potential may not participate unless they have agreed to use two
effective methods of birth control, including a medically accepted barrier or
contraceptive method (e.g., male or female condom) for the duration of the study and
for 3 months + 2 days for males and 6 months + 2 days for females after receiving the
last dose of BAY 1895344 (elimusertib) on the study. Abstinence is an acceptable
method of birth control. Female patients must not breastfeed during treatment and
until 4 months after last study drug administration
• Corticosteroids: Patients receiving corticosteroids who have not been on a stable or
decreasing dose of corticosteroid for at least 7 days prior to enrollment are not
eligible. If used to modify immune adverse events related to prior therapy, >= 14 days
must have elapsed since last dose of corticosteroid
• Patients who are currently receiving another investigational drug are not eligible
• Patients who are currently receiving other anti-cancer agents are not eligible
• Patients who are receiving cyclosporine, tacrolimus or other agents to prevent
graft-versus-host disease post bone marrow transplant are not eligible for this trial
• Patients who are currently receiving drugs that are strong inducers or inhibitors of
CYP3A4 are not eligible. Strong inducers or inhibitors of CYP3A4 should be avoided
from 14 days prior to enrollment to the end of the study. Drugs that are considered
sensitive or narrow therapeutic range CYP3A4 substrates should be avoided for the
duration of protocol therapy
• Dedicated CNS imaging is not required but patients with current active CNS metastasis
whether symptomatic or discovered incidentally without clinical symptoms, will be
excluded from study participation
• Patients who have an uncontrolled infection are not eligible
• Patients who have received a prior solid organ transplantation are not eligible
• Patients who in the opinion of the investigator may not be able to comply with the
safety monitoring requirements of the study are not eligible
Study of TT-00420 Tablet as Monotherapy and Combination Therapy in Patients With Advanced Solid Tumors
This is a Phase Ib/II, multicenter, open-label study to evaluate the safety and preliminary
efficacy of TT-00420 tablet, as monotherapy or in combination regimens, in patients with
advanced solid tumors.
1. ≥ 18 years of age
2. Histopathological or cytologically documented locally advanced or metastatic solid
tumors who have no available standard therapeutic treatment options
3. At least one measurable lesion as defined by RECIST V1.1 criteria for solid tumors
4. Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1
5. Adequate organ function confirmed at screening and within 10 days of initiating
treatment, as evidenced by:
• Absolute neutrophil count (ANC) ≥ 1.5 x 10^9/L
• Hemoglobin (Hgb) ≥ 8 g/dl
• Platelets (plt) ≥ 75 x 10^9/L
• AST/SGOT and ALT/SGPT ≤ 2.5 x Upper Limit of Normal (ULN) or ≤ 5.0 x ULN if liver
metastases are present
• Total bilirubin ≤ 1.5 x ULN
• Calculated creatinine clearance ≥ 50 mL/min (Cockcroft Gault formula)
6. Negative pregnancy test within 72 hours before starting study treatment in all
premenopausal women and women < 12 months after the onset of menopause
7. Must agree to take sufficient contraceptive methods to avoid pregnancy during the
study and until at least 6 months after ceasing study treatment
8. Able to sign informed consent and comply with the protocol
Exclusion Criteria:
1. Women who are pregnant or lactating
2. Women of child-bearing potential (WOCBP) who do not use adequate birth control
3. Patients with any hematologic malignancy, including leukemia (any form), lymphoma, and
multiple myeloma
4. Patients with a history of primary central nervous system tumors or carcinomatous
meningitis.
5. Patients with the following mood disorders as judged by the Investigator or a
psychiatrist:
• Medically documented history of or active major depressive episode, bipolar
disorder (I or II), obsessive-compulsive disorder, schizophrenia; a history of
suicidal attempt or ideation, or homicidal ideation (immediate risk of doing harm
to others)
• ≥ CTCAE grade 3 anxiety
6. Impaired cardiac function or significant diseases, including but not limited to any of
the following:
• left ventricular ejection fraction (LVEF) < 45% as determined by multigated
acquisition (MUGA) scan or echocardiogram (ECHO)
• Congenital long QT syndrome
• QTcF ≥ 480 msec on screening ECG
• Unstable angina pectoris ≤ 3 months prior to starting study drug
• Acute myocardial infarction ≤ 3 months prior to starting study drug
7. Patients with:
• unresolved diarrhea ≥ CTCAE grade 2, or
• impairment of gastrointestinal (GI) function, or
• GI disease that may significantly alter the absorption of TT-00420
8. Other concurrent severe and/or uncontrolled concomitant medical conditions (e.g.,
uncontrolled hypertension, uncontrolled hypertriglyceridemia, or active or
uncontrolled infection) that could cause unacceptable safety risks or compromise
compliance with the protocol
9. Patients who have received chemotherapy, targeted therapy, or immunotherapy ≤ 4 weeks
(6 weeks for nitrosourea or mitomycin-C) prior to starting study drug or who have not
recovered from side effects of such therapy
10. Patients who have received wide field radiotherapy ≤ 4 weeks or limited field
radiation for palliation ≤ 2 weeks prior to starting study drug or who have not
recovered from side effects of such therapy
11. Patients who have undergone major surgery ≤ 4 weeks prior to starting study drug or
who have not recovered from side effects of such therapy
12. Patients who have been treated with any hematopoietic colony-stimulating growth
factors (e.g., G-CSF, GM-CSF) ≤ 4 weeks prior to starting study drug.
13. Patients who are currently receiving treatment with therapeutic doses of warfarin
sodium or any other coumarin-derivative anticoagulants
14. Patients who have received systemic corticosteroids ≤ 2 weeks prior to starting study
drug or who have not recovered from the side effects of such treatment.
15. Patients who are currently receiving treatment with strong CYP3A inhibitors or
inducers ≤ 2 weeks prior to starting study drug.
16. Known diagnosis of human immunodeficiency virus (HIV) infection (HIV testing is not
mandatory; patients with well controlled HIV might be enrolled)
17. Known history of active infection with Hepatitis B or Hepatitis C
18. Has received a live-virus vaccination within 30 days of planned first dose
19. Inability to swallow or tolerate oral medication
20. Has a history or current evidence of any condition, therapy, or laboratory abnormality
that, in the opinion of the investigator, might confound the results of the trial,
interfere with the patient's safe participation and compliance in the trial.
Prostate Cancer, Sarcoma, Gastric Cancer, Bladder Cancer, Cholangiocarcinoma, Gallbladder Cancer, Thyroid Cancer, Triple Negative Breast Cancer, HER2-negative Breast Cancer, Advanced Solid Tumor, Breast - Female, Breast - Male, Lung/Thoracic, Other Digestive Organ, Other Skin, Other Urinary, Prostate, Stomach, Thyroid, Small Intestine, Small-cell Lung Cancer
Stereotactic Radiosurgery (SRS) for Brain Metastasis (SRS)
SRS dose escalation for brain metastases in radiation-naïve patients will establish true
tolerable doses, which may exceed the current standard doses. This may lead to an improvement
in local control, patient survival, and/or quality-of life.
Inclusion Criteria
1. Biopsy-proven non-hematopoietic malignancy, except for small cell lung cancer, germ
cell cancer, or unknown primary tumor.
2. Radiographic evidence by MRI (or by CT scan with CT contrast if ineligible or
intolerant of MRI) of brain metastasis. (If patient is unable to tolerate MRI
contrast, an MRI without contrast is acceptable if lesions are visible)
3. All brain metastases must be outside the brain stem (midbrain, pons and medulla).
4. Patient must have 10 or less brain metastases.
5. The maximum diameter of any lesion must be less than or equal to 3.0 cm.
6. Previous treatment with surgery, radiation, chemotherapy, immunotherapy or any
targeted agents are allowed provided that:
• Radiation was not to the brain.
• Surgery to the brain was > 7 days prior to SRS and there remains at least one
additional brain metastasis that can be targeted with SRS
7. Age ≥ 18 years.
8. ECOG Performance Score of 2 or better/Karnofsky Performance Status score of 50-60 or
better.
9. All men, as well as women of child-bearing potential must agree to use adequate
contraception (hormonal or barrier method of birth control; abstinence) prior to study
entry and for the duration of study participation. Should a woman become pregnant or
suspect she is pregnant while participating in this study, she should inform her
treating physician immediately.
