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
Study of SRP-4045 and SRP-4053 in Participants With Duchenne Muscular Dystrophy (DMD) (ESSENCE)
The main objective of this study is to evaluate the efficacy of SRP-4045 and SRP-4053
compared to placebo in participants with DMD with out-of-frame deletion mutations amenable to
skipping exon 45 and exon 53, respectively.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Susan Iannaccone
13463
Male
6 Years to 13 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT02500381
STU 082015-050
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Inclusion Criteria:
• Genotypically confirmed DMD, with genetic deletion amenable to exon 45 or exon 53
skipping
• Stable dose of oral corticosteroids for at least 24 weeks prior to Week 1, and the
dose is expected to remain constant throughout the study (except for modifications to
accommodate changes in weight).
• Intact right and left biceps or 2 alternative upper muscle groups
• Mean 6MWT ≥300 meters and ≤450 meters
• Stable pulmonary function: forced vital capacity (FVC) ≥50% predicted
Exclusion Criteria:
• Treatment with gene therapy at any time
• Previous treatment with SMT C1100 within 1 week prior to Week 1 and previous treatment
with PRO045 (BMN 045), PRO053 (BMN 053), or PRO051 (BMN 051) within 24 weeks prior to
Week 1
• Current or previous treatment with any other experimental treatment within 12 weeks
prior to Week 1
• Major surgery within 3 months prior to Week 1
• Presence of other clinically significant illness
Other inclusion/exclusion criteria may apply.
A Study of Therapeutic Iobenguane (131-I) and Vorinostat for Recurrent or Progressive High-Risk Neuroblastoma Subjects (OPTIMUM)
The purpose of this study is to evaluate the efficacy and safety of 131I-MIBG in combination
with Vorinostat in patients with Recurrent or Progressive neuroblastoma
1. Subjects with a diagnosis of iobenguane avid, high-risk neuroblastoma based on Revised
INRC criteria at the time of study enrollment with recurrent or progressive disease at
any time prior to enrollment, regardless of overall response to frontline therapy,
where frontline therapy includes a minimum of 4 cycles of induction therapy at any
time prior to enrollment.
2. May have had prior 131I-MIBG therapy, provided:
1. It has been at least 6 months from the date of last 131I-MIBG ;
2. Response was other than progressive disease on first restaging after 131I-MIBG ;
3. Prior 131I-MIBG was given as monotherapy and not in combination with systemic
anticancer agents;
4. Cumulative lifetime dose of 131I-MIBG at enrollment does not exceed 18 mCi/kg.
3. All soft tissue lesions identified on CT/MRI scans must be iobenguane avid lesions on
an (123I)-iobenguane scan, or
1. any progressive non-iobenguane avid lesion is proven by biopsy to be a
non-neuroblastoma lesion.
2. any other non-avid lesion is comprised of a fibrotic or scarred mass as shown by
routine imaging and confirmed by the investigator.
4. Adequate cryopreserved autologous peripheral blood stem cells or bone marrow (at least
2 aliquots of 2.0 × 10exp6 CD34/kg at the time of study enrollment).
5. If a male, must agree to use an adequate contraception method as deemed appropriate by
the Investigator (e.g., vasectomy, condoms) or partner using effective contraception
and to not donate sperm during the study and for 90 days after receiving the last dose
of study drug.
6. If a female of childbearing potential, have a negative serum pregnancy test result
prior to each dosing and, if sexually active, be practicing an effective method of
birth control [e.g., intrauterine device, double-barrier method (i.e., diaphragm, or a
cervical cap) with intravaginal spermicidal foam, cream or gel], or male partner
sterilization throughout the study.
7. Age at study entry ≥1 year.
8. Previous platelet transfusions are permitted, as long as the subject has a platelet
count ≥50,000/μL without transfusion support for at least 1 week.
9. Subjects must have a minimum pulse oximetry measurement of at least 94% at baseline.
10. An absolute neutrophil count ≥750/μL without growth factor for 5 days.
11. Liver function parameter results: total bilirubin ≤2 × upper limit of normal for age,
and Serum alanine aminotransferase (glutamic-pyruvic transaminase) and serum aspartate
aminotransferase (glutamic-oxaloacetic transaminase) ≤ 10 times the upper limit of
normal (for all sites, the upper limit of normal for alanine aminotransferase is
defined as 45 U/L).
12. Normal thyroid function as measured by T4 or TSH or have abnormal results that are not
considered clinically important by the Investigator or may be receiving levothyroxine.
13. Cardiac Function: shortening fraction of ≥ 27% by echocardiogram or ejection fraction
≥ 50% documented by echocardiogram or radionuclide angiogram within 1 month prior to
Visit 1 (Baseline).
14. Karnofsky Performance Status (for subjects >16 years of age) or the Lansky Performance
Status Performance Status (for subjects 1 to 16 years of age) ≥50%.
15. Full recovery from the toxic effects of any prior therapy.
16. Coagulation Function:
1. International Normalized Ratio (INR) < 1.5
2. Partial thromboplastin time (PTT) < 1.5 times upper limit of normal.
Exclusion Criteria:
1. Subjects within 5 half-lives after any antibody-based immunotherapy, or have not
recovered from effects of any biologic therapy.
2. Subjects <12 weeks after myeloablative therapy with autologous stem cell transplant.
3. Subjects who have had an allogeneic stem cell treatment less than 4 months from Visit
1 are excluded. Those who have received allogeneic stem cell treatment more than 4
months from Visit 1 must have recovered and have no active graft versus host disease
(GVHD) to be eligible.
4. Subjects must not have received radiation for a minimum of 2 weeks prior to study
enrollment. Subjects whose only site(s) of disease have been radiated are eligible as
long as the subject has MIBG avidity 2 weeks after completion of radiation. A minimum
of 12 weeks prior to study enrollment is required following prior large field
radiation therapy (ie, craniospinal, whole abdominal, total lung, > 50% marrow space)
5. History of total body irradiation.
6. Subjects do not have adequate renal function defined as GFR ≥ 70 mL/min/1.73 m2 either
by creatinine clearance or radioisotope direct measurement or by calculation with the
Schwartz formula
7. Subjects who are on hemodialysis.
8. Pregnancy or breastfeeding.
9. Significant active infections including active hepatitis B, or hepatitis C infection,
or known infection with human immunodeficiency virus (HIV) (testing for HIV is not
required prior to study entry).
10. Clinically important cardiac, pulmonary, and hepatic impairment.
11. Vorinostat treatment exclusion criteria (subjects, who meet any one of these criteria
and otherwise meet eligibility criteria, are still eligible for 131I-MIBG monotherapy)
1. Since valproic acid has HDAC inhibitory activity, patients must not have received
valproic acid within 30 days of study entry.
2. Since vorinostat may prolong the QT interval, patients must not be receiving
other medications known to prolong the QT interval at the time of study entry .
Pentamidine must not have been received within 1 week of study enrollment.
3. Patients with a history of deep venous thrombosis that was not associated with
the presence of a central venous catheter.
4. Patients who are receiving Coumadin.
Drug: 131I-MIBG, Drug: 131-MIBG + Vorinostat
Neuroblastoma, Neoplasms, Neuroectodermal Tumors, Brain and Nervous System
A Study of Galcanezumab (LY2951742) in Participants 6 to 17 Years of Age With Episodic Migraine (REBUILD-1)
The main purpose of this study is to evaulate the efficacy and safety of galcanezumab in
participants 6 to 17 years of age for the preventive treatment of episodic migraine. The
primary objective is to demonstrate the superiority of galcanezumab versus placebo in the
reduction of monthly migraine headache days across the 3-month double-blind treatment period.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Eric Remster
150068
All
6 Years to 17 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT03432286
STU 072018-085
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Inclusion Criteria:
• Have a diagnosis of migraine with or without aura as defined by the IHS ICHD-3
guidelines (1.1 or 1.2 according to ICHD-3 [2018]), with a history of migraine
headaches of at least 6 months prior to screening.
Exclusion Criteria:
• Participants who are taking, or are expected to take, therapeutic antibodies during
the course of the study (adalimumab, infliximab, trastuzumab, bevacizumab, etc.).
Prior use of therapeutic antibodies is allowed if that use was more than 12 months
prior to baseline.
• Known hypersensitivity to monoclonal antibodies or other therapeutic proteins, or to
galcanezumab or its excipients.
• Current use or prior exposure to galcanezumab, another CGRP antibody, or CGRP receptor
antibody, including those who have previously completed or withdrawn from this study
or any other study investigating a CGRP antibody.
• History of IHS ICHD-3 diagnosis of new daily persistent headache, cluster headache or
migraine subtypes including hemiplegic (sporadic or familial) migraine and migraine
with brainstem aura (previously basilar-type migraine).
• History of significant head or neck injury within 6 months prior to screening; or
traumatic head injury at any time that is associated with significant change in the
quality or frequency of their headaches, including new onset of migraine following
traumatic head injury.
• Participants with a known history of intracranial tumors or developmental
malformations including Chiari malformations.
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)
Dabrafenib Combined With Trametinib After Radiation Therapy in Treating Patients With Newly-Diagnosed High-Grade Glioma
This phase II trial studies how well the combination of dabrafenib and trametinib works after
radiation therapy in children and young adults with high grade glioma who have a genetic
change called BRAF V600 mutation. Radiation therapy uses high energy rays to kill tumor cells
and reduce the size of tumors. Dabrafenib and trametinib may stop the growth of tumor cells
by blocking BRAF and MEK, respectively, which are enzymes that tumor cells need for their
growth. Giving dabrafenib with trametinib after radiation therapy may work better than
treatments used in the past in patients with newly-diagnosed BRAF V600-mutant high-grade
glioma.
