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.
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
Comparing Virtual Reality (VR) to Non-VR for Decreasing Preoperative/Procedural Anxiety
The primary objective of the study is to utilize the modified Yale Preoperative Anxiety scale
(mYPAS), a validated preoperative/procedural anxiety score, to measure preoperative anxiety
via distraction in pediatric oncology patients undergoing port access.
The hypothesis is that using Virtual Reality (VR) will objectively decrease anxiety scores
measured by mYPAS by five percent (primary outcome).
The secondary outcome will be the parents or the legally authorized representative (LAR)
subjective reports of anxiety with the use of VR.
The Kind VR device is used in house at Children's Health in the Dallas and Plano campuses.
The VR device used in this study qualifies as exempt from FDA IDE regulations. It is a
non-significant risk, non-invasive, interactive video device the user wears like goggles. The
study carries minimal risks to the subjects and is designed to minimize patient discomfort
from placement or motion sickness. Furthermore, the device has disposable covers for
protection against infection and can be sanitized between uses, once the disposable covers
are removed. Children's Health System of Texas (CHST) and this research group are not
partnering entities with the Kind VR, and the Kind VR device is not being studied. The effect
of virtual reality (VR) on preprocedural anxiety as measured by questionnaires and the
observations of the modified Yale Preoperative Anxiety Scale (mYPAS) is being studied
Most patients coming to the Clinic of Cancer and Blood Disorders (CCBD) are under chronic
care for their ongoing disease and are likely to be coming to the CCBD at least twice in a
6-month period. The CCBD schedule will be reviewed by the researchers for patients age 5-12,
requiring port access at least twice during the next six-month period. Patient families whose
child meets the basic screening criteria, and have no exclusion criteria, will be approached
privately as possible participants in the study. Up to 100 subjects will be enrolled over a
2-year period. Once the subject/parent or LAR agrees to participate, study staff will
randomize the subjects into which standard of care distraction method for anxiety management
they will receive first in this study.
• Any patient of the Children's Medical Center CCBD
• 5-12 years of age
• Patient requiring their port accessed twice or more within a 6 months period
• Ability to understand and the willingness to sign a written informed consent.
Exclusion Criteria
• Subjects younger than 5 and older than 12
• Patients requiring recovery in PICU or sites other than PACU
• If parents or subject is not willing to participate
• Subjects with severe developmental delays and subjects with developmental challenges
preventing them from keeping the VR device on are also excluded
• Patients who will not be in CCBD for port access at least twice in 6 months
Other: Virtual reality
Lymphoma, Sarcoma, Multiple Myeloma, Brain and Nervous System, Eye and Orbit, Anus, Bones and Joints, Breast - Female, Breast - Male, Carcinoid Tumor, Cervix, Colon, Corpus Uteri, Ear, Esophagus, Gall Bladder, Head and Neck, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung/Thoracic, Melanoma, skin, Nose, Ovary, Pancreas, Prostate, Rectum, Stomach, Throat, Thyroid, Urinary Bladder, Uterine (Endometrial), Vulva, Leukemia, Other, Hodgkins Lymphoma, Leukemia, Not Otherwise Specified, Lymphoid Leukemia, Myeloid and Monocytic Leukemia, Non-Hodgkins Lymphoma, Small Intestine, Soft Tissue, Preoperative Anxiety
The iCat2, GAIN (Genomic Assessment Informs Novel Therapy) Consortium Study
This research study is evaluating the use of specialized testing of solid tumors including
sequencing. The process of performing these specialized tests is called tumor profiling. The
tumor profiling may result in identifying changes in genes of the tumor that indicate that a
particular therapy may have activity. This is called an individualized cancer therapy (iCat)
recommendation. The results of the tumor profiling and, if applicable, the iCat
recommendation will be returned.
• Age -- Age ≤ 30 years at time of initial qualifying solid tumor diagnosis
• Diagnosis -- Histologic diagnosis of solid malignancy (excluding brain tumors and
lymphoma) that meets at least one of the following criteria:
• Refractory, defined as tumor progression after initiation of standard first line
therapy without having achieved a prior partial or complete remission OR Biopsy
proven residual disease at the completion of planned standard initial front-line
therapy.
• Recurrent, defined as tumor progression after achieving a prior partial or
complete remission
• Newly diagnosed high risk disease, defined as having an expected event free
survival of < 50% at 2 years.