A female of child-bearing potential is any woman (regardless of sexual orientation,
marital status, having undergone a tubal ligation, or remaining celibate by choice)
who meets the following criteria:
• Has not undergone a hysterectomy or bilateral oophorectomy; or
• Has not been naturally postmenopausal for at least 12 consecutive months (i.e.,
has had menses at any time in the preceding 12 consecutive months).
Medically acceptable birth control (contraceptives) includes:
• Approved hormonal contraceptives (such as birth control pills, patch, or ring:
Depo-Provera, Implanon), or
• Barrier methods (such as a condom or diaphragm) used with a spermicide (a
substance that kills sperm)
10. Ability to understand and the willingness to sign a written informed consent.
Exclusion Criteria
1. Patients had craniotomy and surgery to the brain within 7 days from the date of SRS.
2. Patients with leptomeningeal metastasis.
NOTE: For the purposes of exclusion, LMD is a clinical diagnosis, defined as positive
CSF cytology and/or equivocal radiologic or clinical evidence of leptomeningeal
involvement. Patients with leptomeningeal symptoms in the setting of leptomeningeal
enhancement by imaging (MRI) would be considered to have LMD even in the absence of
positive CSF cytology, unless a parenchymal lesion can adequately explain the
neurologic symptoms and/or signs. In contrast, an asymptomatic or minimally
symptomatic patient with mild or nonspecific leptomeningeal enhancement (MRI) would
not be considered to have LMD. In that patient, CSF sampling is not required to
formally exclude LMD, but can be performed at the investigator's discretion based on
level of clinical suspicion.
3. Patients with a contraindication to both MRI (with or without contrast) and CT scan
(with contrast)
4. Patients with life expectancy < 3 months.
5. Uncontrolled intercurrent illness including, but not limited to, ongoing or active
infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac
arrhythmia, or psychiatric illness/social situations that, in the opinion of the
investigator, would limit compliance with study requirements.
6. Subjects must not be pregnant or nursing at the time of SRS treatment due to the
potential for congenital abnormalities and the potential of this regimen to harm
nursing infants.
Radiation: Stereotactic Radiosurgery
Brain Neoplasms, Adult, Malignant, Lymphoma, Sarcoma, Multiple Myeloma, Brain and Nervous System, Other, Eye and Orbit, Anklylosing Spondylitis, Anus, Bones and Joints, Breast - Female, Breast - Male, Cardiovascular, Cervix, Colon, Corpus Uteri, Ear, Esophagus, Gall Bladder, Head and Neck, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung/Thoracic, Melanoma, skin, Nose, Other Digestive Organ, Other Endocrine System, Other Female Genital, Other Male Genital, Other Respiratory and Intrathoracic Organs, Other Skin, Other Urinary, Ovary, Pancreas, Prostate, Rectum, Stomach, Throat, Thyroid, Urinary Bladder, Uterine (Endometrial), Vulva, Hodgkins Lymphoma, Lymphoid Leukemia, Small Intestine, Soft Tissue
UT Southwestern; Parkland Health & Hospital System
Study Of Palbociclib Combined With Chemotherapy In Pediatric Patients With Recurrent/Refractory Solid Tumors
This study will evaluate palbociclib in combination with chemotherapy (temozolomide with
irinotecan and/or topotecan with cyclophosphamide) in children, adolescents and young adults
with recurrent or refractory solid tumors. The main purpose of phase 1 portion of this study
is to evaluate the safety of palbociclib in combination with chemotherapy in order to
estimate the maximum tolerated dose. The main purpose of phase 2 portion is to compare the
efficacy of palbociclib in combination with irinotecan and temozolomide vs irinotecan and
temozolomide alone in the treatment of children, adolescents, and young adults with recurrent
or refractory Ewing sarcoma (EWS). Pharmacokinetics and efficacy of palbociclib in
combination with chemotherapy will be evaluated.
Inclusion:
1. Histologically confirmed relapsed or refractory solid tumor as follows:
• For dose escalation and dose determination parts: Histologically confirmed
relapsed or refractory solid tumor (including CNS tumors but not lymphomas).
Patients with Diffuse Intrinsic Pontine Glioma do not require histological only
radiographic confirmed relapse to enroll.
• For dose expansion and tumor specific cohorts: Histologically confirmed relapsed
or refractory solid tumor including but not limited to EWS, rhabdoid tumor,
rhabdomyosarcoma, neuroblastoma, and medulloblastoma. Patients with Diffuse
Intrinsic Pontine Glioma do not require histological only radiographic confirmed
relapse to enroll. EWS is not eligible for TOPO and CTX tumor-specific cohorts.
• For randomized Phase 2 part: Histologically confirmed Ewing sarcoma.
Histopathology confirmation of EWSR1-ETS or FUS-ETS rearrangement is required or
availability of formalin fixed paraffin embedded (FFPE) tumor tissue sample for
central testing. Patient must have relapsed or refractory disease with no known
bone marrow metastases and at least evaluable disease.
2. Age ≥2 and <21 years at the time of study entry.
3. Lansky performance status ≥50% for patients ≤16 years of age, or Eastern Cooperative
Oncology Group (ECOG) 0, 1 or 2 for patients >16 years of age.
4. Adequate bone marrow function.
• Absolute neutrophil count ≥1000/mm3;
• Platelet count ≥100,000/mm3 (transfusion independent, no platelet transfusion in
past 7 days prior study entry);
• Hemoglobin ≥8.5 g/dL (transfusion allowed).
5. Adequate renal function: Serum creatinine level based on age/gender must within
protocol specified limits.
6. Adequate liver function, including:
• Aspartate aminotransferase (AST) and alanine aminotransferase (ALT)
≤2.5 × upper limit of normal (ULN) or ≤5 × ULN for age, if attributable to
disease involvement of the liver;
• Total bilirubin ≤1.5 × ULN for age, unless the patient has documented Gilbert's
syndrome.
7. Patients enrolled to Phase 1 portion of the study and tumor specific cohorts must have
measurable disease as defined by RECIST version 1.1 or modified RANO criteria for CNS
disease or INRC for neuroblastoma. Patients with EWS enrolled to Phase 2 portion of
the study are eligible with evaluable disease (eg, bone only disease with no soft
tissue component).
8. Recovered to CTCAE Grade ≤1, or to baseline, from any non-hematological acute
toxicities of prior surgery, chemotherapy, immunotherapy, radiotherapy,
differentiation therapy or biologic therapy, with the exception of alopecia.
9. Serum/urine pregnancy test (for all girls ≥8 years of age) negative at screening and
at the baseline visit.
10. Evidence of a personally signed and dated informed consent document indicating that
the patient or a legally acceptable representative/parent(s)/legal guardian of minors,
has been informed of all pertinent aspects of the study. Minor study patients also
must provide age appropriate assent according to the local guidelines, where
applicable.
11. Willing and able to comply with scheduled visits, treatment plan, laboratory tests,
and other procedures.
Exclusion:
1. Phase 1 and tumor specific cohorts: For palbociclib with IRN and TMZ combination,
prior treatment with a CDK4/6 inhibitor or progression while on treatment with an
IRN-containing regimen that includes TMZ. Patients who have received the combination
of IRN and TMZ and did not progress while on these medications are eligible. For
patients enrolling in the palbociclib with TOPO and CTX combination, prior treatment
with a CDK4/6 inhibitor or progression while on treatment with a TOPO-containing
regimen that includes CTX. Patients who have received the combination of TOPO and CTX
and did not progress while on these medications are eligible. Phase 2 :prior treatment
with a CDK4/6 inhibitor or prior treatment with an IRN and/or TMZ-containing regimen.
2. Prior intolerability to IRN and/or TMZ plus/minus palbociclib with IRN and TMZ
combination and prior intolerability to TOPO and/or CTX for TOPO and CTX combination.