• PRE-ENROLLMENT ELIGIBILITY SCREENING (STEP 0): Patients must be >= 12 months and =< 21
years of age at the time of enrollment on Step 0
• Note: This age range encompasses pre-screening for all HGG patients. Individual
treatment protocols may have different age criteria
• PRE-ENROLLMENT ELIGIBILITY SCREENING (STEP 0): Patient is suspected of having
localized newly-diagnosed HGG, excluding metastatic disease
• PRE-ENROLLMENT ELIGIBILITY SCREENING (STEP 0): Patient and/or their parents or legal
guardians have signed informed consent for eligibility screening on APEC14B1 Part A.
• PRE-ENROLLMENT ELIGIBILITY SCREENING (STEP 0): The specimens obtained at the time of
diagnostic biopsy or surgery must be submitted through APEC14B1 as soon as possible
(ASAP), preferably within 5 calendar days of the procedure.
Please note: See the APEC14B1 Manual of Procedures for a full list of detailed instructions
for submitting required materials and for shipping details
• Patients must be >= 3 years and =< 21 years of age at the time of enrollment
• Patients must have eligibility confirmed by Rapid Central Pathology and Molecular
Screening Reviews performed on APEC14B1
• Newly diagnosed high-grade glioma with BRAF^V600-mutation
• Results for H3 K27M by immunohistochemistry (IHC) or sequencing
• Histologically confirmed high-grade glioma (World Health Organization [WHO] grade
III or IV) including but not limited to: anaplastic astrocytoma (AA), anaplastic
pleomorphic xanthoastrocytoma (aPXA), anaplastic gangliogliomas (aGG),
glioblastoma (GB), and high-grade astrocytoma, not otherwise specified (NOS)
• Patients must have had histologic verification of a high-grade glioma diagnosis.
Cerebrospinal fluid (CSF) cytology by lumbar puncture must be done if clinically
indicated and determined to be safe prior to study enrollment. If cytology proves
positive, the patient would be considered to have metastatic disease and would,
therefore, be ineligible.
• A pre- and post-operative brain magnetic resonance imaging (MRI) with and without
contrast and a baseline spine MRI with contrast must be obtained prior to enrollment.
The requirement for a post-operative MRI is waived for patients who undergo biopsy
only. If the spine MRI is positive, the patient would be considered to have metastatic
disease and would be ineligible.
• Patients must have a performance status corresponding to Eastern Cooperative Oncology
Group (ECOG) scores of 0, 1, or 2. Use Karnofsky for patients > 16 years of age and
Lansky for patients =< 16 years of age.
• Peripheral absolute neutrophil count (ANC) >= 1000/uL (within 7 days prior to
enrollment).
• Platelet count >= 100,000/uL (transfusion independent) (within 7 days prior to
enrollment).
• Hemoglobin >= 8.0 g/dL (may receive red blood cell [RBC] transfusions) (within 7 days
prior to enrollment).
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70
mL/min/1.73 m^2 (within 7 days prior to enrollment) or
• A serum creatinine based on age/gender as follows (within 7 days prior to enrollment):
• Age 3 to < 6 years (Male 0.8 mg/dL, Female 0.8 mg/dL)
• Age 6 to < 10 years (Male 1 mg/dL, Female 1 mg/dL)
• Age 10 to < 13 years (Male 1.2 mg/dL, Female 1.2 mg/dL)
• Age 13 to 16 < years (Male 1.5 mg/dL, Female 1.4 mg/dL)
• Age >= 16 years (Male 1.7 mg/dL, Female 1.4 mg/dL)
• Total bilirubin =< 1.5 x upper limit of normal (ULN) for age (within 7 days prior to
enrollment), and
• Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 135
U/L (within 7 days prior to enrollment). For the purpose of this study, the ULN for
SGPT is 45 U/L.
• Patients with a seizure disorder may be enrolled if their seizures are well controlled
while on non-enzyme inducing anticonvulsants permitted on this study.
• Patients must be enrolled and protocol therapy must be projected to begin no later
than 31 days after definitive surgery (day 0). If a biopsy only was performed, the
biopsy date will be considered the date of definitive surgery. For patients who have a
biopsy or incomplete resection at diagnosis followed by additional surgery, the date
of the last resection will be considered the date of definitive surgery.
• 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.
Exclusion Criteria:
• Patients with intrinsic brainstem or primary spinal cord tumors will be excluded.
• Patients with metastatic disease (defined as neuraxis dissemination either by imaging
or by cytology) will be excluded.
• Patients must not have received any prior tumor-directed therapy including
chemotherapy, radiation therapy, immunotherapy, or bone marrow transplant for the
treatment of HGG other than surgical intervention and/or corticosteroids.
• Previous treatment with dabrafenib or another RAF inhibitor, trametinib or another MEK
inhibitor, or an ERK inhibitor.
• Patients with a history of a malignancy with confirmed activating RAS mutation.
• History of allergic reactions attributed to compounds of similar chemical or biologic
composition to dabrafenib, trametinib, and their excipients.
• Uncontrolled medical conditions (e.g., diabetes mellitus, hypertension, liver disease,
or uncontrolled infection), psychological, familial, sociological, or geographical
conditions that do not permit compliance with the protocol; or unwillingness or
inability to follow the procedures required in the protocol.
• Presence of active gastrointestinal (GI) disease or other condition (e.g., small bowel
or large bowel resection) that will interfere significantly with the absorption of
drugs.
• History of hepatitis B virus, or hepatitis C virus infection (patients with laboratory
evidence of cleared hepatitis B virus and/or hepatitis C virus may be enrolled).
• History or current diagnosis of cardiac disease indicating significant risk of safety
for patients participating in the study such as uncontrolled or significant cardiac
disease, including any of the following:
• Recent myocardial infarction (within the last 6 months);
• Uncontrolled congestive heart failure;
• Unstable angina (within last 6 months);
• Clinically significant (symptomatic) or known, uncontrolled cardiac arrhythmias
(e.g., sustained ventricular tachycardia, and clinically significant second or
third degree atrioventricular [AV] block without a pacemaker) except sinus
arrhythmia within the past 24 weeks prior to the first dose of study treatment;
• Coronary angioplasty or stenting (within last 6 months);
• Intra-cardiac defibrillators;
• Abnormal cardiac valve morphology (>= grade 2) documented by echocardiogram.
• Patients with a history or current evidence of retinal vein occlusion (RVO) or central
serous retinopathy (CSR), or predisposing factors to RVO or CSR (e.g., uncontrolled
glaucoma or ocular hypertension).
• Patients with presence of interstitial lung disease or pneumonitis.
• Female patients who are pregnant are ineligible since there is yet no available
information regarding human fetal or teratogenic toxicities.
• Lactating females are not eligible unless they have agreed not to breastfeed their
infants for the duration of the study and for 4 months following discontinuation of
study therapy.
• Female patients of childbearing potential are not eligible unless a negative pregnancy
test result has been obtained.
• Sexually active patients of reproductive potential (male or female) are not eligible
unless they have agreed to use an effective contraceptive method for the duration of
their study participation and for 4 months following discontinuation of study therapy.
Male patients (including those who have had a vasectomy) taking dabrafenib and
trametinib combination therapy must use a condom during intercourse while on study and
for 16 weeks after stopping treatment, and should not father a child during these
periods. Women of childbearing potential should use effective non-hormonal
contraception during therapy and for 4 weeks following discontinuation of dabrafenib
and at least 4 months following the last dose of trametinib in patients taking
combination therapy. Women should be advised that dabrafenib may decrease the efficacy
of hormonal contraceptives and an alternate method of contraception, such as barrier
methods, should be used.
Cladribine Tablets After Treatment With Natalizumab (CLADRINA)
The purpose of this study is to generate hypotheses regarding the safety, efficacy, and
immunological impact of cladribine tablets after treatment with natalizumab in patients with
relapsing-remitting multiple sclerosis (RRMS) and active secondary progressive multiple
sclerosis (active SPMS).
Call 214-648-5005 studyfinder@utsouthwestern.edu
Olaf Stuve
58631
All
18 Years to 60 Years old
Phase 4
This study is NOT accepting healthy volunteers
NCT04178005
STU-2019-1618
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Inclusion Criteria:
Patients who meet the following inclusion criteria will be eligible for enrollment in the
study:
1. Age between 18 and 60 years, inclusive.
2. Diagnosis of relapsing forms of MS, to include RRMS and active SPMS, diagnosed with
McDonald Criteria 2005, 2010, and/or 2017 (1-3)
3. EDSS 0 •5.5 (Functional system changes in cerebral (or mental) functions and in bowel
and bladder functions not used in determining EDSS for protocol eligibility).
4. Has had a minimum of 12 months of continuous natalizumab therapy (300 mg/d), including
patients receiving extended interval dosing of natalizumab (e.g., less frequently than
every-4-week infusion).
5. Negative history for any relapses at least 28 days prior to enrollment.
6. Weighing between 40 kilograms or more.
7. Female subjects of childbearing potential must use effective methods of contraception
to prevent pregnancy for 4 weeks before initiation of cladribine tablets and must
agree to continue to practice adequate contraception for at least 6 months after the
last dose. Women using systemically acting hormonal contraceptives should add a
barrier method during cladribine treatment and for at least 4 weeks after the last
dose in each treatment year.
8. Female subjects must not be pregnant; female subjects must not be lactating or
breast-feeding at least 10 days after the last dose.
9. Male subjects must be willing to use a condom during dosing and for six months after
the last dose. Alternatively, their female partner must use another form of
contraception (such as an intra-uterine device [IUD], barrier method with spermicide,
or hormonal contraceptive [e.g., implant, injectable, patch or oral]) during dosing
and for six months after last dose.
10. Understands and is capable of following through with study protocol requirements and
assessments.
11. Willing to provide voluntary and informed consent based on the Health Insurance
Portability and Accountability Act (HIPPA).
Exclusion Criteria:
Patients who meet any of the following exclusion criteria will not be eligible for
enrollment in the study:
1. Natalizumab failure based on clinician's discretion.
2. Not active progressive MS (4).
3. A diagnosis of PML or any suspicion of PML.
4. A diagnosis of Clinically Isolated Syndrome
5. Known hypersensitivity to cladribine.
6. Any prior exposure to cladribine.
7. Lymphocyte count not within normal limits of the local, hospital laboratory.
8. Previous or current exposure to mitoxantrone, azathioprine, methotrexate,
cyclophosphamide, myelosuppressive treatments, total lymphoid irradiation.