• Lacks definitive diagnosis or classical genomic findings after histologic review
and standard molecular testing (rare tumor group).
• Examples include (eligibility not limited to these examples):
• Histology typically associated with a fusion in which fusion is not detected.
• Ewing-like sarcoma
• Undifferentiated sarcoma
• Inflammatory myofibroblastic tumor without ALK fusion
• Infantile fibrosarcoma without NTRK fusion
• Specimen Samples
• Sufficient tumor specimen available to meet the minimum requirements for
profiling from diagnosis or progression / recurrence
--- OR
• Surgery / biopsy planned as part of clinical care that is anticipated to yield
sufficient material to meet the minimum requirements for profiling; OR
• Patient has already had molecular profiling and patient has not yet started
matched targeted therapy based on the report .
Exclusion Criteria:
• No Therapy Planned
-- Patients who have declined further anticancer therapy will be excluded.
• Performance Status
-- Patients with Lansky (age < 16 years) or Karnofsky (age ≥16 years) score < 50 will
be excluded.
• Life Expectancy -- Patients with anticipated life expectancy < 3 months will be
excluded.
Genetic: Genetic testing and GAIN report
Sarcoma, Pediatric Solid Tumor, Brain and Nervous System, Other, Eye and Orbit, Anklylosing Spondylitis, Anus, Bones and Joints, Breast - Female, Breast - Male, Carcinoid Tumor, Cervix, Colon, Corpus Uteri, Ear, Esophagus, Gall Bladder, Head and Neck, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung/Thoracic, Melanoma, skin, Nose, Other Digestive Organ, Other Endocrine System, Other Female Genital, Other Male Genital, Other Respiratory and Intrathoracic Organs, Other Skin, Other Urinary, Ovary, Pancreas, Prostate, Rectum, Stomach, Throat, Thyroid, Urinary Bladder, Uterine (Endometrial), Vulva, Heart, Kaposis sarcoma, Small Intestine, Soft Tissue, Ill - Defined Sites
Effects of Hypoglossal Nerve Stimulation on Cognition and Language in Down Syndrome and Obstructive Sleep Apnea
This study is a prospective, single-arm study conducted under a common implant and follow-up
protocol. The objective will be to follow fifty-seven (57) adolescents and young adults
(10-21 years of age), with Down syndrome, moderate to severe sleep apnea, and
post-adenotonsillectomy, for 12 months after undergoing implant of the Inspire Upper Airway
Stimulation (UAS) System. The study is being conducted in order to evaluate objective change
in cognition and expressive language after implant and therapy with the Inspire UAS System.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Ron Mitchell
124198
All
10 Years to 21 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT04801771
STU-2021-0286
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Inclusion Criteria:
• Diagnosis of Down syndrome
• Age 10-21 years
• Prior adenotonsillectomy
• Severe OSA (AHI > 10, AHI < 50, no more than 25% AHI attributable to central events)
based on prior in-lab PSG performed after adenotonsillectomy and within 18 months of
enrollment
• Approval from at least two of the three physician reviewers based upon the results of
a routine drug-induced sleep endoscopy (DISE) having occurred within 12 months of
enrollment
• Subjects must have either tracheotomy or be ineffectively treated with CPAP due to
non-compliance, discomfort, un-desirable side effects, persistent symptoms despite
compliance use, or refusal to use the device
• Children and their parents/guardians must be willing to have stimulation hardware
permanently implanted, and be willing to participate in follow-up visits,
postoperative PSG, and questionnaire completion
• Children's parents/guardians must complete a questionnaire confirming that their child
is capable of communicating feelings of pain or discomfort. They must also confirm
they are able to assess their child for adverse effects related to device implantation
• Children and their parents/guardians must be proficient in English
Exclusion Criteria:
• Body mass index (BMI) above the 95th percentile for subject's age
• Circumferential airway collapse at the level of the velopharynx observed during DISE
• Other medical conditions resulting in medical instability (eg. congestive heart
failure, recent open heart surgery, immunosuppression, or chronic lung disease or
aspiration)
• Presence of another medical condition requiring future magnetic resonance imaging
(MRI) of the chest
• Patients with another implantable device which could interact unintentionally with the
Inspire system
• Any contraindication for general anesthesia
• History of bleeding or clotting disorders and those on blood thinning or NSAID
medications for the week prior to implantation surgery. Subjects will be asked to
refrain from the use of NSAIDS for two weeks after implantation or any revision
surgeries
• Subject is currently taking muscle relaxant medication
• Life expectancy less than 12 months
• Subject's inability to communicate pain or discomfort to their caretaker/parent, based
on parental or investigator assessment
• Nonverbal candidates will be excluded due to an inability to complete testing
procedures including expressive language sampling
• Subjects with a co-occurring diagnosis of autism spectrum disorder
• Subjects that have a positive β-HCG
• Subjects deemed unfit for participation by the investigator for any other reason
Device: Inspire Upper Airway Stimulation (UAS) System
Obstructive Sleep Apnea, Down Syndrome, Ear, Nose, Throat
SPI-1005 for Prevention and Treatment of Aminoglycoside Induced Ototoxicity
The primary objective of this study is to determine the safety and tolerability of SPI-1005
treatment in CF patients with active pulmonary exacerbation that are receiving an IV course
of tobramycin, using histories, physical exams, vital signs (VS), adverse event (AE)
reporting, hematology (CBC) chemistry (Chem-20).