3. Use of strong cytochrome P450 (CYP) 3A inhibitors or inducers. Patients who are
receiving strong uridine diphosphate-glucuronosyl transferase 1A1 (UGT1A1) inhibitors
within 12 days of Cycle 1 Day 1 (C1D1) are not eligible for the palbociclib with IRN
and TMZ combination. Patients who are receiving strong UGT1A1 inhibitors within 12
days of C1D1 are eligible for the palbociclib with TOPO and CTX combination (See
Section 5.7.1 for list of products.)
4. Prior growth factors (including filgrastim) within 7 days before study entry or
PEG-filgrastim within 14 days before study entry.
5. Radiation therapy within 14 days before study entry.
6. Systemic anti cancer therapy within 2 weeks prior to study entry and 6 weeks for
nitrosoureas.
7. Previous high dose chemotherapy requiring stem cell rescue within 90 days or
persistent AE >Grade 1.
8. Prior irradiation to >50% of the bone marrow (see Appendix 9).
9. Participation in other studies involving investigational drug(s) within 2 weeks or 5
half lives, whichever is longer, prior to study entry.
10. Major surgery within 4 weeks prior to study entry. Surgical biopsies or central line
placement are not considered major surgeries.
11. For IRN and TMZ with/without palbociclib combinations: known or suspected
hypersensitivity to palbociclib, IRN and/or TMZ. For combination of palbociclib with
TOPO and CTX: known or suspected hypersensitivity to palbociclib, TOPO and/or CTX.
12. Patients with known symptomatic brain tumors or brain metastases and require steroids,
unless they have been on a stable or on a decreasing steroid dose for >14 days.
13. Patients with previously diagnosed brain metastases are eligible if they have
completed their prior treatment and have recovered from the acute effects of radiation
therapy or surgery prior to study entry for these metastases for at least 14 days post
radiation and 4 weeks post-surgery and are neurologically stable.
14. Hereditary bone marrow failure disorder.Phase 2 portion patients with bone marrow
involvement are excluded.
15. QTc >470 msec.
16. History of clinically significant or uncontrolled cardiac disease, including:
• History of or active congestive heart failure; if patient had congestive heart
failure resolve and >1 year from resolution, patient will be considered eligible;
• Clinically significant ventricular arrhythmia (such as ventricular tachycardia,
ventricular fibrillation or Torsades de Pointes);
• Diagnosed or suspected congenital or acquired prolonged QT syndrome;
• Need for medications known to prolong the QT interval;
• Uncorrected hypomagnesemia or hypokalemia because of potential effects on the QT
interval;
• Left ventricular ejection fraction <50% or shortening fraction <28%.
17. Recent or ongoing clinically significant gastrointestinal disorder that may interfere
with absorption of orally administered drugs (eg, gastrectomy).
18. Evidence of serious active or uncontrolled bacterial, fungal or viral infection or
known history of hepatitis B virus, hepatitis C virus, or human immunodeficiency virus
infection or acquired immunodeficiency syndrome-related illness.
19. Other severe acute or chronic medical or laboratory test abnormality that may increase
the risk associated with study participation or investigational product administration
or may interfere with the interpretation of study results, and in the judgment of the
Investigator, would make the patient inappropriate for entry into this study.
20. Investigator site staff members directly involved in the conduct of the study and
their family members, site staff members otherwise supervised by the investigator, or
patients who are Pfizer employees, including their family members, directly involved
in the conduct of the study.
21. Fertile male patients and female patients of childbearing potential who are unwilling
or unable to use a highly effective method of contraception as outlined in this
protocol for the duration of the study and for at least 90 after the last dose of
investigational product.
Neuroblastoma, Sarcoma, Solid Tumors, Ewing Sarcoma, Medulloblastoma, Diffuse Intrinsic Pontine Glioma, Rhabdomyosarcoma, Rhabdoid Tumor, Brain and Nervous System, Eye and Orbit, Anus, Bones and Joints, Breast - Female, Breast - Male, Cervix, Colon, Corpus Uteri, Ear, Esophagus, Gall Bladder, Head and Neck, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung/Thoracic, Nose, Other Digestive Organ, Other Endocrine System, Other Female Genital, Other Male Genital, Other Respiratory and Intrathoracic Organs, Other Skin, Other Urinary, Ovary, Pancreas, Prostate, Rectum, Stomach, Throat, Thyroid, Urinary Bladder, Uterine (Endometrial), Vulva, Kaposis sarcoma, Small Intestine, Soft Tissue, Ill - Defined Sites
An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy (TOPAZ)
The TOPAZ study will assess the safety and efficacy of SRK-015 in later-onset Spinal Muscular
Atrophy (SMA Type 2 and Type 3) in pediatric and adult patients.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Kaitlin Batley
162753
All
2 Years to 21 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT03921528
STU-2019-0631
Show full eligibility criteria
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Inclusion Criteria:
• Age 5 through 21 years old at the time of screening for Cohorts 1 and 2; Age ≥2 years
old at the time of screening for Cohort 3.
• Documented diagnosis of 5q SMA.
• Diagnosed as later-onset (e.g., Type 2 or Type 3) SMA prior to receiving any treatment
with therapy approved for SMA.
• Non-ambulatory patients must be able to sit independently (sits up straight with head
erect for at least 10 seconds; does not use arms or hands to balance body or support
position) per World Health Organization (WHO) motor milestones definition at
screening.
• Ambulatory patients must have the ability to independently ambulate without aids or
orthotics over 10 meters in 30 seconds or less at screening.
• Receiving the same background SMA therapy (e.g., on an approved survival motor neuron
(SMN) upregulator therapy such as nusinersen, or not on any SMA therapy) for at least
6 months prior to screening and anticipated to remain on that therapy throughout the
duration of the study.
• If receiving the SMN upregulator therapy nusinersen, must have completed the
loading regimen and initiated maintenance dosing (i.e., completed at least one
maintenance dose) with at least 4 weeks after the first maintenance dose having
elapsed prior to screening.
• Nutritional status stable over the past 6 months and anticipated to be stable
throughout the duration of the study.
• Have no physical limitations that would prevent the patient from undergoing motor
function outcome measures throughout the duration of the study.
• Able to receive study drug infusions and provide blood samples through the use of a
peripheral intravenous (IV) or a long-term IV access device that the patient has
placed for reasons independent from the study throughout the duration of the study.
• Able to adhere to the requirements of the protocol, including travel to the study
center and completing all study procedures and study visits.
• For patients who are expected to have reached reproductive maturity by the end of the
study, adhere to study specific contraception requirements.
Exclusion Criteria:
• Use of tracheostomy with positive pressure.
• Use of chronic daytime non-invasive ventilatory support for >16 hours daily in the 2
weeks prior to dosing, or anticipated to regularly receive such daytime ventilator
support chronically over the duration of the study.
• Any acute or co-morbid condition interfering with the well-being of the patient within
14 days of screening, including active systemic infection, the need for acute
treatment or inpatient observation due to any reason.
• Severe scoliosis and/or contractures at screening. Based on clinical judgement, any
scoliosis or contractures present must be stable over the past 6 months, anticipated
to be stable for the duration of the study and not prevent the patient from being
evaluated on any functional outcome measures throughout the duration of the study.
• Pregnant or breastfeeding.
• Major orthopedic or other interventional procedure, including spine or hip surgery,
considered to have the potential to substantially limit the ability of the patient to
be evaluated on any functional outcome measures, within 6 months prior to screening,
or anticipated for the duration of the study.
• Prior history of a hypersensitivity reaction to a monoclonal antibody (mAb) or
recombinant protein bearing an Fc domain (such as a soluble receptor- Fc fusion
protein).
• Use of systemic corticosteroids within 60 days prior to screening. Inhaled or topical
steroids are allowed.
• Treatment with investigational drugs within 3 months prior to screening.
• Use of therapies with potentially significant muscle effects (such as androgens,
insulin-like growth factor, growth hormone, systemic betaagonist, botulinum toxin, or
muscle relaxants) or muscle-enhancing supplements within 60 days prior to screening.
• Patient has any other condition, which in the opinion of the Investigator may
compromise safety or compliance, would preclude the patient from successful completion
of the study, or interfere with the interpretation of the results.