9. Receiving oral or systemic corticosteroid treatments within the 28 days prior to
enrollment.
10. Receiving cytokine base treatment, Intra Venous Immuno Globulin (IVIG) or Plasma
pheresis, 3 months prior to enrollment in the study.
11. Having platelet count or neutrophil count below the lower limit of the normal range
within the 28 days prior to enrollment in the study.
12. Positive for HIV, or positive hepatitis C antibody test or hepatitis B surface antigen
test and/or core antibody test for IgG and/or IgM.
13. History of tuberculosis (TB), presence of active tuberculosis, or latent tuberculosis
as detected by local standard of practice like imaging (e.g., chest X-ray, chest CT
scan, MRI) and/or positive QuantiFERON-TB Gold test and/or skin test and/or clinical
examination or has had latent TB disease at any time in the past.
14. Immunocompromised subjects, including subjects currently receiving immunosuppressive
or myelosuppressive therapy with, e.g., monoclonal antibodies, methotrexate,
cyclophosphamide, cyclosporine or azathioprine, or chronic use of corticosteroids.
15. Active malignancy or history of malignancy.
16. Received a live vaccine within 6 weeks prior to cladribine tablet administration or
intends to receive a live vaccination during the trial. After the last dose of
cladribine tablets, the subject should avoid live vaccine as long as the subject's
white blood cell counts are not within normal limits.
17. Allergy or hypersensitivity to gadolinium and/or any other contraindication to perform
an MRI.
18. Has any renal condition that would preclude the administration of gadolinium (e.g.
acute or chronic severe renal insufficiency (GFR < 30 mL/min/1.73m2)
-
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
Noninvasive Brain Stimulation on Memory in Individuals With Mild Cognitive Impairment and History of Brain Injury
The study will examine the efficacy of high definition transcranial direct current
stimulation (HD-tDCS) and its influence on episodic memory in patients with amnestic mild
cognitive impairment and a history of Traumatic brain injury. Ten sessions of HD-tDCS to the
dorsal anterior cingulate region is expected to result in improvements in episodic memory
measures immediately following the last session and at a 3-month follow-up.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Christian LoBue
127352
All
50 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT04504630
STU-2019-1769
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Inclusion Criteria:
• Age 50 and older
• Native English speakers
• 12 years of education or higher
• Active diagnosis of aMCI
• History of TBI based on VA/DOD criteria
Exclusion Criteria:
• TBI within the past 2 years
• Lifetime history of stroke, transient ischemic attack, heart attack, or congestive
heart failure
• Lifetime history of epilepsy
• Major psychiatric disorders (i.e., posttraumatic stress disorder, bipolar disorder,
schizophrenia)
• Substance use disorder
• Has metal fragments in head
• Taking medications that may interact with the HD-tDCS effect (i.e., amphetamines,
L-dopa, carbamazepine, sulpiride, pergolide, lorazepam, dextromethorphan,
D-cycloserine, flunarizine, or ropinirole)
Device: High Definition Transcranial Direct Current Stimulation, Device: Sham HD-tDCS
Traumatic Brain Injury, Amnestic Mild Cognitive Impairment, Mild Traumatic Brain Injury, Brain and Nervous System
1. Participant is able and willing to provide informed consent (≥18 years), or informed
consent is obtained by the parent or legal guardian for minor participants, with the
minor providing age appropriate assent, according to local law and regulations;
2. Life expectancy ≥ 12 weeks;
3. Known high-risk neuroblastoma with failure to respond to standard therapy (combination
chemotherapy with or without radiation and surgery), or development of PD at any time;
4. Adequate recovery from acute toxic effects of any prior therapy, as deemed by the
Investigator or treating Sub-Investigator;
5. Adequate liver function as defined by the following laboratory values obtained within
28 days prior to administration of 64Cu-SARTATE: Aspartate aminotransferase (AST) and
alanine aminotransferase (ALT) < 3.0 x upper limit of normal (ULN);
6. Adequate renal function;
7. Adequate laboratory parameters: Absolute neutrophil count > 1.0 x 109/L; Platelet
count > 50 x 109/L; Serum bilirubin <1.5 x ULN;
8. Karnofsky or Lansky performance status ≥50;
9. All participants must have a hematopoietic stem cell product available (minimum CD34+
cell dose is ≥2 x 106 cells/kg);
10. Sexually active participants of reproductive potential must practice an effective
method of birth control while participating on this study, to avoid possible damage to
the fetus. Abstinence is considered acceptable;
11. 64Cu-SARTATE uptake on the 4 hour scan (SUVmax) of any lesion equal to or higher than
that of the liver in order to move on to the therapy phase of the study.
Exclusion Criteria:
1. Participants with disease of any major organ system that would compromise their
ability to tolerate therapy, as deemed by the Investigator or treating
Sub-Investigator;
2. Any other active malignancy, or a history of prior malignancy within the past 3 years;
3. History of cardiac failure as evidenced by: dyspnea at rest, exercise intolerance,
oxygen requirement, clinically significant cardiac dysfunction;
4. Planned administration of chemotherapy, anti-cancer cytokine therapy, immunotherapy,
radiotherapy or other investigational agents within 2 weeks prior to the
administration of 64Cu-SARTATE and 4 weeks prior to the administration of
67Cu-SARTATE;
5. Administration of therapeutic dose of 131I-MIBG within 8 weeks prior to the
administration of 64Cu-SARTATE;
6. EBRT to both kidneys or a single functioning kidney within 12 months prior to the
administration of 64Cu-SARTATE;
7. Administration of any investigational agents within 28 days prior to administration of
64Cu-SARTATE;
8. Treatment with long acting somatostatin analogues (administered within 28 days prior
to the administration of 64Cu-SARTATE), or short acting somatostatin analogues
(administered within 24 hours prior to the administration of 64Cu-SARTATE);
9. Known sensitivity or allergy to somatostatin analogues;
10. Previous PRRT;
11. Female participants who are pregnant or lactating;
12. Participants who are on hemodialysis;
13. QTc interval ≥ 0.45 seconds as measured by Screening ECG;
14. Participants with uncontrolled infection(s);
15. Any medical condition which the Investigator feels may interfere with the procedures
or evaluations of the study;
16. Participants 12 month and younger will be excluded from cohorts where the planned
single or cumulative administered activity is modelled to deliver a radiation dose to
the marrow that exceeds 2 Gy.
Drug: 67Cu-SARTATE, Drug: 64Cu-SARTATE
Neuroblastoma, Refractory Neuroblastoma, Relapsed Neuroblastoma, Brain and Nervous System
A Study to Investigate the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution in Children and Adults With Epileptic Encephalopathy Including Dravet Syndrome and Lennox-Gastaut Syndrome
This is an international, multicenter, open-label, long-term safety study of ZX008 in
subjects with Dravet syndrome, Lennox-Gastaut syndrome or epileptic encephalopathy
Call 214-648-5005 studyfinder@utsouthwestern.edu
Deepa Sirsi
103210
All
2 Years and over
Phase 3
This study is NOT accepting healthy volunteers
NCT03936777
STU-2019-0939
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Inclusion Criteria:
• Male or nonpregnant, nonlactating female
• Satisfactory completion of a core study
• Has a rare seizure disorder, such as epileptic encephalopathy and has successfully
completed another Zogenix-sponsored clinical trials with ZX008
• Subject's caregiver is willing and able to be compliant with study procedures, visit
schedule and study drug accountability
Exclusion Criteria:
• Current cardiac valvulopathy or pulmonary hypertension that is clinically significant
• Moderate or severe hepatic impairment
• Receiving monoamine oxidase inhibitors, serotonin agonists, serotonin antagonists, and
serotonin reuptake inhibitors within 14 days of receiving ZX008
Compatibility of C7 With Ketogenic Diet in Patients Diagnosed With G1D
To explore triheptanoin (C7 oil) compatibility with the ketogenic diet by evaluating EEG, and
seizure rate, glycemia and ketosis in proven G1D patients receiving a ketogenic diet.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Juan Pascual
85158
All
30 Months to 35 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT03301532
STU 102015-091
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Inclusion Criteria:
• Diagnosis of glucose transporter type 1 deficiency (G1D), confirmed by clinical
genotyping at a CLIA-certified laboratory.
• Stable on ketogenic diet at 2.5:1 to 4:1 ratio (i.e., no changes in ratio will have
taken place for 2 months). The initiation of a ketogenic diet is previous to •and
thus is not part of this study.
• Males and females 30 months to 35 years and 11 months old inclusive.
Exclusion Criteria:
• Subjects with evidence of independent, unrelated metabolic and/or genetic disease.
• Subjects with a chronic gastrointestinal disorder, such as irritable bowel syndrome,
crohn's disease, or colitis that could increase the subject's risk of developing
diarrhea or stomach pain.
• Subjects with a BMI (body mass index) greater than or equal to 30.
• Subjects currently not on ketogenic diet.
• Women who are pregnant or breast feeding may not participate. Women who plan to become
pregnant during the course of the study, or who are unwilling to use birth control to
prevent pregnancy (including abstinence) may not participate. Females age 10 and over
will be asked to provide a urine sample for a pregnancy test via dipstick. Subjects
will be asked to agree to abstinence or another form of birth control for the duration
of the study.
• Allergy/sensitivity to C7
• Previous use of triheptanoin less than 1 month prior to study initiation.
• Treatment with medium chain triglycerides in the last 24 hours.
• Subjects exhibiting signs of dementia, or diagnosed with any degenerative brain
disorder (such as Alzheimer's disease) that would confound assessment of cognitive
changes, in the opinion of the investigator.
• Active drug or alcohol use or dependence that, in the opinion of the investigator,
would interfere with adherence to study requirements.