The secondary objectives of this study are to determine the pharmacokinetics of oral SPI-1005
at 200, 400 and 600 mg BID for 21 days. Peak/trough assessments will be determined for
ebselen, its major metabolite and selenium. Clinical assessments of the severity of
sensorineural hearing loss, speech discrimination, vertigo severity, tinnitus severity and
lung function will be made compared between treatment arms and the placebo arm of this study.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Raksha Jain
19733
All
18 Years and over
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT02819856
STU 052017-042
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Inclusion Criteria:
• Cystic fibrosis patients about to receive IV tobramycin for acute pulmonary
exacerbation.
• Voluntarily consent to participate in the study.
• Females of childbearing potential should be using and committed to continue using one
of the following acceptable birth control methods:
• Sexual abstinence (inactivity) for 14 days prior to screening through study
completion; or IUD in place for at least 3 months prior to study through study
completion; or Barrier method (condom or diaphragm) with spermicide for at least 14
days prior to screening through study completion; or Stable hormonal contraceptive for
at least 3 months prior to study through study completion.
• Ability to perform all behavioral tests as indicated.
Exclusion Criteria:
• Current use or within 60 days prior to study enrollment the following IV ototoxic
medications: aminoglycoside antibiotics (gentamicin, tobramycin, amikacin,
streptomycin); platinum-containing chemotherapies (cisplatin, carboplatin,
oxaliplatin); or loop diuretic (furosemide).
• History of idiopathic sensorineural hearing loss, otosclerosis, or vestibular
schwannoma.
• History of middle ear or inner ear surgery.
• Current conductive hearing loss or middle ear effusion.
• Significant cardiovascular, hepatic, renal, hematologic, endocrine, immunologic, or
psychiatric disease.
• History of hypersensitivity or idiosyncratic reaction to compounds related to ebselen.
• Participation in another investigational drug or device study within 30 days prior to
study enrollment.
• Female patients who are pregnant or breastfeeding.
Randomized Controlled Trial of Valganciclovir for Cytomegalovirus Infected Hearing Impaired Infants (ValEAR)
The overall goal of this study is to determine the clinical benefit and safety of antiviral
therapy for asymptomatic congenital cytomegalovirus (cCMV) infected hearing-impaired infants.
We will conduct a multi-center double-blind randomized placebo-controlled trial to determine
whether hearing-impaired infants with asymptomatic cCMV have better hearing and language
outcomes if they receive valganciclovir antiviral treatment. We will also determine the
safety of antiviral valganciclovir therapy for asymptomatic cCMV-infected hearing impaired
infants. This study will be unique in that the cohort enrolled will only include
hearing-impaired infants with asymptomatic cCMV.
Primary Objective: To determine if treatment of cCMV-infected hearing impaired infants with
isolated hearing loss with the antiviral drug valganciclovir reduces the mean slope of total
hearing thresholds over the 20 months after randomization compared to untreated cCMV-infected
infants with isolated hearing loss.
Main Secondary Objectives:
1. To determine if valganciclovir treatment improves the following outcomes when compared
to the control group:
1. The slope of best ear hearing thresholds over the 20 months after randomization.
2. The MacArthur-Bates Communicative Development Inventory (CDI) percentile score for
words produced at 20 months of age.
2. To evaluate safety measures based on all grade 3 or greater new adverse events
designated by the NIAID Division of AIDS (DAIDS) toxicity tables.