Interscalene Bupivacaine With Either Liposomal Bupivacaine or Continuous Peripheral Nerve Block for Shoulder Arthroscopy
This investigation will be a prospective, randomized trial. The study population will consist
of adult patients scheduled to undergo major shoulder arthroscopy procedures with
anesthesiology at the Ambulatory Services Center (Outpatient Surgery Center). Forty-six
subjects will be enrolled in this study and will be randomized into one of two arms: 1)
Single-injection bupivacaine HCl plus a subsequent bupivacaine CISB, 2) Single-injection
bupivacaine HCl plus liposomal bupivacaine included in the same injection. These procedures
will take place, using an ultrasound-guided method, approximately one hour prior to surgical
procedure. Subjects will be followed for seven days to assess pain control by the Modified
Brief Pain Inventory (MBPI) survey and outcome factors related to study and procedure using
the American Shoulder and Elbow Surgeon Shoulder Score (ASES Shoulder Score).
Call 214-648-5005 studyfinder@utsouthwestern.edu
Anthony Machi
159003
All
18 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT03816982
STU 082018-017
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:
• Adult patients ≥ 18 years
• Individuals presenting for major shoulder arthroscopy procedures at Ambulatory
Services Center (Outpatient Surgery Center) -that include rotator cuff repair and
biceps tenodesis
• Patient is able to provide informed consent to participate in the study.
Exclusion Criteria:
• Allergy to amide local anesthetic, liposomal bupivacaine or other medication involving
liposomal formulation
• Preexisting neurological deficits involving or potentially involving the ipsilateral
brachial plexus
• Preexisting contralateral vocal fold paralysis or recurrent laryngeal paralysis
• Psychiatric or cognitive disorders that could interfere with perioperative evaluation
including drug or alcohol abuse
• Chronic pain conditions
• Preoperative opioid consumption greater than 20 mg oral morphine equivalent.
• Any contraindication to interscalene nerve block including any local disorder of the
skin where blockade is to be performed which would prevent safe performance of the
block
• Any coagulation abnormality which would be a contraindication for block placement
• Preoperative chronic renal dysfunction requiring renal replacement therapy or a serum
creatinine greater than 1.4 mg/dL
• Body mass index >50
• Pregnancy
• Incarceration
• ASA classification greater than 3
• Inability to provide informed consent
Procedure: Single-injection bupivacaine HCl plus liposomal bupivacaine, Procedure: Single-injection bupivacaine HCl plus a subsequent bupivacaine CISB
Nivolumab or Nivolumab and Azacitidine in Patients With Recurrent, Resectable Osteosarcoma
The purpose of this study is to evaluate the safety and efficacy of nivolumab, or nivolumab
in combination with azacitidine in participants with recurrent, resectable osteosarcoma
• Participants must have had a histologic diagnosis of osteosarcoma at original
diagnosis
• Disease Status: Patients with an isolated pulmonary recurrence of osteosarcoma can be
enrolled on this study.
• Any history of metastatic disease at a site other than lung would make the
patient ineligible for this study.
• The patient's treating team must consider the patient's disease to be resectable
and the patient must be willing to undergo resection of all disease, including
any lung lesion meeting criteria for likely metastatic disease, defined as: 3 or
more lesions ≥ 3 mm in diameter OR a single lesion ≥ 5 mm.
• Patients with bilateral disease are eligible provided their disease is considered
resectable. Resectable pulmonary nodules are defined as nodules that can be
removed without performing a pneumonectomy (e.g., nodules immediately adjacent to
the main stem bronchus or main pulmonary vessels).
• Must have a performance status corresponding to Eastern Cooperative Oncology Group
(ECOG) scores of 0, 1 or 2, using the Karnofsky scale for patients > 16 years of age
and the Lansky scale for patients ≤ 16 years of age
• Prior Therapy: Participants must have fully recovered from the acute toxic effects of
all prior chemotherapy, immunotherapy, or radiotherapy prior to the start of protocol
therapy.
• Participants must have normal organ and marrow function within 7 days of starting
protocol therapy
• All participants and/or their parents or legal guardians must have the ability to
understand and the willingness to sign a written informed consent/assent document
• Additional criteria may apply
Exclusion Criteria:
• Pregnancy or Breast Feeding
• Males and females of reproductive potential may not participate unless they have
agreed to the use of, at minimum, two methods of contraception, with one method being
highly effective and the other method being either highly effective or less effective
as outlined in study protocol documentation
• Concomitant Medications: Patients receiving the following are not eligible:
• Corticosteroids or other immunosuppressive medications
• Patients who are currently receiving other investigational agents or other anti-cancer
therapy
• Intercurrent Illnesses: Patients with uncontrolled intercurrent illness including, but
not limited to:
• Ongoing or active infection
• Symptomatic congestive heart failure
• Unstable angina pectoris
• Cardiac arrhythmia
• Psychiatric illness/social situations that would limit compliance with study
requirements
• Autoimmune disorders: Patients with a history of any Grade autoimmune disorder are not
eligible.
• Asymptomatic laboratory abnormalities (e.g., ANA, rheumatoid factor, altered thyroid
function studies) will not render a patient ineligible in the absence of a diagnosis
of an autoimmune disorder.
• Patients with ≥ Grade 2 hypothyroidism due to history of autoimmunity are not
eligible. Note: Hypothyroidism due to previous irradiation or thyroidectomy will not
impact eligibility
• Allergies: Patients with a history of allergic reactions attributed to compounds of
similar chemical or biologic composition to Nivolumab (e.g., another humanized
antibody) or Azacitidine are not eligible
• Safety and Monitoring: Patients who are considered unable to comply with the safety
monitoring requirements of the study are not eligible
• Patients with known HIV or hepatitis B or C are excluded
• Patients who have received prior solid organ transplantation are not eligible
• Patients who have received prior anti-PD-1 directed therapy (mAb or small molecule)
are not eligible
Drug: Nivolumab, Drug: Azacitidine, Procedure: Post Treatment Surgery
Sarcoma, Osteosarcoma, Soft Tissue, Osteosarcoma in Children, Osteosarcoma Recurrent
Nivolumab in Treating Patients With Autoimmune Disorders and Advanced, Metastatic, or Unresectable Cancer
This phase Ib trial studies the side effects of nivolumab and to see how well it works in
treating patients with autoimmune disorders and cancer that has spread to other places in the
body or cannot removed by surgery. Immunotherapy with monoclonal antibodies, such as
nivolumab, may help the body's immune system attack the cancer, and may interfere with the
ability of tumor cells to grow and spread.
• Patients must have histologically confirmed malignancy that is radiologically
evaluable and metastatic or unresectable. Eligible tumor types include solid tumors
and malignancies in which there is known evidence of clinical activity for single
agent PD-1 or PD-L1 antibodies. Nivolumab is Food and Drug Administration
(FDA)-approved for the treatment of melanoma, non-small cell lung cancer (NSCLC),
Merkel cell cancer, bladder cancer, renal cell carcinoma (RCC), gastric cancer,
hepatocellular carcinoma (HCC), cervical cancer, head and neck cancer, Hodgkin
lymphoma (HL), metastatic small cell lung cancer (SCLC), and any solid tumor with
microsatellite instability (MSI)-high status confirmed. Patients with HL are eligible
but must follow standard response criteria. Additional tumor types may be eligible on
a case by case basis upon discussion with principal investigator (PI)
• Patients who have previously received other forms of immunotherapy (high-dose [HD]
IL-2, IFN, CTLA-4) are allowed. Patients must not have received cytokine immunotherapy
for at least 4 weeks before nivolumab administration. Patients who have received prior
anti-CTLA4 will be allowed and the washout period is 6 weeks
• Age >= 18 years; children are excluded from this study but may be eligible for future
pediatric phase 1 combination trials
• Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2 (Karnofsky >=
60)
• Life expectancy of greater than 12 weeks
• Leukocytes >= 2,000/mcL
• Absolute neutrophil count >= 1,500/mcL
• Platelets >= 100,000/mcL
• Total bilirubin =< 1.5 x institutional upper limit of normal (ULN)
• Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase
[SGOT])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT])
=< 2.5 x institutional ULN unless the patient has dermatomyositis and in the opinion
of the investigator the elevation is due to diabetes mellitus (DM)
• Creatinine ULN OR glomerular filtration rate (GFR) >= 30 mL/min (if using the
Cockcroft-Gault formula)
• Human immunodeficiency virus (HIV)-infected patients on effective antiretroviral
therapy with undetectable viral load within 6 months are eligible for this trial
• If evidence of chronic hepatitis B virus (HBV) infection, HBV viral load must be
undetectable on suppressive therapy if indicated
• If history of hepatitis C virus (HCV) infection, must be treated with undetectable HCV
viral load
• Patients with treated brain metastases are eligible if there is no evidence of
progression for at least 4 weeks after central nervous system (CNS)-directed
treatment, as ascertained by clinical examination and brain imaging (magnetic
resonance imaging [MRI] or computed tomography [CT] scan) during the screening period.