• Inability or unwillingness of subject or legal guardian/representative to give written
informed consent, or assent for children age 10-17,
• Addition of a new antiseizure drug in the previous 3 months.
Autologous Stem Cell Study for Adult TBI (Phase 2b)
The purpose of this study is to determine the effect of intravenous infusion of autologous
bone marrow mononuclear cells (BMMNC) on brain structure and neurocognitive/functional
outcomes after severe traumatic brain (TBI) injury in adults. The primary objective is to
determine if the intravenous infusion of autologous BMMNC after severe TBI results in
structural preservation of global gray matter (GM) volume and white matter (WM) volume and
integrity; as well as select regions of interest in the corpus callosum. THe secondary
objectives are to determine if autologous BMMNC infusion improves functional and
neurocognitive deficits in adults after TBI; reduces the neuroinflammatory response to TBI;
evaluate spleen size and splenic blood flow over time using ultrasound and corresponding
changes in inflammatory cytokines; and infusion related toxicity and long-term follow-up
safety evaluations.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Stephen Figueroa
95844
All
18 Years to 55 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT02525432
STU-2019-1500
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Inclusion Criteria:
1. Adults 18 to 55 years of age on the day of injury,
2. Non-penetrating closed head trauma.
3. Glasgow Coma Score between (GCS) between 3 and 8, (best un-medicated
post-resuscitation score during screening).
4. Ability to obtain legally authorized representative consent for participation and
complete the BMMNC/Sham harvest and cell/placebo infusion within 48 hours of the
initial injury.
5. Ability to speak English or Spanish.
Exclusion Criteria:
1. Known history of:
1. previous brain injury,
2. intellectual deficiency or psychiatric condition likely to invalidate our ability
to assess post-injury changes in cognition or behavior,
3. neurologic impairment and/or deficit,
4. seizure disorder requiring anti-convulsant therapy,
5. recently treated significant infection,
6. renal disease/altered renal function (post-resuscitation serum creatinine > 1.5
mg/dL),
7. chronic hepatic disease or altered liver function (post-resuscitation SGPT > 150
U/L, and/or T. Bilirubin >1.3 mg/dL),
8. cancer,
9. Chemical or ETOH dependency,
10. immunosuppression (admission WBC < 3X103),
11. HIV positive status;
2. Obliteration of perimesencephalic cistern on initial head CT/MRI suggesting prolonged
hypoxic ischemic insult;
3. Initial hospital ICP > 40 mm Hg;
4. Hemodynamic instability at the time of screening defined as SBP < 90mmHg, ongoing
fluid resuscitation and/or requirement for inotropic support to maintain MAP at or
above normals for age •does not include CPP based inotropic support;
5. Uncorrectable coagulopathy at the time of screening;
6. Unstable pelvic fractures that in the P.I.'s opinion would preclude the bone marrow /
sham harvest;
7. Pulmonary contusions defined as a chest x-ray with non-anatomic opacification and
PaO2:FiO2 ratio < 250 associated with the mechanism of injury;
8. Greater than AAST Grade III solid or hollow visceral injury of the abdomen and/or
pelvis diagnosed by CT or other imaging;
9. Spinal cord injury diagnosed by CT or MR imaging or by clinical findings;
10. Persistent hypoxia defined as SaO2 < 94% for > 30 minutes occurring at any time from
hospital admission to time of consent;
11. Positive pregnancy test (if applicable);
12. Concurrent participation in an interventional drug/device research study;
13. Unwillingness to return for follow-up visits;
14. Contraindications to MRI.
Eflornithine (DFMO) and Etoposide for Relapsed/Refractory Neuroblastoma
Difluoromethylornithine (DFMO) will be used in an open label, multicenter, study in
combination with etoposide for subjects with relapsed/refractory neuroblastoma.
• All patients must have a pathologically confirmed diagnosis of neuroblastoma, ≤ 30.99
years of age with history of relapsed/refractory neuroblastoma.
• All patients must have completed upfront therapy with at least 4 cycles of aggressive
multi-drug chemotherapy.
• Specific Criteria by Arm:
Arms 1 and 2:
Subjects with no active disease:
i. No evidence of residual disease by CT/MRI and MIBG scan (or PET for patients who have a
history of MIBG non-avid disease).
o Note: Patients with residual masses detected by CT/MRI may be considered in CR if their
MIBG is negative or if MIBG positive and evaluated by PET and found to have negative PET
scans; biopsy confirmation may be considered if there is still reasonable concern for
persistent disease but is not required.
ii. No evidence of disease metastatic to bone marrow.
Arm 3:
Measurable or evaluable disease, including at least one of the following:
Measurable tumor by CT or MRI; or a positive MIBG and PET; or positive bone marrow
biopsy/aspirate in at least one site.
• Timing from prior therapy: Enrollment (first dose of DFMO) no later than 60 days from
last dose of the most recent therapy.
• Subjects must have fully recovered from the acute toxic effects of all prior anti-
cancer chemotherapy and be within the following timelines:
1. Myelosuppressive chemotherapy: Must not have received within 2 weeks of
enrollment onto this study (6 weeks if prior nitrosourea).
2. Hematopoietic growth factors: At least 5 days since the completion of therapy
with a growth factor.
3. Biologic (anti-neoplastic agent): At least 7 days since the completion of therapy
with a biologic agent. 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. The duration of this interval must be
discussed with the Study Chair.
4. Immunotherapy: At least 6 weeks since the completion of any type of
immunotherapy, e.g. tumor vaccines, CAR-T cells.
5. Anti-GD2 Monoclonal antibodies: At least 2 weeks must have elapsed since prior
treatment with a monoclonal antibody.
6. XRT: At least 14 days since the last treatment except for radiation delivered
with palliative intent to a non-target site.
7. Stem Cell Transplant:
1. Allogeneic: No evidence of active graft vs. host disease
2. Allo/Auto: ≥ 2 months must have elapsed since transplant.
8. MIBG Therapy: At least 8 weeks since treatment with MIBG therapy
• Subjects must have a Lansky or Karnofsky Performance Scale score of 60% or higher.
• Life expectancy > 2 months
• All clinical and laboratory studies for organ functions to determine eligibility must
be performed within 7 days prior to first dose of study drug unless otherwise
indicated below.
• Subjects must have adequate organ functions at the time of registration:
• Hematological: Total absolute neutrophil count ANC ≥750/μL
• Liver: Subjects must have adequate liver function as defined by AST and ALT <5x
upper limit of normal (Normal=45), Bilirubin <1.5x upper limit normal
(Normal=1.0). Normal PT, PTT, fibrinogen.
• Renal: Adequate renal function defined as (perform one of the following):
Creatinine clearance or radioisotope GFR 70 mL/min/1.73 m2 or greater or a serum
creatinine based on age/gender
• Females of childbearing potential must have a negative pregnancy test. Patients of
childbearing potential must agree to use an effective birth control method. Female
patients who are lactating must agree to stop breast-feeding.
• Written informed consent in accordance with institutional and FDA guidelines must be
obtained from all subjects (or patients' legal representative).
Exclusion Criteria:
• BSA of <0.25 m2.
• Subjects that received DFMO at a dose higher than 1000mg/m2 BID prior to this study
are not eligible.
• Subjects that received a dose of DFMO in combination with etoposide are not eligible.
• Investigational Drugs: Subjects who are currently receiving another investigational
drug are excluded from participation.
• Anti-cancer Agents: Subjects who are currently receiving other anticancer agents are
not eligible. Subjects must have fully recovered from hematological and bone marrow
suppression effects of prior chemotherapy.
• Infection: Subjects who have an uncontrolled infection are not eligible until the
infection is judged to be well controlled in the opinion of the investigator.
• Subjects who, in the opinion of the investigator, may not be able to comply with the
safety monitoring requirements of the study, or in whom compliance is likely to be
suboptimal, should be excluded.
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
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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
Study to Compare the Efficacy and Safety of NT 201 (Botulinum Toxin) With Placebo for the Treatment of Lower Limb Spasticity Caused by Stroke or Traumatic Brain Injury (PATTERN)
The purpose of this study is to determine whether a single treatment with administration of
400 Units NT 201 (botulinum toxin) is superior to placebo (no medicine) for the treatment of
lower limb spasticity caused by stroke or traumatic brain injury (Main Period). Participants
will be assigned to the treatment groups by chance and neither the participants nor the
research staff who interact with them will know the allocation.