Call 214-648-5005 studyfinder@utsouthwestern.edu
Kenneth Lee
93887
All
1 Month to 12 Months old
Phase 2
This study is NOT accepting healthy volunteers
NCT03107871
STU-2019-1329
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Inclusion Criteria:
• Age greater than or equal to 1 month and less than or equal to 12 months at the time
of randomization; AND
• Positive congenital CMV by urine culture or polymerase chain reaction test(PCR), OR
saliva culture or PCR followed by confirmatory urine PCR by 21 days of age, OR urine
culture or PCR after 21 days of age followed by newborn dry blood spot PCR; AND
• Confirmed sensorineural hearing loss (SNHL) by auditory brainstem response (ABR)
testing. For ABR assessments, hearing loss is defined as levels greater than 25 dB
normal hearing levels (NHL) at 1, 2, or 4 kHz in one or both ears.
Exclusion Criteria:
• Imminent demise; OR
• Known hypersensitivity reaction to valganciclovir, ganciclovir, or any components of
the investigational product formulation; OR
• ALT (Alanine Aminotransferase) five times baseline U/L, hepatomegaly, or significant
gastrointestinal disorders (e.g., eosinophilic esophagitis, ulcerative colitis); OR
• Absolute neutrophil count (ANC) less than 500 cells/mm^3, Hemoglobin less than 8 g/dL,
or platelets less than 50,000/mm^3, splenomegaly, or significant hematologic disorders
(e.g., hemophilia, leukemia, sickle cell anemia); OR
• Creatinine clearance less than 60 mL/min/1.73m^2 or significant renal disorders (e.g.,
nephrotic syndrome); OR
• Receiving other antiviral medications or immune globulin therapy; OR
• Receiving other investigational drugs; OR
• Breast feeding from a mother receiving antiviral or immunosuppressive medication; OR
• Known HIV positive mother (risk of immunosuppression); OR
• Subject is currently using list of prohibited medication specified by the package
insert; OR
• Other known cause contributing to SNHL (e.g., meningitis, aminoglycoside ototoxicity);
OR
• Bilateral profound SNHL or auditory neuropathy spectrum disorder; OR
• Existing conductive hearing loss or mixed permanent hearing loss is present; OR
• Evidence of intracranial calcification; OR
• Evidence of hydrocephalus; OR
• Microcephaly; OR
• Presence of petechiae; OR
• Intrauterine growth retardation; OR
• Chorioretinitis, optic atrophy or pale optic nerves; OR
• Parent or guardian unable to speak English or Spanish; OR
• Subject exposed to a language other than English or Spanish a majority of the time; OR
• Subject unable to complete hearing assessments or parent/guardian unable to complete
communication questionnaires; OR
• < 32 weeks gestational age at birth; OR
• Weight at the time of birth < 1800 g.
Stereotactic Radiosurgery (SRS) for Brain Metastasis (SRS)
SRS dose escalation for brain metastases in radiation-naïve patients will establish true
tolerable doses, which may exceed the current standard doses. This may lead to an improvement
in local control, patient survival, and/or quality-of life.
Inclusion Criteria
1. Biopsy-proven non-hematopoietic malignancy, except for small cell lung cancer, germ
cell cancer, or unknown primary tumor.
2. Radiographic evidence by MRI (or by CT scan with CT contrast if ineligible or
intolerant of MRI) of brain metastasis. (If patient is unable to tolerate MRI
contrast, an MRI without contrast is acceptable if lesions are visible)
3. All brain metastases must be outside the brain stem (midbrain, pons and medulla).
4. Patient must have 10 or less brain metastases.
5. The maximum diameter of any lesion must be less than or equal to 3.0 cm.
6. Previous treatment with surgery, radiation, chemotherapy, immunotherapy or any
targeted agents are allowed provided that:
• Radiation was not to the brain.
• Surgery to the brain was > 7 days prior to SRS and there remains at least one
additional brain metastasis that can be targeted with SRS
7. Age ≥ 18 years.
8. ECOG Performance Score of 2 or better/Karnofsky Performance Status score of 50-60 or
better.
9. All men, as well as women of child-bearing potential must agree to use adequate
contraception (hormonal or barrier method of birth control; abstinence) prior to study
entry and for the duration of study participation. Should a woman become pregnant or
suspect she is pregnant while participating in this study, she should inform her
treating physician immediately.