Patients with new or progressive brain metastases (active brain metastases) or
leptomeningeal disease are eligible if the treating physician determines that
immediate CNS specific treatment is not required and is unlikely to be required for at
least 4 weeks (or scheduled assessment after the first cycle of treatment), and a
risk-benefit analysis (discussion) by the patient and the investigator favors
participation in the clinical trial
• The effects of nivolumab on the developing human fetus are unknown. For this reason,
women of child-bearing potential (WOCBP) and men must agree to use adequate
contraception (hormonal or barrier method of birth control; abstinence) prior to study
entry and for the duration of study participation. WOCBP receiving nivolumab will be
instructed to adhere to contraception for a period of 5 months after the last dose of
investigational product. Men receiving nivolumab and who are sexually active with
WOCBP will be instructed to adhere to contraception for a period of 7 months after the
last dose of investigational product. Women of childbearing potential must have a
negative serum or urine pregnancy test (minimum sensitivity 25 IU/L or equivalent
units of human chorionic gonadotropin [HCG]) within 24 hours prior to the start of
nivolumab. Women must not be breastfeeding. Women who are not of childbearing
potential (i.e., who are postmenopausal or surgically sterile as well as azoospermic
men) do not require contraception. WOCBP is defined as any female who has experienced
menarche and who has not undergone surgical sterilization (hysterectomy or bilateral
oophorectomy) or who is not postmenopausal. Menopause is defined clinically as 12
months of amenorrhea in a woman over 45 in the absence of other biological or
physiological causes. In addition, women under the age of 55 must have a documented
serum follicle stimulating hormone (FSH) level less than 40 mIU/mL. These durations
have been calculated using the upper limit of the half-life for nivolumab (25 days)
and are based on the protocol requirement that WOCBP use contraception for 5
half-lives plus 30 days, and men who are sexually active with WOCBP use contraception
for 5 half-lives plus 90 days. Should a woman become pregnant or suspect she is
pregnant while she or her partner is participating in this study, she (or the
participating partner) should inform the treating physician immediately
• Ability to understand and the willingness to sign a written informed consent document
Exclusion Criteria:
• Patients who have had chemotherapy or radiotherapy within 4 weeks (6 weeks for
nitrosoureas or mitomycin C) prior to entering the study or those who have not
recovered from adverse events (AEs) due to agents administered more than 4 weeks
earlier have not resolved or stabilized. Palliative (limited-field) radiation therapy
(RT) is permitted, if all of the following criteria are met:
• Repeat imaging demonstrates no new sites of bone metastases
• The lesion being considered for palliative radiation is not a target lesion
• Patients with prior therapy with an anti-PD-1 or anti-PD-L1
• Patients with prior allogeneic hematologic transplant
• Patients who are receiving any other investigational agents
• Uncontrolled intercurrent illness including, but not limited to, ongoing or active
infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac
arrhythmia, or psychiatric illness/social situations that would limit compliance with
study requirements
• Patients who have had evidence of active or acute diverticulitis, intra-abdominal
abscess, gastrointestinal (GI) bleeding, obstruction, and abdominal carcinomatosis
which are known risk factors for bowel perforation should be evaluated for the
potential need for additional treatment before coming on study. For the IBD (UC and
CD) cohort, an endoscopic assessment, disease activity index, and disease specific
inclusion/exclusion criteria will substitute for these factors in determining
eligibility with the exception of abdominal carcinomatosis, which should prompt
further evaluation
Biological: Nivolumab
Systemic Lupus Erythematosus, Lymphoma, Sarcoma, Multiple Myeloma, Multiple Sclerosis, Rheumatoid Arthritis, Ulcerative Colitis, Mycosis Fungoides, Dermatomyositis, Advanced Malignant Solid Neoplasm, Metastatic Malignant Solid Neoplasm, Crohn Disease, Hematopoietic and Lymphoid Cell Neoplasm, Brain and Nervous System, Eye and Orbit, Anus, Bones and Joints, Breast - Female, Breast - Male, Cervix, Colon, Corpus Uteri, Esophagus, Head and Neck, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung/Thoracic, Melanoma, skin, Other Digestive Organ, Other Endocrine System, Other Female Genital, Other Male Genital, Other Respiratory and Intrathoracic Organs, Other Skin, Other Urinary, Ovary, Pancreas, Prostate, Rectum, Stomach, Thyroid, Urinary Bladder, Leukemia, Other, Hodgkins Lymphoma, Kaposis sarcoma, Leukemia, Not Otherwise Specified, Lymphoid Leukemia, Myeloid and Monocytic Leukemia, Non-Hodgkins Lymphoma, Other Hematopoietic, Small Intestine, Soft Tissue, Unknown Sites, Inflammatory Bowel Disease, Autoimmune Disease, Sjogren Syndrome, Systemic Scleroderma, Unresectable Malignant Solid Neoplasm
Trastuzumab Deruxtecan for the Treatment of HER2+ Newly Diagnosed or Recurrent Osteosarcoma
This phase II trial studies the effects of trastuzumab deruxtecan in treating patients with
HER2 positive osteosarcoma that is newly diagnosed or has come back (recurrent). Trastuzumab
deruxtecan is a monoclonal antibody, called trastuzumab, linked to a chemotherapy drug called
deruxtecan. Trastuzumab attaches to HER2 positive cancer cells in a targeted way and delivers
deruxtecan to kill them.
• Patients must be > 12 years and =< 39 years of age at the time of study enrollment
• Patients must have had histologic verification of osteosarcoma at original diagnosis
or relapse
• Patients with diagnoses of osteosarcoma and confirmed HER2 expression of > 10% of
osteosarcoma cells are eligible for the intervention
• Note: There is a mandatory tissue submission for HER2 staining during the
Step 0 Eligibility Screening process. Metastatic tissue, when possible from
the most recent relapse, is strongly preferred for HER2 staining over
archival tissue from primary resection or diagnostic biopsy. The evaluation
period for HER2 staining to determine eligibility for therapy will be less
than 4 weeks from screening enrollment
• Patients must have measurable disease according to Response Evaluation Criteria in
Solid Tumors (RECIST) 1.1. Patients with clinically inactive brain metastases may be
included in the study. Patients with treated brain metastases that are no longer
symptomatic and who require no treatment with corticosteroids or anticonvulsants may
be included in the study if they have recovered from the acute toxic effect of
radiotherapy. Lastly, patient must have unresectable lesions or lesions with no
intention to surgically remove in the 6 months following enrollment
• Patient's current disease state must be one for which they have received at least
standard initial therapy, defined as systemic therapy combined with either radiation
or surgery for local control of the primary tumor at diagnosis. Prior therapy after
relapse is not required
• Patients must have a performance status corresponding to Eastern Cooperative Oncology
Group (ECOG) scores of 0 or 1. Use Karnofsky for patients > 16 years of age and Lansky
for patients =< 16 years of age. Patients who are non-ambulatory as a result of prior
surgical treatment for osteosarcoma should be considered ambulatory for the purposes
of assessing performance status
• Patients must have recovered from the acute toxic effects of all prior anti-cancer
therapy and must meet the following minimum duration from prior anti-cancer directed
therapy prior to enrollment. If after the required timeframe, the numerical
eligibility criteria are met, e.g., blood count criteria, the patient is considered to
have recovered adequately
• Cytotoxic chemotherapy or other anti-cancer agents known to be myelosuppressive:
For agents not listed, the duration of this interval must be discussed with the
study chair and the study-assigned Research Coordinator prior to enrollment
• >= 21 days after the last dose of myelosuppressive chemotherapy (42 days if
prior nitrosourea)
• Anti-cancer agents not known to be myelosuppressive (e.g., not associated with
reduced platelet or absolute neutrophil count [ANC] counts): >= 7 days after the
last dose of agent
• Antibodies: >= 4 weeks (28 days) must have elapsed from infusion of last dose of
antibody, and toxicity related to prior antibody therapy must be recovered to
grade =< 1
• Corticosteroids
• Hematopoietic growth factors: >= 14 days after the last dose of a long-acting
growth factor (e.g. pegfilgrastim) or 7 days for short-acting growth factor. For
agents that have known adverse events occurring beyond 7 days after
administration, this period must be extended beyond the time during which adverse
events are known to occur
• Interleukins, interferons and cytokines (other than hematopoietic growth
factors): >= 21 days after the completion of interleukins, interferon or
cytokines (other than hematopoietic growth factors)
• Stem cell Infusions (with or without total body irradiation [TBI]):
• Allogeneic (non-autologous) bone marrow or stem cell transplant, or any stem
cell infusion including donor lymphocyte infusion (DLI) or boost infusion:
>= 84 days after infusion and no evidence of graft versus host disease
(GVHD)
• Autologous stem cell infusion including boost infusion: >= 30 days
• Vellular therapy: >= 30 days after the completion of any type of cellular therapy
(e.g., modified T cells, natural killer [NK] cells, dendritic cells, etc.)