The following 4 to 5 treatment cycles will investigate the safety and tolerability of
treatment with NT 201 (botulinum toxin) when administered in doses between 400 and 800 Units
(Open Label Extension Period). All participants will receive the treatment and the dose will
depend on whether only lower limb spasticity or combined upper and lower limb spasticity are
treated.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Fatma Gul
12837
All
18 Years to 85 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT03992404
STU-2019-0894
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Inclusion Criteria:
• Female or male subject ≥ 18 years and ≤ 85 years at screening
• Diagnosis of lower limb spasticity with or without upper limb spasticity of the same
body side caused by stroke or traumatic brain injury
• Disabling ankle flexor spasticity presenting as pes equinus or pes equinovarus
• Modified Ashworth Scale-Bohannon [MAS] score of 2 or 3 points in the ankle plantar
flexor of the target lower limb (supine position, knee extended)
• Minimum passive range of motion in ankle of the target lower limb (supine position,
knee extended): 10°dorsiflexion and 20°plantarflexion
• At least 4 months since last botulinum neurotoxin [BoNT] injection for treatment of
spasticity or any other condition
• For subjects receiving anticoagulation therapy, the investigator confirms and
documents that the subject has an:
• Activated partial thromboplastin time [aPTT] ≤ 80 seconds (subjects on dabigatran or
other direct thrombin inhibitors) or
• International normalized ratio [INR] value of ≤ 2.5 (subjects on coumarins or other
anticoagulants monitored by INR)
Exclusion Criteria:
• Generalized disorders of muscle activity (e.g. myasthenia gravis, Lambert Eaton
syndrome, amyotrophic lateral sclerosis) or any other significant peripheral
neuromuscular dysfunction which might interfere with the study
• Bilateral lower limb paresis/paralysis/spasticity or tetraparesis/paralysis/spasticity
• Body weight < 50 kg
• Severe atrophy of the target limb muscles
• Previous, ongoing or planned treatments of spasticity with intrathecal baclofen
• Previous, ongoing, or planned treatments of spasticity in the target lower limb with
any of the following procedures: Surgical Intervention; Alcohol or phenol block;
Muscle afferent block
• Physiotherapy or use of orthoses or splints at the target limb initiated less than 4
weeks before screening or expected to change during the double blind phase of the
study
• Current or planned treatment with parenterally administered drugs that interfere with
neuromuscular transmission (e.g. intrathecal baclofen, tubocurarine type muscle
relaxants used in anesthesia), or local anesthetics in the treated region within 2
weeks prior to screening
• Infection or inflammation at the injection sites
• Subjects with presence or history of aspiration pneumonia, recurrent lower respiratory
tract infections, or compromised respiratory function as per investigator's clinical
judgment
• Pregnancy (as verified by a positive pregnancy test) or breast feeding
Drug: NT 201, Drug: Placebo
Lower Limb or Combined Lower Limb and Upper Limb Spasticity Due to Stroke or Traumatic Brain Injury
UT Southwestern; Parkland Health & Hospital System
A Study Testing the Effect of Immunotherapy (Ipilimumab and Nivolumab) in Patients With Recurrent Glioblastoma With Elevated Mutational Burden
This phase II trial studies the effect of immunotherapy drugs (ipilimumab and nivolumab) in
treating patients with glioblastoma that has come back (recurrent) and carries a high number
of mutations. Cancer is caused by changes (mutations) to genes that control the way cells
function. Tumors with high number of mutations may respond well to immunotherapy.
Immunotherapy with monoclonal antibodies such as ipilimumab and nivolumab may help the body's
immune system attack the cancer and may interfere with the ability of tumor cells to grow and
spread. Giving ipilimumab and nivolumab may lower the chance of recurrent glioblastoma with
high number of mutations from growing or spreading compared to usual care (surgery or
chemotherapy).
• PRE-REGISTRATION:
• Histologically confirmed glioblastoma (World Health Organization [WHO] grade IV)
presenting at first or second recurrence including secondary glioblastoma
• Presence of measurable disease, as defined by a bidimensionally measurable lesion on
magnetic resonance imaging (MRI) with a minimum diameter of 10 mm in both dimensions,
prior to resection or biopsy of recurrent tumor
• Tissue available from surgical resection or biopsy of recurrent tumor =< 28 days prior
to pre-registration, or planned surgery or biopsy of recurrent tumor =< 28 days after
pre-registration
• Does not require > 4 mg dexamethasone beyond the perioperative period defined as the
time =< 2 weeks after surgical procedure
• No prior treatment with laser ablation at the time of recurrent tumor tissue sampling.
Patients who have previously undergone laser ablation >= 4 months prior to recurrent
tumor tissue sampling can be included
• Age >= 18 years
• Eastern Cooperative Oncology Group (ECOG) performance status =< 2
• Able to undergo brain MRI with contrast
• Absolute neutrophil count >= 1500/mm^3
• Platelet count >= 100,000/mm^3
• Total bilirubin =< 1.5 x upper limit of normal (ULN)
• If Gilbert syndrome, then total bilirubin =< 3 x ULN
• Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) =< 2.0 x ULN
• Creatinine =< 1.5 x ULN OR creatinine clearance (CrCl) >= 50 mL/min (if using the
Cockcroft-Gault formula)
• History of active malignancy (outside of the patient's glioblastoma) that has required
treatment within the previous 2 years. Participant with prior history of in situ
cancer or basal or squamous cell skin cancer are eligible
• REGISTRATION ELIGIBILITY CRITERIA: Tissue obtained from biopsy or resection at first
or second recurrence exhibits TMB >= 10 on FoundationOne CDx testing
Exclusion Criteria:
• No active autoimmune disease or history of autoimmune disease
• These include but are not limited to patients with a history of immune related
neurologic disease, multiple sclerosis, autoimmune (demyelinating) neuropathy,
Guillain-Barre syndrome, myasthenia gravis; systemic autoimmune disease such as
systemic lupus erythematosus (SLE), connective tissue diseases, scleroderma,
inflammatory bowel disease (IBD), Crohn's, ulcerative colitis, hepatitis; and
patients with a history of toxic epidermal necrolysis (TEN), Stevens-Johnson
syndrome, or phospholipid syndrome should be excluded because of the risk of
recurrence or exacerbation of disease
• Patients with vitiligo, endocrine deficiencies including thyroiditis managed with
replacement hormones including physiologic corticosteroids are eligible. Patients
with rheumatoid arthritis and other arthropathies, Sjogren's syndrome and
psoriasis controlled with topical medication and patients with positive serology,
such as antinuclear antibodies (ANA), anti-thyroid antibodies should be evaluated
for the presence of target organ involvement and potential need for systemic
treatment but should otherwise be eligible
• No prior treatment with checkpoint blockade therapies (anti-CTLA4, anti-PD1/PD-L1) or
bevacizumab
Biological: Ipilimumab, Biological: Nivolumab
Recurrent Glioblastoma, Secondary Glioblastoma
UT Southwestern; Parkland Health & Hospital System
A Phase 2a Study of TPN-101 in Patients With Progressive Supranuclear Palsy (PSP)
This is a Phase 2a study to assess the safety and tolerability of TPN-101 patients with PSP.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Padraig O'Suilleabhain
35895
All
41 Years to 86 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT04993768
STU-2021-0594
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Inclusion Criteria:
1. Clinical diagnosis of probable progressive supranuclear palsy (PSP)
2. Presence of PSP symptoms for less than 5 years
3. Has a reliable caregiver/informant to accompany the patient to all study visits.
4. Score ≥ 18 on the Mini Mental State Exam (MMSE) at Screening
5. Patient must reside outside a skilled nursing facility or dementia care facility at
the time of Screening, and admission to such a facility must not be planned. Residence
in an assisted living facility is allowed
Exclusion Criteria:
Patients must not meet any of the following criteria:
1. Presence of other significant neurological or psychiatric disorders
2. History of clinically significant brain abnormality
3. Presence of cerebellar ataxia, choreoathetosis, early symptomatic autonomic
dysfunction, or moderate to severe resting tremor, responsive to levodopa
4. Known history of serum or plasma progranulin level less than one standard deviation
below the normal patient mean
5. Known presence of disease-associated mutation in TARDBP, GRN, CHMPB2, or VCP genes; or
any other frontotemporal lobar degeneration causative genes not associated with
underlying tau pathology
6. History of clinically significant hematological, endocrine, cardiovascular, renal,
hepatic, or gastrointestinal disease
Response and Biology-Based Risk Factor-Guided Therapy in Treating Younger Patients With Non-high Risk Neuroblastoma
This phase III trial studies how well response and biology-based risk factor-guided therapy
works in treating younger patients with non-high risk neuroblastoma. Sometimes a tumor may
not need treatment until it progresses. In this case, observation may be sufficient.
Measuring biomarkers in tumor cells may help plan when effective treatment is necessary and
what the best treatment is. Response and biology-based risk factor-guided therapy may be
effective in treating patients with non-high risk neuroblastoma and may help to avoid some of
the risks and side effects related to standard treatment.
• Patients must be:
• < 12 months (< 365 days) of age at diagnosis with INRG stage L1; or
• < 18 months (< 547 days) of age at diagnosis with INRG stage L2 or stage Ms
neuroblastoma/ganglioneuroblastoma
• Enrollment on ANBL00B1 or APEC14B1 is required for all newly diagnosed patients
• Patients must have newly diagnosed v-myc avian myelocytomatosis viral oncogene
neuroblastoma derived homolog (MYCN) non-amplified neuroblastoma (International
Classification of Diseases for Oncology [ICD-O] morphology 9500/3) or MYCN
non-amplified ganglioneuroblastoma verified by histology
• Patients must meet the specified criteria for one of the treatment groups defined
below; genomic features include MYCN gene amplification, segmental chromosome
aberrations (somatic copy number loss at 1p, 3p, 4p, or 11q or somatic copy number
gain at 1q, 2p, or 17q) and deoxyribonucleic acid (DNA) index
• "Favorable" genomic features are defined by one or more whole-chromosome gains or
hyperdiploid tumor (DNA index > 1) in the absence of segmental chromosome
aberrations as defined above
• "Unfavorable" genomic features are defined by the presence of any segmental
chromosome aberration (somatic copy number loss at 1p, 3p, 4p, or 11q or somatic
copy number gain at 1q, 2p, or 17q) or diploid tumor (DNA index = 1); this
includes copy neutral loss of heterozygosity (LOH)
• Only patients with MYCN non-amplified tumors are eligible for this study
• Group A: patients < 12 months (< 365 days) of age with newly diagnosed INRG stage L1
neuroblastoma/ganglioneuroblastoma who meet the following criteria:
• Greatest tumor diameter < 5 cm of adrenal or non-adrenal origin
• Patients with non-adrenal primaries are eligible, but must have positive uptake
on metaiodobenzylguanidine (MIBG) scan or elevated catecholamine metabolites
(urine or serum) to support the diagnosis of neuroblastoma
• No prior tumor resection or biopsy
• Group A will be further split into two subsets, which are mutually exclusive, for
statistical purposes
• Group A1:
• > 6 months and < 12 months of age with an adrenal primary tumor < 5 cm in
greatest diameter OR
• Patients less than 6 months of age with an adrenal primary tumor > 3.1 and <
5 cm in greatest diameter OR
• < 12 months of age with a non-adrenal primary site < 5 cm in greatest
diameter
• Group A2: =< 6 months of age with an adrenal primary site and tumor =< 3.1 cm in
greatest diameter.