A female of child-bearing potential is any woman (regardless of sexual orientation,
marital status, having undergone a tubal ligation, or remaining celibate by choice)
who meets the following criteria:
• Has not undergone a hysterectomy or bilateral oophorectomy; or
• Has not been naturally postmenopausal for at least 12 consecutive months (i.e.,
has had menses at any time in the preceding 12 consecutive months).
Medically acceptable birth control (contraceptives) includes:
• Approved hormonal contraceptives (such as birth control pills, patch, or ring:
Depo-Provera, Implanon), or
• Barrier methods (such as a condom or diaphragm) used with a spermicide (a
substance that kills sperm)
10. Ability to understand and the willingness to sign a written informed consent.
Exclusion Criteria
1. Patients had craniotomy and surgery to the brain within 7 days from the date of SRS.
2. Patients with leptomeningeal metastasis.
NOTE: For the purposes of exclusion, LMD is a clinical diagnosis, defined as positive
CSF cytology and/or equivocal radiologic or clinical evidence of leptomeningeal
involvement. Patients with leptomeningeal symptoms in the setting of leptomeningeal
enhancement by imaging (MRI) would be considered to have LMD even in the absence of
positive CSF cytology, unless a parenchymal lesion can adequately explain the
neurologic symptoms and/or signs. In contrast, an asymptomatic or minimally
symptomatic patient with mild or nonspecific leptomeningeal enhancement (MRI) would
not be considered to have LMD. In that patient, CSF sampling is not required to
formally exclude LMD, but can be performed at the investigator's discretion based on
level of clinical suspicion.
3. Patients with a contraindication to both MRI (with or without contrast) and CT scan
(with contrast)
4. Patients with life expectancy < 3 months.
5. Uncontrolled intercurrent illness including, but not limited to, ongoing or active
infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac
arrhythmia, or psychiatric illness/social situations that, in the opinion of the
investigator, would limit compliance with study requirements.
6. Subjects must not be pregnant or nursing at the time of SRS treatment due to the
potential for congenital abnormalities and the potential of this regimen to harm
nursing infants.
Radiation: Stereotactic Radiosurgery
Brain Neoplasms, Adult, Malignant, Lymphoma, Sarcoma, Multiple Myeloma, Brain and Nervous System, Other, Eye and Orbit, Anklylosing Spondylitis, Anus, Bones and Joints, Breast - Female, Breast - Male, Cardiovascular, Cervix, Colon, Corpus Uteri, Ear, Esophagus, Gall Bladder, Head and Neck, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung/Thoracic, Melanoma, skin, Nose, Other Digestive Organ, Other Endocrine System, Other Female Genital, Other Male Genital, Other Respiratory and Intrathoracic Organs, Other Skin, Other Urinary, Ovary, Pancreas, Prostate, Rectum, Stomach, Throat, Thyroid, Urinary Bladder, Uterine (Endometrial), Vulva, Hodgkins Lymphoma, Lymphoid Leukemia, Small Intestine, Soft Tissue
UT Southwestern; Parkland Health & Hospital System
Study Of Palbociclib Combined With Chemotherapy In Pediatric Patients With Recurrent/Refractory Solid Tumors
This study will evaluate palbociclib in combination with chemotherapy (temozolomide with
irinotecan and/or topotecan with cyclophosphamide) in children, adolescents and young adults
with recurrent or refractory solid tumors. The main purpose of phase 1 portion of this study
is to evaluate the safety of palbociclib in combination with chemotherapy in order to
estimate the maximum tolerated dose. The main purpose of phase 2 portion is to compare the
efficacy of palbociclib in combination with irinotecan and temozolomide vs irinotecan and
temozolomide alone in the treatment of children, adolescents, and young adults with recurrent
or refractory Ewing sarcoma (EWS). Pharmacokinetics and efficacy of palbociclib in
combination with chemotherapy will be evaluated.
Inclusion:
1. Histologically confirmed relapsed or refractory solid tumor as follows:
• For dose escalation and dose determination parts: Histologically confirmed
relapsed or refractory solid tumor (including CNS tumors but not lymphomas).
Patients with Diffuse Intrinsic Pontine Glioma do not require histological only
radiographic confirmed relapse to enroll.
• For dose expansion and tumor specific cohorts: Histologically confirmed relapsed
or refractory solid tumor including but not limited to EWS, rhabdoid tumor,
rhabdomyosarcoma, neuroblastoma, and medulloblastoma. Patients with Diffuse
Intrinsic Pontine Glioma do not require histological only radiographic confirmed
relapse to enroll. EWS is not eligible for TOPO and CTX tumor-specific cohorts.