• Radiation therapy (XRT)/external beam irradiation including protons: >= 4 weeks
(28 days) including palliative radiation therapy to the chest. >= 14 days after
palliative local XRT to areas other than the chest or for whole brain
radiotherapy
• Radiopharmaceutical therapy (e.g., radiolabeled antibody, samarium): >= 42 days
after systemically administered radiopharmaceutical therapy
• Patients must not have received prior HER2 therapies including antibody drug
conjugates (e.g. TDM-1 or DS-8201a), HER2 directed cellular therapies, HER2
receptor therapy (e.g. trastuzumab, pertuzumab) or small molecule antagonists of
HER2 (e.g lapatinib or neratinib)
• Patients must be at least 7 days from the date of last surgery
• Peripheral absolute neutrophil count (ANC) >= 1000/mm^3, (granulocyte
colony-stimulating factor [G-CSF] administration is not allowed within 1 week prior to
Step 1 screening assessment) (for patients with solid tumors without known bone marrow
involvement)
• Platelet count >= 100,000/mm^3 (transfusion independent, defined as not receiving
platelet transfusions for at least 7 days prior to enrollment) (for patients with
solid tumors without known bone marrow involvement)
• Hemoglobin >= 8.0 g/dL at baseline (Red Blood Cell transfusion is not allowed within 1
week prior to screening assessment) (for patients with solid tumors without known bone
marrow involvement)
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 60
mL/min/1.73 m^2 or a serum creatinine based on age/gender as follows:
• Age: 12 to < 13 years; Maximum serum creatinine (mg/dL): 1.2 (male); 1.2 (female)
• Age: 13 to < 16 years; Maximum serum creatinine (mg/dL): 1.5 (male); 1.4 (female)
• Age: >= 16 years; Maximum serum creatinine (mg/dL): 1.7 (male); 1.4 (female)
• For participants less than 18 years of age that screen fail only based on
creatinine, a 24 hour urine collection may be used instead to confirm
eligibility. A calculated GFR > 60 mL/min/1.73 m^2 using a 24 hour
collection will meet criteria for inclusion on this trial
• Bilirubin (sum of conjugated + unconjugated or total) =< 1.5 x upper limit of normal
(ULN) for age. For patients with documented Gilbert's syndrome (unconjugated
hyperbilirubinemia) bilirubin must be < 3 x ULN for age (patients with solid tumors)
• Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) and
serum glutamic-oxaloacetic transaminase (SGOT) (aspartate aminotransferase [AST]) =< 3
x ULN. (if liver metastases present =< 5 x ULN). For the purpose of this study, the
ULN for ALT is 45 U/L regardless of baseline and the ULN for AST is 50 U/L regardless
of baseline (patients with solid tumors)
• Serum albumin >= 2.5 g/dL (patients with solid tumors)
• International normalized ratio (INR)/prothrombin time (PT) and either partial
thromboplastin or activated partial thromboplastin time (aPTT) =< 1.5 x ULN (patients
with solid tumors)
• Shortening fraction of >= 27% by echocardiogram, or ejection fraction of >= 50% by
either an echocardiogram (ECHO) or multigated acquisition (MUGA) scan within 28 days
before Step 1 enrollment
• Corrected QT interval (QTc) prolongation to < 470 ms (females) or < 450 ms (males)
based on average triplicate 12-lead electrocardiogram (ECG)
• Pulse oximetry > 93% on room air
• Patients with seizure disorder may be enrolled if on anticonvulsants and well
controlled as evidenced by no increase in seizure frequency in the prior 7 days
• Nervous system disorders (Common Terminology Criteria for Adverse Events [CTCAE]
version [v]5) resulting from prior chemotherapy, surgery, and/or radiation must be =<
grade 2, with the exception of decreased tendon reflex (DTR). Any grade of DTR is
eligible
• All patients and/or their parents or legally authorized representatives must sign a
written informed consent. Assent, when appropriate, will be obtained according to
institutional guidelines
Exclusion Criteria:
• Pregnant, planning to become pregnant, or breast-feeding women will not be entered on
this study because there is yet no available information regarding human fetal or
teratogenic toxicities. Pregnancy tests must be obtained in girls who are
post-menarchal. Males or females of reproductive potential may not participate unless
they have agreed to use two effective methods of birth control, including a medically
accepted barrier or contraceptive method (e.g., male or female condom) for the
duration of the study and upon completion of the study and for at least 7 months for
females and 4 months for males after the last dose of study drug. Abstinence is an
acceptable method of birth control
• Methods considered as highly effective methods of contraception include:
• Combined (estrogen and progestogen containing) hormonal contraception
associated with inhibition of ovulation:
• Oral
• Intravaginal
• Transdermal
• Progestogen-only hormonal contraception associated with inhibition of
ovulation:
• Oral
• Injectable
• Implantable
• Intrauterine device (IUD)
• Intrauterine hormone-releasing system (IUS)
• Bilateral tubal occlusion
• Vasectomized partner
• Complete sexual abstinence defined as refraining from heterosexual
intercourse. Periodic abstinence (calendar, symptothermal, post-ovulation
methods) is not an acceptable method of contraception
• Non-child-bearing potential defined as pre-menopausal females with a documented tubal
ligation or hysterectomy; or postmenopausal defined as 12 months of spontaneous
amenorrhea (in questionable cases, a blood sample with simultaneous
follicle-stimulating hormone [FSH] > 40 mIU/mL and estradiol < 40 pg/mL [< 147 pmol/L]
is confirmatory). Females on hormone replacement therapy (HRT) and whose menopausal
status is in doubt will be required to use one of the contraception methods outlined
for women of child-bearing potential if they wish to continue their HRT during the
study. Otherwise, they must discontinue HRT to allow confirmation of post-menopausal
status prior to study enrollment. For most forms of HRT, at least 2-4 weeks will
elapse between the cessation of therapy and the blood draw; this interval depends on
the type and dosage of HRT. Following confirmation of their post-menopausal status,
they can resume use of HRT during the study without use of a contraceptive method
• Male subjects must not freeze or donate sperm starting at Screening and throughout the
study period, and at least 4 months after the final study drug administration.