• Group B: patients < 18 months (< 547 days) of age with newly diagnosed INRG stage L2
neuroblastoma/ganglioneuroblastoma who meet the following criteria:
• No life threatening symptoms or no impending neurologic or other organ function
compromise (e.g. epidural or intraspinal tumors with existing or impending
neurologic impairment, periorbital or calvarial-based lesions with existing or
impending cranial nerve impairment, anatomic or mechanical compromise of critical
organ function by tumor [abdominal compartment syndrome, urinary obstruction,
etc.]); horner syndrome is not considered neurologic compromise
• No prior tumor resection, tumor biopsy ONLY
• Only patients with both favorable histology and favorable genomic features will
remain on study as part of Group B; the institution will be notified of
histologic and genomic results within 3 weeks of specimen submission on ANBL00B1
or APEC14B1
• Group C: patients < 18 months (< 547 days) of age with newly diagnosed INRG stage Ms
neuroblastoma/ganglioneuroblastoma
• No prior radiotherapy or chemotherapy, with the exception of dexamethasone, which is
allowed
• 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
Exclusion Criteria:
• Patients with MYCN amplified tumors are not eligible
• Group B and C patients who do not enroll on ANBL1232 within 4 weeks of definitive
diagnostic procedure
• Group A and C patients, not required to undergo tumor biopsy, who do not enroll on
ANBL1232 within 4 weeks of confirmatory imaging study
Computer Training Program for Younger Patients With a Brain Tumor Who Underwent Radiation Therapy
This randomized clinical trial studies how well an adaptive computerized cognitive training
program works compared to a non-adaptive computerized cognitive training program in treating
younger patients with brain tumor who underwent radiation therapy. Providing a computer
training program may improve the well-being and quality of life of patients with cognitive
(physical and mental) function difficulties caused by radiation therapy to the brain.
• Patient must be newly diagnosed or relapsed/progressed with a brain tumor that has not
previously been treated with CRT
• Note: COG therapeutic study participation is not required for ACCL10P1 enrollment
• Patient enrollment must occur within 4 calendar months following completion of CRT
• Reminder: after patient enrollment, baseline testing followed by randomization
must occur within 2-4 months after completion of CRT
• The patient must have an identified caregiver who is willing and able to oversee the
training practice during the intervention period (ie, for 5-9 weeks starting
approximately 3 months after completion of CRT)
• The patient must have access to a telephone and phone number where they can be reached
• The patient and caregiver must have reading, speaking and listening comprehension of
English
• All patients and/or their parents or legal guardians must sign a written informed
consent (patient assent is also recommended when applicable according to each
institution's policy)
• All institutional, Food and Drug Administration (FDA), and National Cancer Institute
(NCI) requirements for human studies must be met
Exclusion Criteria:
• Patients with pontine glioma are not eligible
• Patients with an estimated survival of less than one year are not eligible
• Patients with a history of traumatic brain injury prior to tumor diagnosis are not
eligible
• Patients with a motor, visual, or auditory handicap that prevents computer use (e.g.,
unresolved posterior fossa syndrome) are not eligible to participate in this trial
• Patients with full-scale intelligence quotient (IQ) < 70 per previous testing OR
existing diagnosis of/educational classification as a student with an intellectual
disability are not eligible
Procedure: Cognitive Assessment, Other: Computer-Assisted Cognitive Training, Procedure: Psychosocial Assessment and Care, Other: Quality-of-Life Assessment, Other: Questionnaire Administration
Brain Neoplasm, Recurrent Brain Neoplasm, Brain and Nervous System
A Study to Compare the Long-term Outcomes After Two Different Anaesthetics (TREX)
There is considerable evidence that most general anaesthetics modulate brain development in
animal studies. The impact is greater with longer durations of exposure and in younger
animals. There is great controversy over whether or not these animal data are relevant to
human clinical scenarios.
The changes seen in preclinical studies are greatest with GABA agonists and NMDA antagonists
such as volatile anaesthetics (eg sevoflurane), propofol, midazolam, ketamine, and nitrous
oxide. There is less evidence for an effect with opioid (such as remifentanil) or with alpha
2 agonists (such as dexmedetomidine).
Some, but not all, human cohort studies show an association between exposure to anaesthesia
in infancy or early childhood and later changes in cognitive tests, school performance or
risk of developing neurodevelopmental disorders. The evidence is weak due to possible
confounding.
A recent well designed cohort study (the PANDA study) comparing young children that had
hernia repair to their siblings found no evidence for a difference in a range of detailed
neuropsychological tests. In that study most children were exposed to up to two hours of
anaesthesia. The only trial (the GAS trial) has compared children having hernia repair under
regional or general anesthesia and has found no evidence for a difference in neurodevelopment
when tested at two years of age. The GAS and PANDA studies confirm the animal data that short
exposure is unlikely to cause any neurodevelopmental impact.
The impact of longer exposures is still unknown. In humans the strongest evidence for an
association between surgery and poor neurodevelopmental outcome is in infants having major
surgery. However, this is also the group where confounding is most likely.
The aim of our study is to see if a new combination of anaesthetic drugs results in a better
long-term developmental outcome than the current standard of care for children having
anaesthesia expected to last 2 hours or longer.
Children will be randomised to receive either a low dose
sevoflurane/remifentanil/dexmedetomidine or standard dose sevoflurane anaesthetic.
They will receive a neurodevelopmental assessment at 3 years of age to assess global
cognitive function.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Peter Szmuk
80418
All
up to 2 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT03089905
STU 052017-065
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Inclusion Criteria:
• Younger than 2 years (chronological age)
• Scheduled for anaesthesia that is expected to last at least 2 hours (and/or total
operating room time is scheduled to be at least 2.5 hours)
• Has a legally acceptable representative capable of understanding the informed consent
document and providing consent on the participant's behalf.
Exclusion Criteria:
• Known neurologic, chromosomal or congenital anomaly which is likely to be associated
with poor neurobehavioural outcome
• Existing diagnosis of behavioural or neurodevelopmental disability
• Prematurity (defined as < 36 weeks gestational age at birth)
• Birth weight less than 2 kg.
• Congenital cardiac disease requiring surgery
• Intracranial neurosurgery and intracranial craniofacial surgery (isolated cleft lip is
not an exclusion)
• Previous cumulative exposure to general anaesthesia exceeding 2 hours
• Planned future cumulative exposure to anaesthesia exceeding 2 hours before the age of
3 years.
• Any specific contra-indication to any aspect of the protocol
• Previous adverse reaction to any anaesthetic
• Circumstances likely to make long term follow-up impossible
• Living in a household where the primary language spoken at home is not a language in
which we can administer the Wechsler Preschool and Primary School Intelligence Scale
• Planned postoperative sedation with any agent except opioids (e.g. benzodiazepines,
dexmedetomidine, ketamine, barbiturates, propofol, clonidine, chloral hydrate, and
other non-opioid sedatives). For example if such sedation is planned for
post-operative ventilation
Primary Objective:
Long-term safety and pharmacokinetics (PK) of neoGAA
Secondary Objective:
Long-term effect of neo-GAA on pharmacodynamic and exploratory efficacy variables
Call 214-648-5005 studyfinder@utsouthwestern.edu
Jaya Trivedi
46764
All
Not specified
Phase 2/Phase 3
This study is NOT accepting healthy volunteers
NCT02032524
STU 012014-036
Show full eligibility criteria
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Inclusion criteria:
Patients with Pompe disease who previously completed a neoGAA study. The patient and/or
their parent/legal guardian is willing and able to provide signed informed consent, and
the patient, if <18 years of age, is willing to provide assent if deemed able to do so.
The patient (and patient's legal guardian if patient is <18 years of age) must have the
ability to comply with the clinical protocol.
The patient, if female and of childbearing potential, must have a negative pregnancy test
[urine beta-human chorionic gonadotropin] at baseline.
Exclusion criteria:
The patient is concurrently participating in another clinical study using investigational
treatment.
The patient, in the opinion of the Investigator, is unable to adhere to the requirements
of the study.
The patient has clinically significant organic disease (with the exception of symptoms
relating to Pompe disease), including clinically significant cardiovascular, hepatic,
pulmonary, neurologic, or renal disease, or other medical condition, serious intercurrent
illness, or extenuating circumstance that, in the opinion of the Investigator, precludes
participation in the study or potentially decreases survival.
The above information is not intended to contain all considerations relevant to a
patient's potential participation in a clinical trial.
Difluoromethylornithine (DFMO) will be used in an open label, single agent, multicenter,
study for patients with neuroblastoma in remission. In this study subjects will receive 730
Days of oral difluoromethylornithine (DFMO) at a dose of 750 mg/m2 ± 250 mg/m2 BID (strata 1,
2, 3, and 4) OR 2500 mg/m2 BID (stratum 1B) on each day of study. This study will focus on
the use of DFMO in high risk neuroblastoma patients that are in remission as a strategy to
prevent recurrence.
• All patients must have a pathologically confirmed diagnosis of neuroblastoma, < 30.99
years of age and classified as high risk at the time of diagnosis. Exception: patients
who are initially diagnosed as non-high-risk neuroblastoma, but later converted
(and/or relapsed) to high risk neuroblastoma are also eligible.
• All patients must be in complete remission (CR):
1. No evidence of residual disease on scan
2. No evidence of disease metastatic to bone marrow.
• Specific Criteria by Stratum:
Stratum 1/1B: All patients must have completed standard upfront therapy that replicates
treatment which patients who were enrolled on ANBL0032 received, including:
intensive induction chemotherapy and (if feasible) resection of primary tumor, followed by:
consolidation with high-dose chemotherapy with stem cell transplant and radiotherapy,
followed by: immunotherapy with Ch14.18/IL-2/GM-CSF (dinutuximab) and retinoic acid;.
All subjects on Stratum 1/B must have also met the following criteria:
• A pre-transplant disease status evaluation that met International Neuroblastoma Response
Criteria (INRC) for CR (complete response), VGPR (very good partial response), or PR
(partial response) for primary site, soft tissue metastases and bone metastases. Patients
who meet those criteria must also meet the protocol-specified criteria for bone marrow
response prior to transplant as outlined below: No more than 10% tumor involvement (based
on total nucleated cellular content) seen on any specimen from a bilateral bone marrow
aspirate/biopsy.