• For randomized Phase 2 part: Histologically confirmed Ewing sarcoma.
Histopathology confirmation of EWSR1-ETS or FUS-ETS rearrangement is required or
availability of formalin fixed paraffin embedded (FFPE) tumor tissue sample for
central testing. Patient must have relapsed or refractory disease with no known
bone marrow metastases and at least evaluable disease.
2. Age ≥2 and <21 years at the time of study entry.
3. Lansky performance status ≥50% for patients ≤16 years of age, or Eastern Cooperative
Oncology Group (ECOG) 0, 1 or 2 for patients >16 years of age.
4. Adequate bone marrow function.
• Absolute neutrophil count ≥1000/mm3;
• Platelet count ≥100,000/mm3 (transfusion independent, no platelet transfusion in
past 7 days prior study entry);
• Hemoglobin ≥8.5 g/dL (transfusion allowed).
5. Adequate renal function: Serum creatinine level based on age/gender must within
protocol specified limits.
6. Adequate liver function, including:
• Aspartate aminotransferase (AST) and alanine aminotransferase (ALT)
≤2.5 × upper limit of normal (ULN) or ≤5 × ULN for age, if attributable to
disease involvement of the liver;
• Total bilirubin ≤1.5 × ULN for age, unless the patient has documented Gilbert's
syndrome.
7. Patients enrolled to Phase 1 portion of the study and tumor specific cohorts must have
measurable disease as defined by RECIST version 1.1 or modified RANO criteria for CNS
disease or INRC for neuroblastoma. Patients with EWS enrolled to Phase 2 portion of
the study are eligible with evaluable disease (eg, bone only disease with no soft
tissue component).
8. Recovered to CTCAE Grade ≤1, or to baseline, from any non-hematological acute
toxicities of prior surgery, chemotherapy, immunotherapy, radiotherapy,
differentiation therapy or biologic therapy, with the exception of alopecia.
9. Serum/urine pregnancy test (for all girls ≥8 years of age) negative at screening and
at the baseline visit.
10. Evidence of a personally signed and dated informed consent document indicating that
the patient or a legally acceptable representative/parent(s)/legal guardian of minors,
has been informed of all pertinent aspects of the study. Minor study patients also
must provide age appropriate assent according to the local guidelines, where
applicable.
11. Willing and able to comply with scheduled visits, treatment plan, laboratory tests,
and other procedures.
Exclusion:
1. Phase 1 and tumor specific cohorts: For palbociclib with IRN and TMZ combination,
prior treatment with a CDK4/6 inhibitor or progression while on treatment with an
IRN-containing regimen that includes TMZ. Patients who have received the combination
of IRN and TMZ and did not progress while on these medications are eligible. For
patients enrolling in the palbociclib with TOPO and CTX combination, prior treatment
with a CDK4/6 inhibitor or progression while on treatment with a TOPO-containing
regimen that includes CTX. Patients who have received the combination of TOPO and CTX
and did not progress while on these medications are eligible. Phase 2 :prior treatment
with a CDK4/6 inhibitor or prior treatment with an IRN and/or TMZ-containing regimen.
2. Prior intolerability to IRN and/or TMZ plus/minus palbociclib with IRN and TMZ
combination and prior intolerability to TOPO and/or CTX for TOPO and CTX combination.
3. Use of strong cytochrome P450 (CYP) 3A inhibitors or inducers. Patients who are
receiving strong uridine diphosphate-glucuronosyl transferase 1A1 (UGT1A1) inhibitors
within 12 days of Cycle 1 Day 1 (C1D1) are not eligible for the palbociclib with IRN
and TMZ combination. Patients who are receiving strong UGT1A1 inhibitors within 12
days of C1D1 are eligible for the palbociclib with TOPO and CTX combination (See
Section 5.7.1 for list of products.)
4. Prior growth factors (including filgrastim) within 7 days before study entry or
PEG-filgrastim within 14 days before study entry.
5. Radiation therapy within 14 days before study entry.
6. Systemic anti cancer therapy within 2 weeks prior to study entry and 6 weeks for
nitrosoureas.
7. Previous high dose chemotherapy requiring stem cell rescue within 90 days or
persistent AE >Grade 1.
8. Prior irradiation to >50% of the bone marrow (see Appendix 9).
9. Participation in other studies involving investigational drug(s) within 2 weeks or 5
half lives, whichever is longer, prior to study entry.