Preservation of sperm should be considered prior to enrolment in this study
• Female subjects must not donate, or retrieve for their own use, ova from the time of
Screening and throughout the study treatment period, and for at least 7 months after
the final study drug administration
• Patients receiving corticosteroids who have not been on a stable or decreasing dose of
corticosteroid for at least 7 days prior to enrollment are not eligible. If used to
modify immune adverse events related to prior therapy, >= 14 days must have elapsed
since last dose of corticosteroid
• Patients who are currently receiving another investigational drug are not eligible
• Patients who are currently receiving other anti-cancer agents are not eligible
• Patients who are receiving cyclosporine, tacrolimus or other agents to prevent
graft-versus-host disease post bone marrow transplant are not eligible for this trial
• Patients who are receiving chloroquine or hydroxychloroquine within 14 days are not
eligible for this trial
• Patients who have received a prior solid organ transplantation are not eligible
• Patients with a medical history of myocardial infarction within 180 days before
enrollment, symptomatic congestive heart failure (CHF) (New York Heart Association
Class II to IV) or troponin levels consistent with myocardial infarction as defined
according to the manufacturer 28 days prior to enrollment are not eligible
• Patients who have a pleural effusion, ascites or pericardial effusion that requires
drainage, peritoneal shunt, or Cell-free and Concentrated Ascites Reinfusion Therapy
(CART) are not eligible
• Patients who have spinal cord compression or clinically active central nervous system
metastases, defined as untreated and symptomatic, or requiring therapy with
corticosteroids or anticonvulsants to control associated symptoms are not eligible
• Patients with a known history of severe hypersensitivity to DS-8201a or any excipient
contained in the DS-8201a drug formulation are not eligible
• Patients who have an uncontrolled infection or non-healing surgical site are not
eligible
• Patients with a known history of substance abuse or any other clinically significant
medical conditions (i.e. psychological conditions) that may, in the opinion of the
investigator, interfere with the patient's participation in the clinical study or
evaluation of the clinical study results are not eligible
• Patients who have pulmonary compromise, ex hypoxia, resulting from intercurrent
pulmonary illnesses including, but not limited to, any underlying pulmonary disorder
(i.e. pulmonary emboli within three months of the study enrollment, severe asthma,
severe chronic obstructive pulmonary disease (COPD), restrictive lung disease, pleural
effusion etc.), or prior pneumonectomy are not eligible
• Patients who have a history of (non-infectious) ILD (interstitial lung
disease)/pneumonitis that required steroids, has current ILD/pneumonitis, or where
suspected ILD/pneumonitis cannot be ruled out by imaging at screening are not eligible
• Patients who have a pleural effusion, ascites or pericardial effusion that requires
drainage, peritoneal shunt, or cell-free and concentrated ascites reinfusion therapy
(CART) are not eligible. (Drainage and CART are not allowed within 2 weeks prior to
screening assessment)
• Patients who, in the opinion of the investigator, may not be able to comply with the
safety monitoring requirements of the study are not eligible
Biological: Trastuzumab Deruxtecan
Recurrent Osteosarcoma, Osteosarcoma, Bones and Joints
Assessment of ANK-700 in Patients With Relapsing Remitting Multiple Sclerosis (MoveS-it)
A safety study of ANK-700 in patients with relapsing remitting multiple sclerosis. The study
has two parts:
Part A - first in human study in which patients receive a single dose of ANK-700 Part B -
patients will receive three doses of either ANK-700 or placebo
Call 214-648-5005 studyfinder@utsouthwestern.edu
Benjamin Greenberg
105091
All
18 Years to 55 Years old
Phase 1
This study is NOT accepting healthy volunteers
NCT04602390
STU-2020-0880
Show full eligibility criteria
Hide eligibility criteria
Inclusion Criteria:
• Diagnosed with RRMS per revised McDonald criteria (2017) with an EDSS score ≤ 6.5 at
screening
• Neurologically stable with no evidence of relapse within the 28 days before signing
the informed consent form (ICF)
• Either not currently receiving disease modifying MS therapy, or currently using
fumarate drugs (dimethyl fumarate or diroximel fumarate)
• Patients must use a highly effective method of birth control or are sterile or
postmenopausal as confirmed by study Investigator
• Patient has signed and understands the ICF
Exclusion Criteria:
• Diagnosis of primary progressive MS or secondary progressive MS
• Uncontrolled or significant medical conditions (including active infection or chronic
hepatitis) which, in the opinion of the Investigator, preclude participation
• Patients treated with glatiramer acetate, parenteral steroids or adrenocorticotropic
hormone, β-interferon, plasma exchange, fingolimod, ozanimod, or siponimod within the
3 months prior to first dose
• Patients treated with cytotoxic agents (including, but not limited to, cladribine,
mitoxantrone, cyclophosphamide, azathioprine, and methotrexate), laquinimod,
teriflunomide, or IV gamma globulin within 12 months prior to first dose
• Patients treated with monoclonal antibody therapy (including natalizumab, daclizumab,
rituximab, ofatumumab, and ocrelizumab) within 24 months prior to first dose
• Patients previously treated with alemtuzumab, total lymphoid irradiation, mesenchymal
stem cell or hematopoietic stem cell transplantation, or tolerance-inducing therapies
for MS
• Contraindication to or inability to undergo gadolinium-enhanced magnetic resonance
imaging (MRI) scan
• Use of any investigational drug or experimental procedure within previous 6 months
that would interfere with the assessment of ANK-700
• Patients who are pregnant or breastfeeding
Olanzapine Versus Megestrol Acetate for the Treatment of Loss of Appetite Among Advanced Cancer Patients
This phase III trial compares the effects of olanzapine versus megestrol acetate in treating
loss of appetite in patients with cancer that has spread to other places in the body
(advanced). Olanzapine may stimulate and increase appetite. This study aims to find out if
olanzapine is better than the usual approach (megestrol acetate) for stimulating appetite and
preventing weight loss.
• Women and men of reproductive potential should agree to use an appropriate method of
birth control throughout their participation in this study due to the teratogenic
potential of the therapy utilized in this trial. Appropriate methods of birth control
include abstinence, oral contraceptives, implantable hormonal contraceptives or double
barrier method (diaphragm plus condom)
• Diagnosis of advanced cancer
• Patient-reported 2-month weight loss of at least 5 pounds (2.3 kilograms) and/or
physician-estimated caloric intake of less than 20 calories/kilogram of body weight
per day
• The patient must perceive loss of appetite and/or weight as a problem; and have an
appetite score of 4 or worse on the "Please rate your appetite…." question that
requires a patient response on a 0-10 numeric rating scale
• Not receiving ongoing tube feedings or parenteral nutrition at the time of
registration
• Not currently using systemic adrenal steroids (with the exception of short-term
dexamethasone within 3 days of chemotherapy for control of chemotherapy side effects)
• No use of androgens, progesterone analogs, or other appetite stimulants within the
past month
• Patient should not have poorly controlled hypertension or congestive heart failure at
registration
• Patient should not have an obstruction of the alimentary canal, malabsorption, or
intractable vomiting (defined as vomiting more than 3 times per day over the preceding
week)
• Not currently using olanzapine for another medical condition or had previously used
olanzapine for chronic nausea or for any pre-existing psychotic disorder
• Patient should not have had a previous blood clot at any time in the past
• No history of poorly controlled diabetes
• No symptomatic leptomeningeal disease or known brain metastases as these patients may
have difficulty taking oral medications
• No history of hypersensitivity to olanzapine or megestrol acetate
• No COVID-19 infection in the past that, in the opinion of the treating physician, had
left patients with compromised taste, which has not resolved at the time of
registration
• Not pregnant and not nursing, because this study involves an investigational agent
whose genotoxic, mutagenic and teratogenic effects on the developing fetus and newborn
are unknown. Therefore, for women of childbearing potential only, a negative urine or
serum pregnancy test done =< 14 days prior to registration is required
• Age >= 18 years
• Eastern Cooperative Oncology Group (ECOG) performance status 0, 1 or 2
• Estimated life expectancy of 3 months or longer
• Serum creatinine =< 2.0 mg/dL
• Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) =< 3 x upper limit
of normal (ULN)
• Fasting glucose > 1410 mg/dl
• Granulocytes > 1000/hpf
• No treatment with another antipsychotic agent, such as risperidone, quetiapine,
clozapine, butyrophenone within 30 days of enrollment
• In order to complete the mandatory patient-completed measures, participants must be
able to speak and/or read English or Spanish. Sites seeking to enroll Spanish-speaking
patients should have access to Spanish speaking staff on site or through the use of a
translation service to be able to conduct the informed consent discussion in Spanish,
and to conduct the weekly phone calls
Exclusion Criteria:
• Psychiatric illness which would prevent the patient from giving informed consent
• Medical condition such as uncontrolled infection (including human immunodeficiency
virus [HIV]), uncontrolled diabetes mellitus or cardiac disease which, in the opinion
of the treating physician, would make this protocol unreasonably hazardous for the
patient
• Patients who cannot swallow oral formulations of the agents
• Patients with impaired decision-making capacity (such as with a diagnosis of dementia
or memory loss) are not eligible for this study
• No presence of a hormone-sensitive tumor, such as breast, endometrial, or prostate
cancer (this exclusion criterion is intended to circumvent any confounding
antineoplastic effects of megestrol acetate)
A Study of Avapritinib in Pediatric Patients With Solid Tumors Dependent on KIT or PDGFRA Signaling
This is a Phase 1/2, multicenter, open-label trial of avapritinib in patients aged 2 to less
than 18 years of age with with relapsed/refractory (R/R) solid tumors with mutations
(including non-synonymous point mutations, insertions, and deletions) in KIT or PDGFRA, or
gliomas with the H3K27M mutation, and no available alternative treatment options. This is a
single-arm trial in which all patients will receive avapritinib. The study consists of 2
parts: dose confirmation, safety, and PK (Part 1) and initial efficacy, safety, and PK at the
Part 2 recommended dose (Part 2).