Stratum 2: Neuroblastoma that is in first complete remission following standard upfront
therapy different from that described for Stratum 1.
Stratum 3: Neuroblastoma that failed to have a response of at least PR following induction
chemotherapy and surgical resection of the primary tumor, but that has achieved CR
following additional therapy.
Stratum 4: Patients who have achieved a second or subsequent CR following relapse(s).
• Pre-enrollment tumor survey: Prior to enrollment on this study, a determination of
mandatory disease staging must be performed:
• Tumor imaging studies including
• Bilateral bone marrow aspirates and biopsy
• This disease assessment is required for eligibility and preferably should be done
within 2 weeks prior to enrollment, but must be done within a maximum of 4 weeks
before enrollment.
• Timing from prior therapy:
Stratum 1/1B: Enrollment no later than 60 days after completion of upfront therapy, (last
dose of cis-retinoic acid) with a maximum of 6 cycles of cis-retinoic acid maintenance
therapy.
Stratum 2, 3 and 4: Enrollment no later than 60 days from last dose of the most recent
therapy.
• Patients must have a Lansky or Karnofsky Performance Scale score of > 50% and patients
must have a life expectancy of ≥ 2 months.
• All clinical and laboratory studies for organ functions to determine eligibility must
be performed within 7 days prior to enrollment unless otherwise indicated below.
• Patients must have adequate organ functions at the time of registration:
• Hematological: Total absolute phagocyte count ≥1000/μL
• Liver: Subjects must have adequate liver function
• Renal: Adequate renal function
• Females of childbearing potential must have a negative pregnancy test. Patients of
childbearing potential must agree to use an effective birth control method. Female
patients who are lactating must agree to stop breast-feeding.
• Written informed consent in accordance with institutional and FDA (food and drug
administration) guidelines must be obtained from all subjects (or patients' legal
representative).
Exclusion Criteria:
• BSA (Body Surface Area) of <0.25 m2.
• Investigational Drugs: Subjects who are currently receiving another investigational
drug are excluded from participation.
• Anti-cancer Agents: Subjects who are currently receiving other anticancer agents are
not eligible. Subjects must have fully recovered from hematological and bone marrow
suppression effects of prior chemotherapy.
• Infection: Subjects who have an uncontrolled infection are not eligible until the
infection is judged to be well controlled in the opinion of the investigator.
• Subjects who, in the opinion of the investigator, may not be able to comply with the
safety monitoring requirements of the study, or in whom compliance is likely to be
suboptimal, should be excluded.
Maximum Tolerated Dose, Safety, and Efficacy of Rhenium Nanoliposomes in Recurrent Glioma (ReSPECT)
This is a multi-center, sequential cohort, open-label, volume and dose escalation study of
the safety, tolerability, and distribution of 186RNL given by convection enhanced delivery to
patients with recurrent or progressive malignant glioma after standard surgical, radiation,
and/or chemotherapy treatment. The study uses a modified Fibonacci dose escalation, followed
by an expansion at the maximum tolerated dose (MTD) to determine efficacy. The starting
absorbed dose is 1mCi in a volume of 0.660mL.
1. At least 18 years of age
2. Ability to understand the purposes and risks of the study and has signed a written
informed consent form approved by the investigator's IRB/Ethics Committee
3. Histologically confirmed glioma
4. Progression by Response Assessment in Neuro-Oncology (RANO) criteria following
standard treatment options with known survival benefit (Temozolomide, Radiation, and
Tumor Treating Fields [unless unwilling])
5. Patients who receive treatment with antiepileptic medications must have a two week
history of stable dose of antiepileptic without seizures prior to dosing
6. Patients with corticosteroid requirements to control cerebral edema must be maintained
at a stable or decreasing dose for a minimum of two weeks without progression of
clinical symptoms
7. A volume of enhancing tumor which falls within the treatment field volume being
evaluated in the respective cohort (see 4.1 Design)
8. Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2
9. Life expectancy of at least 2 months
10. Acceptable liver function:
• Bilirubin ≤ 1.5 times upper limit of normal
• AST (SGOT) and ALT (SGPT) ≤ 3.0 times upper limit of normal (ULN);
11. Acceptable renal function:
• Serum creatinine ≤1.5xULN
12. Acceptable hematologic status (without hematologic support):
• ANC ≥1000 cells/uL
• Platelet count ≥100,000/uL
• Hemoglobin ≥9.0 g/dL
13. All women of childbearing potential must have a negative serum pregnancy test and male
and female subjects must agree to use effective means of contraception (surgical
sterilization or the use or barrier contraception with either a condom or diaphragm in
conjunction with spermicidal gel or an IUD) with their partner from entry into the
study through 6 months after the last dose
For part 2:
14. Bevacizumab naïve glioblastoma with no more than 1 recurrence
Exclusion Criteria:
1. The subject has evidence of acute intracranial or intratumoral hemorrhage either by
MRI or computerized tomography (CT) scan. Subjects with resolving hemorrhage changes,
punctate hemorrhage, or hemosiderin are eligible.
2. The subject is unable to undergo MRI scan (eg, has pacemaker).
3. The subject has not recovered to National Cancer Institute (NCI) Common Terminology
Criteria for Adverse Events (CTCAE) v4.0 Grade ≤ 1 from AEs (except alopecia, anemia
and lymphopenia) due to surgery, antineoplastic agents, investigational drugs, or
other medications that were administered prior to study.
4. The subject is pregnant or breast-feeding.
5. The subject has serious intercurrent illness, as determined by the treating physician,
that would compromise either patient safety or study outcomes such as:
• hypertension (two or more blood pressure readings performed at screening of > 150
mmHg systolic or > 100 mmHg diastolic) despite optimal treatment
• Non-healing wound, ulcer, or bone fracture
• Clinically significant cardiac arrhythmias
• Untreated hypothyroidism
• Uncontrolled systemic infection
• Symptomatic congestive heart failure or unstable angina pectoris within 3 months
prior study drug
• Myocardial infarction, stroke, transient ischemic attack within 6 months
• Known active malignancy (other than glioma) except non-melanoma skin cancer or
carcinoma in-situ in the cervix
6. The subject has inherited bleeding diathesis or coagulopathy with the risk of bleeding
7. The subject has received any of the following prior anticancer therapy:
• Non-standard radiation therapy such as brachytherapy, systemic radioisotope
therapy, or intra-operative radiotherapy (IORT) to the target site.
• Radiation therapy within 12 weeks of screening
• Systemic therapy (including investigational agents and small-molecule kinase
inhibitors) or non-cytotoxic hormonal therapy (eg, tamoxifen) within 14 days or 5
half-lives, whichever is shorter, prior first dose of study drug
• Biologic agents (antibodies, immune modulators, vaccines, cytokines) within 21
days prior to first dose of study drug
• Nitrosoureas or mitomycin C within 42 days, or metronomic/protracted low-dose
chemotherapy within 14 days, or other cytotoxic chemotherapy within 28 days,
prior to first dose of study drug
• Prior treatment with carmustine wafers
• Patients who are currently receiving any other investigational agents and/or who
have received an investigational agent in the prior 28 days
8. Multifocal progression or involvement of the leptomeninges
9. Psychiatric illness/social situations that would limit compliance with the study
requirements
10. Infratentorial disease
Safeguarding the Brain of Our Smallest Infants Phase III (SafeBoosC)
the SafeBoosC-III trial investigates the benefit and harms of treatment based on
near-infrared spectroscopy monitoring compared with treatment as usual. The hypothesis is
that treatment based on near-infrared spectroscopy monitoring for extremely preterm infants
during the first 72 hours of life will result in a reduction in severe brain injury or death
at 36 weeks postmenstrual age.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Lina Chalak
35027
All
up to 6 Hours old
N/A
This study is NOT accepting healthy volunteers
NCT03770741
STU-2019-1707
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Inclusion Criteria:
• Infants born with postmenstrual age less than 28 weeks
• Signed informed consent, unless the NICU has chosen to use 'opt-out' or deferred
consent as consent method.
Exclusion Criteria:
• Missing written parental informed consent (if the 'opt-out' method is used for
consent, lack of a record that the clinical staff have explained the trial and the
'opt-out' consent process to parents and/or a record in the infant's clinical file of
parents' decision to opt-out, are exclusion criteria)
• Decision not to conduct full life support
• No possibility to place cerebral NIRS oximeter within six hours after birth
Other: Modify cardio-respiratory support to avoid cerebral hypoxia, Other: Treatment as usual
Brain Oxygen Optimization in Severe TBI, Phase 3 (BOOST3)
BOOST3 is a randomized clinical trial to determine the comparative effectiveness of two
strategies for monitoring and treating patients with traumatic brain injury (TBI) in the
intensive care unit (ICU). The study will determine the safety and efficacy of a strategy
guided by treatment goals based on both intracranial pressure (ICP) and brain tissue oxygen
(PbtO2) as compared to a strategy guided by treatment goals based on ICP monitoring alone.