10. Major surgery within 4 weeks prior to study entry. Surgical biopsies or central line
placement are not considered major surgeries.
11. For IRN and TMZ with/without palbociclib combinations: known or suspected
hypersensitivity to palbociclib, IRN and/or TMZ. For combination of palbociclib with
TOPO and CTX: known or suspected hypersensitivity to palbociclib, TOPO and/or CTX.
12. Patients with known symptomatic brain tumors or brain metastases and require steroids,
unless they have been on a stable or on a decreasing steroid dose for >14 days.
13. Patients with previously diagnosed brain metastases are eligible if they have
completed their prior treatment and have recovered from the acute effects of radiation
therapy or surgery prior to study entry for these metastases for at least 14 days post
radiation and 4 weeks post-surgery and are neurologically stable.
14. Hereditary bone marrow failure disorder.Phase 2 portion patients with bone marrow
involvement are excluded.
15. QTc >470 msec.
16. History of clinically significant or uncontrolled cardiac disease, including:
• History of or active congestive heart failure; if patient had congestive heart
failure resolve and >1 year from resolution, patient will be considered eligible;
• Clinically significant ventricular arrhythmia (such as ventricular tachycardia,
ventricular fibrillation or Torsades de Pointes);
• Diagnosed or suspected congenital or acquired prolonged QT syndrome;
• Need for medications known to prolong the QT interval;
• Uncorrected hypomagnesemia or hypokalemia because of potential effects on the QT
interval;
• Left ventricular ejection fraction <50% or shortening fraction <28%.
17. Recent or ongoing clinically significant gastrointestinal disorder that may interfere
with absorption of orally administered drugs (eg, gastrectomy).
18. Evidence of serious active or uncontrolled bacterial, fungal or viral infection or
known history of hepatitis B virus, hepatitis C virus, or human immunodeficiency virus
infection or acquired immunodeficiency syndrome-related illness.
19. Other severe acute or chronic medical or laboratory test abnormality that may increase
the risk associated with study participation or investigational product administration
or may interfere with the interpretation of study results, and in the judgment of the
Investigator, would make the patient inappropriate for entry into this study.
20. Investigator site staff members directly involved in the conduct of the study and
their family members, site staff members otherwise supervised by the investigator, or
patients who are Pfizer employees, including their family members, directly involved
in the conduct of the study.
21. Fertile male patients and female patients of childbearing potential who are unwilling
or unable to use a highly effective method of contraception as outlined in this
protocol for the duration of the study and for at least 90 after the last dose of
investigational product.
Neuroblastoma, Sarcoma, Solid Tumors, Ewing Sarcoma, Medulloblastoma, Diffuse Intrinsic Pontine Glioma, Rhabdomyosarcoma, Rhabdoid Tumor, Brain and Nervous System, Eye and Orbit, Anus, Bones and Joints, Breast - Female, Breast - Male, Cervix, Colon, Corpus Uteri, Ear, Esophagus, Gall Bladder, Head and Neck, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung/Thoracic, Nose, Other Digestive Organ, Other Endocrine System, Other Female Genital, Other Male Genital, Other Respiratory and Intrathoracic Organs, Other Skin, Other Urinary, Ovary, Pancreas, Prostate, Rectum, Stomach, Throat, Thyroid, Urinary Bladder, Uterine (Endometrial), Vulva, Kaposis sarcoma, Small Intestine, Soft Tissue, Ill - Defined Sites
FX-322 in Adults With Acquired Sensorineural Hearing Loss
This is a Phase 2, prospective, randomized, double-blind, placebo-controlled, single-dose,
multicenter study to evaluate the efficacy of FX-322, administered by intratympanic
injection, in adults with acquired sensorineural hearing loss (SNHL).
Call 214-648-5005 studyfinder@utsouthwestern.edu
Joe Kutz
94228
All
18 Years to 65 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT05086276
STU-2021-0808
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Inclusion Criteria:
1. Subject has read and voluntarily signed the Informed Consent Form (ICF) after all
questions have been answered and prior to any study-mandated procedure.
2. Adult aged 18-65 years inclusive at Screening.
3. Documented medical history consistent with acquired, adult onset, sensorineural
hearing loss associated with noise-induced SNHL (NIHL) or idiopathic sudden SNHL
(SSNHL) (documented audiogram at least 6 months prior to screening required).