• Patient has confirmed diagnosis of a R/R solid or CNS tumor with a mutation in KIT or
PDGFRA (confirmed by local mutational testing of tumor sample) that has progressed
despite standard therapy and no alternative treatment option is available OR Confirmed
diagnosis of H3K27M mutant glioma that has failed standard therapy or for which no
standard therapy that may convey clinical benefit exists, as judged by the
Investigator.
• Patients with CNS disease should be on a stable dose (≤10% change) of corticosteroids
for at least 7 days prior to first dose of avapritinib, with no plans for dose
escalation.
• Disease extent
1. Part 1: All patients must have at least 1 measurable lesion as defined by RECIST
v1.1 or RANO (for CNS tumors). If radiation therapy has been administered, at
least
1 measurable lesion must not have been irradiated, or must have clearly
progressed since being irradiated.
2. Part 2: At least one measurable lesion as defined by RECIST v1.1 (RANO for CNS
tumors). If radiation therapy has been administered, at least 1 measurable lesion
must not have been irradiated within the previous 12 weeks, or must have clearly
progressed since being radiated (per RANO). For up to 5 patients with H3K27M
mutant gliomas where there is no standard therapy that may convey clinical
benefit as judged by the investigator, progression of disease of a measurable
lesion after irradiation is not required.
• A Lansky (≤16 years of age) or Karnofsky (>16 years of age) score of at least 50. If
the patient is unable to walk due to paralysis, but is mobile in a wheelchair, the
patient is considered ambulatory for the purpose of assessing their performance
status.
• Patient agrees to utilize contraception consistent with local regulations
Exclusion Criteria:
• Patient has any of the following within 14 days before the first dose of study
treatment:
1. Platelet count <75 × 109/L (<100 × 109/L if a CNS tumor).
2. Absolute neutrophil count (ANC) <1.0 × 109/L.
3. Hemoglobin <8.0 g/dL (RBC transfusion ≥14 days before test is permitted to meet
criterion).
4. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >3 × the upper
limit of normal (ULN) for age; except in patients with tumor involvement of the
liver who must not have AST and ALT >5 × ULN for age.
5. Total bilirubin >1.5 mg/dL for age; and in presence of Gilbert's syndrome, total
bilirubin.
> 3 × ULN or direct bilirubin > 1.5 × ULN.
6. Serum creatinine >1.5 × ULN for age.
7. International normalized ratio (INR) or prothrombin time (PT) >ULN (>1.5 × ULN if
on prophylactic reversible anticoagulants).
• Patient has a QT interval corrected using Fridericia's formula (QTcF) >470 msec.
Patient has a familial or personal history of prolonged QT syndrome or Torsades de
pointes.
• Patient has clinically significant, uncontrolled, cardiovascular disease including
congestive heart failure Grade III or IV according to the New York Heart Association
(NYHA) classification; myocardial infarction or unstable angina within the previous 6
months, uncontrolled hypertension (>99th percentile for age), or clinically
significant, uncontrolled arrhythmias, including bradyarrhythmias that may cause QT
prolongation (eg, Type II second degree heart block or third-degree heart block).
• Patient received the following systemic antineoplastic therapies:
1. Systemic antineoplastic therapy (including experimental therapy within 5
half-lives or 28 days [6 weeks if prior nitrosurea], whichever is shorter).
2. Focal external beam radiotherapy, including stereotactic radiosurgery, within 6
weeks prior to the first dose of avapritinib to either target or non-target
lesions. Systemic radiopharmaceuticals, including nonstereotactic radiosurgery,
within 2 weeks of the first dose of avapritinib (within 6 weeks for patients with
CNS tumors). Craniospinal irradiation within 12 weeks prior to the first dose of
avapritinib.
3. All AEs related to other antineoplastic therapies (eg, systemic antineoplastics,
radiotherapy) must have resolved to Grade ≤1 (Grade ≤2 for peripheral neuopathy
and/or ototoxicity) prior to the first dose of avapritinib.
• Patient has previously received treatment with avapritinib.
• Patient received autologous stem cell transplant (SCT) following myeloablative therapy
or chimeric antigen receptor T cell (CAR-T) therapy within 3 months prior to the first
dose of avapritinib or prior allogeneic SCT within 1 year and no evidence of Grade 1
or greater graft-versus-host disease and no immunosuppressants for graft-versus-host
disease (steroids for primary malignancy being permitted). Patients who received stem
cell reinfusion following nonmyeloablative therapy are eligible once they meet the
peripheral blood count criteria in Exclusion Criterion #1.
• Patient requires on going treatment or has received treatment within 28 days before
the start of avapritinib administration with drugs or foods that are strong CYP3A
inhibitors or inducers or EIAEDs (eg, carbamazepine, phenytoin, phenobarbital, and
primidone). Please refer to Appendix 1 for a list of these drugs and/or foods.
• Patient has had a major surgical procedure within 14 days of the first dose of study
treatment (procedures such as central venous catheter placement, tumor needle biopsy,
and feeding tube placement are not considered major surgical procedures).
• Patient has a history of another primary malignancy that has been diagnosed or
required therapy within 3 years before the first dose of avapritinib. The following
prior malignancies are not exclusionary: completely resected basal cell and squamous
cell skin cancer, curatively treated localized prostate cancer, and completely
resected carcinoma in situ of any site.
• Female subjects of childbearing potential who are unwilling, if not post-menopausal or
surgically sterile, to abstain from sexual intercourse or employ highly effective
contraception from the time of informed consent and for at least 6 weeks after the
last dose of study treatment. Male subjects who are unwilling, if not surgically
sterile, to abstain from sexual intercourse or employ highly effective contraception
from the time of informed consent and for at least 6 weeks after the last dose of
study treatment. Refer to Section 5.4.2 for acceptable methods of contraception.
• Patient is pregnant
• Patient is breastfeeding.
• Patient has prior or ongoing clinically significant illness, medical condition,
surgical history, physical finding, or laboratory abnormality that, in the
Investigator's opinion, could affect the safety of the patient; alter the absorption,
distribution, metabolism, or excretion of the study drug; or impair the assessment of
study results.
• History of thrombosis requiring treatment within the past 6 months.
• Patients who require anticoagulants, with the exception of stable doses of
prophylactic reversible anticoagulants.
• Patients who are unable to swallow tablets (in Part 1) or mini-tablets (in Part 2)
within the sprinkle capsules.
• Patients with a known risk of intracranial bleeding, such as a brain aneurysm that has
not been removed or repaired, or a history of intracranial bleeding within the past
year, or radiographic evidence of hemorrhage on Screening MRI. Exceptions are patients
with primary CNS tumors who are eligible unless CNS bleeding has occurred within 2
weeks of the first dose of avapritinib and patients with punctate hemorrhages <3 mm.
• History of a seizure disorder that is not well controlled on current antiepileptic
medications. EIAEDs carbamazepine, phenytoin, phenobarbital, and primidone are
prohibited.
• Patient is unwilling or unable to comply with scheduled visits, treatment
administration plan, laboratory tests, or other study procedures and study
restrictions