Both of these alternative strategies are used in standard care. It is unknown if one is more
effective than the other. In both strategies the monitoring and goals help doctors adjust
treatments including the kinds and doses of medications and the amount of intravenous fluids
given, ventilator (breathing machine) settings, need for blood transfusions, and other
medical care. The results of this study will help doctors discover if one of these methods is
more safe and effective.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Ahamed Idris
58880
All
14 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT03754114
STU-2019-0560
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Inclusion Criteria:
• Non-penetrating traumatic brain injury
• Glasgow Coma Scale (GCS) 3-8 measured off paralytics
• Glasgow Coma Scale motor score < 6 if endotracheally intubated
• Evidence of intracranial trauma on CT scan
• Able to place intracranial probes and randomize within 6 hours of arrival at enrolling
hospital
• Able to place intracranial probes and randomize within 12 hours from injury
• Age greater than or equal to 14 years
Exclusion Criteria:
• Non-survivable injury
• Bilaterally absent pupillary response in the absence of paralytic medication
• Contraindication to the placement of intracranial probes
• Treatment of brain tissue oxygen values prior to randomization
• Planned use of devices which may unblind treating physicians to brain tissue hypoxia
• Systemic sepsis at screening
• Refractory hypotension
• Refractory systemic hypoxia
• PaO2/FiO2 ratio < 200
• Known pre-existing neurologic disease with confounding residual neurological deficits
• Known inability to perform activities of daily living (ADL) without assistance prior
to injury
• Known active drug or alcohol dependence that, in the opinion of site investigator,
would interfere with physiological response to brain tissue oxygen treatments
• Pregnancy
• Prisoner
• On EFIC Opt-Out list as indicated by a bracelet or medical alert
A Study to Compare the Safety and Efficacy of Dysport® and Botox® in Adults With Upper Limb Spasticity. (DIRECTION)
This study is aiming to demonstrate the non-inferiority of AbobotulinumtoxinA (aboBoNT-A)
versus OnabotulinumtoxinA (onaBoNT-A) as the primary safety endpoint, and the superiority of
aboBoNT-A over onaBoNT-A with respect to duration of response as the key secondary efficacy
endpoint when used at optimal doses according to approved prescribing information of each
product.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Fatma Gul
12837
All
18 Years to 75 Years old
Phase 4
This study is NOT accepting healthy volunteers
NCT04936542
STU-2021-0659
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Inclusion Criteria:
• Participant must be 18 to 75 years of age inclusive, at the time of signing the
informed consent
• 2a. [US/France] Participants with stable Upper Limb Spasticity (ULS) for at least 3
months, in whom treatment of only one upper limb is necessary for the duration of the
study;
• 2b. [Canada] Participants with stable post-stroke ULS for at least 3 months, in whom
treatment of only one upper limb is necessary for the duration of the study
• Participants who are either naïve to Botulinum toxin type A (BoNT-A) for ULS or who
have been previously treated with BoNT-A for ULS;
• Participants with MAS score of at least 2 at elbow, wrist and finger flexors;
• Participants with DAS score of at least 2 on the Principal Target of Treatment (PTT)
(one of four functional domains: dressing, hygiene, limb position and pain);
• Participants who require BoNT-A injection in all of the following muscles: flexor
carpi radialis, flexor carpi ulnaris, flexor digitorum profundus, flexor digitorum
superficialis and biceps brachii;
• Participants for whom injection of a total dose of 900 Units aboBoNT-A or 360 Units
onaBoNT-A is considered by the investigator to be clinically appropriate;
• Participants who have been stable for at least 3 months prior to study entry in terms
of oral antispasticity, anticoagulant and/or anticholinergic medication if treated,
and for at least 1 month prior to study entry in terms of occupational and/or
physiotherapy treatment, if treated, and are considered by the investigator likely to
remain stable for the duration of the study;
Exclusion Criteria:
• Major limitations in the passive range of motion in the paretic upper limb;
• Major neurological impairment (other than limb paresis) that could negatively affect
functional performance;
• Participants clinically requiring injection into any upper limb muscles other than the
five muscles of one arm listed in Section 5.1, or requiring injection into both arms
or any lower limb within the timeframe of the study;
• Hypersensitivity to any BoNT product or excipients;
• Hypersensitivity to cow's milk protein (casein);
• Infection at the proposed injection site(s);
• Known peripheral motor neuropathic diseases, amyotrophic lateral sclerosis or
neuromuscular junction disorders (e.g. myasthenia gravis or Lambert-Eaton syndrome);
• Any medical condition (including dysphagia or breathing difficulties/compromised
respiratory function) that in the opinion of the investigator, might jeopardize the
participant's safety;
• Women who are pregnant or lactating
• Participants treated with BoNT of any type for any indication (e.g. bladder injection,
headache or cosmetic) within the previous 12 weeks or planned/likely to be treated
during the course of the study;
• Prior history of non-responsiveness to BoNT treatment;
• Previous surgery, or administration of alcohol or phenol in the study limb 6 months or
earlier from study enrolment or planned/likely to be treated during the course of the
study;
• Participants treated with intrathecal baclofen, aminoglycosides or other agents
interfering with neuromuscular transmission (e.g. curare-like agents), within the
previous 4 weeks or planned/likely to be treated during the course of the study;
• Participants who received a COVID-19 vaccine injection within 7 days before the first
planned study intervention injection, or planned/likely to be injected within 7 days
after the first planned study intervention injection
• BoNT naïve participants with a history of facial neurogenic disorder (facial
paralysis, polyradiculoneuropathy) (only for France).
A Single Arm Study Evaluating the Efficacy, Safety and Tolerability of Ofatumumab in Patients With Relapsing Multiple Sclerosis (OLIKOS)
A single arm study evaluating the continued efficacy, safety and tolerability of ofatumumab
in patients with relapsing multiple sclerosis who are transitioning from aCD20 mAb therapy
Call 214-648-5005 studyfinder@utsouthwestern.edu
Lauren Tardo
164490
All
18 Years to 60 Years old
Phase 3
This study is also accepting healthy volunteers
NCT04486716
STU-2020-1277
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Inclusion Criteria:
Participants eligible for inclusion in this study must meet all of the following criteria:
1. Written informed consent must be obtained before any assessment is performed.
2. Male or female participants aged 18 to 60 years (inclusive) at screening.
3. Diagnosis of relapsing MS (RMS) according to the 2017 Revised McDonald criteria
(Thompson et al. 2018), including CIS, RRMS or SPMS with disease activity as defined
by (Lublin et al. 2014).
4. Disability status at Screening with an EDSS score of 0 to 5.5 (inclusive).
5. Received at least 2 courses of intravenous aCD20 mAb (loading doses are considered 1
course):
• Participants currently treated with ocrelizumab must have received (meet all three
criteria below):
1. 2 fully infused initial 300 mg ocrelizumab iv infusions 2. At least 1 fully infused 600
mg ocrelizumab iv infusions 6 months (+/- one month) 3. Last fully infused ocrelizumab dose
must have occurred within 4-9 months prior to baseline
•Participants currently treated with rituximab must have received (meet both criteria
below):
1. At least 2 fully infused courses of rituximab 500 mg •1000 mg iv every 6 months (+/-
one month).
1. Initial loading regimens of rituximab i.e. 500 mg •1000 mg on day 1 and on day
15, are allowed but this is consider a single course and must be followed by
additional infusion(s) every 6 months (+/- one month)
2. Last fully infused rituximab dose must have occurred within 4-9 months prior to
baseline.
6. Participants discontinuing aCD20 therapy for reasons including, but not limited to:
physician/participant preference, access to commercial drug (e.g. insurance coverage
issues) or for other logistical reasons (such as geographical relocation, travel, etc.) are
eligible for this study. 7. Neurologically stable within 1 month prior to first study drug
administration.
8. Must be able to use a smart device or have a caregiver that can assist.
Exclusion Criteria:
Participants meeting any of the following criteria are not eligible for inclusion in this
study:
1. Participants that have demonstrated suboptimal response to aCD20 therapy to include:
a. Signs of MRI activity, defined as ≥ 2 active Gd+ T1 lesions, or any new or newly
enlarging T2 lesions, documented within the past 6 months
• If a prior MRI within the last 6 months is not available, then new or newly
enlarging T2 lesions should be considered "not documented" and the patient may
continue screening b. Documented relapse while on stable, previous aCD20
treatment.
• Relapses during the first 3 months of intravenous aCD20 therapy are allowable if
the participant is then relapse-free for the 12 months following the relapse
while on intravenous aCD20 therapy c. Any signs of clinical worsening as measured
by EDSS or any clinical measure documented within the last 6 months
2. Discontinuing aCD20 mAb therapy due to the following treatment- emergent adverse
events:
1. Severe infusion-related reactions (Grade 3 or above)
2. Recurrent infections defined as ≥ 2 severe infections or ≥ 3 respiratory
infections or the need for ≥ 2 courses of antibiotics since starting aCD20
therapy, if the Investigator believes this is related to therapy.
3. Decreased IgG requiring treatment with Intravenous immunoglobulin
3. Participants with primary progressive MS (Polman et al 2011) or SPMS without disease
activity (Lublin et al 2014).
4. Participants meeting criteria for neuromyelitis optica (Wingerchuk et al 2015).
5. Pregnant or nursing (lactating) women
6. Women of child-bearing potential, defined as all women physiologically capable of
becoming pregnant, unless they are using highly effective methods of contraception
during dosing and for at least 6 months after stopping study medication.
7. Participants with active chronic disease (or stable but treated with immune therapy)
of the immune system other than MS (e.g. rheumatoid arthritis, scleroderma, Sjögren's
syndrome, Crohn's disease, ulcerative colitis, etc.) or with immunodeficiency syndrome
(hereditary immune deficiency, drug-induced immune deficiency).
8. Participants with active systemic bacterial, viral or fungal infections, or known to
have acquired immunodeficiency syndrome (AIDS).
9. Participants with neurological symptoms consistent with PML or with confirmed PML.
10. Participants at risk of developing or having reactivation of syphilis or tuberculosis
11. Participants at risk of developing or having reactivation of hepatitis.
12. Have received any live or live-attenuated vaccines (including for varicella-zoster
virus or measles) within 4 weeks prior to first study drug administration. a. There is
presently no contraindication for the use of an inactivated, viral-vector-or mRNA
based Sars-CoV-2 vaccine in patients who are immunocompromised. However, different
Sars-CoV-2 vaccines may have various mechanisms of action and different associated
potential risks. Please review local prescribing information of any specific
Sars-CoV-2 vaccine and comply with local prescribing information requirements for
specific contra-indications and special warnings and precautions for use.
Drug: Ofatumumab
Relapsing Multiple Sclerosis, Brain and Nervous System