4. A pure tone average at the Screening Visit of 35-85 dB at 500Hz, 1000Hz, 2000Hz, and
4000Hz in the ear to be injected.
5. Ability to communicate well with the Investigator and is willing to comply with and
complete all the study procedures.
6. Female subjects must be of non-childbearing potential or will need to utilize two
methods of highly effective contraception during the study participation (e.g.
hormonal contraception and condom or an intrauterine device and condom) or remain
abstinent. Male subjects should use condoms with spermicide during the course of the
study or remain abstinent. Subjects should not donate sperm or ova during the study
period.
7. Have met additional masked criteria as determined by the Electronic Data Capture
system.
Exclusion Criteria:
1. Subject has previously been randomized in a FX-322 clinical trial.
2. Perforation of tympanic membrane or other tympanic membrane disorders that would
interfere with the delivery and safety assessment of an intratympanic medication or
reasonably be suspected to affect tympanic membrane healing after injection in study
ear. This includes a current tympanostomy tube.
3. Any conductive hearing loss of greater than 15 dB at a single frequency or greater
than 10dB at two or more contiguous octave frequencies in the study ear at the
Screening visit.
4. Active chronic middle ear disease or a history of major middle ear surgery, as an
adult, in the ear to be injected.
5. Subject has had an intratympanic injection in either ear within 3 months of the
screening visit.
6. Evidence of or previous diagnosis of auditory neuropathy, traumatic brain injury,
"central" hearing loss, or genetic hearing loss.
7. History of chronic, recurrent clinically significant vestibular symptoms.
8. History of bilateral sudden sensorineural hearing loss or recurrent sudden
sensorineural hearing loss.
9. History of clinically significant systemic autoimmune disease (e.g. rheumatoid
arthritis, Sjogren's syndrome, multiple sclerosis, psoriasis).
10. History of head or neck radiation, treatment, or exposure to platinum based
chemotherapy drugs or aminoglycosides.
11. Exposure to another investigational drug within 28 days prior to screening visit.
12. Evidence of any active or chronic disease or condition that could interfere with, or
for which the treatment of might interfere with, the conduct of the study, or that
would pose an unacceptable risk to the subject in the opinion of the investigator
following a detailed medical history, physical examination, and vital signs (systolic
and diastolic blood pressure, pulse rate, body temperature).
13. Positive urine pregnancy test or breast-feeding.
14. Any known factor, condition, or disease that, in the view of the Investigator, might
interfere with treatment compliance, study conduct or interpretation of the results.
Olanzapine Versus Megestrol Acetate for the Treatment of Loss of Appetite Among Advanced Cancer Patients
This phase III trial compares the effects of olanzapine versus megestrol acetate in treating
loss of appetite in patients with cancer that has spread to other places in the body
(advanced). Olanzapine may stimulate and increase appetite. This study aims to find out if
olanzapine is better than the usual approach (megestrol acetate) for stimulating appetite and
preventing weight loss.
• Women and men of reproductive potential should agree to use an appropriate method of
birth control throughout their participation in this study due to the teratogenic
potential of the therapy utilized in this trial. Appropriate methods of birth control
include abstinence, oral contraceptives, implantable hormonal contraceptives or double
barrier method (diaphragm plus condom)
• Diagnosis of advanced cancer
• Patient-reported 2-month weight loss of at least 5 pounds (2.3 kilograms) and/or
physician-estimated caloric intake of less than 20 calories/kilogram of body weight
per day
• The patient must perceive loss of appetite and/or weight as a problem; and have an
appetite score of 4 or worse on the "Please rate your appetite…." question that
requires a patient response on a 0-10 numeric rating scale
• Not receiving ongoing tube feedings or parenteral nutrition at the time of
registration
• Not currently using systemic adrenal steroids (with the exception of short-term
dexamethasone within 3 days of chemotherapy for control of chemotherapy side effects)
• No use of androgens, progesterone analogs, or other appetite stimulants within the
past month
• Patient should not have poorly controlled hypertension or congestive heart failure at
registration
• Patient should not have an obstruction of the alimentary canal, malabsorption, or
intractable vomiting (defined as vomiting more than 3 times per day over the preceding
week)
• Not currently using olanzapine for another medical condition or had previously used
olanzapine for chronic nausea or for any pre-existing psychotic disorder
• Patient should not have had a previous blood clot at any time in the past
• No history of poorly controlled diabetes
• No symptomatic leptomeningeal disease or known brain metastases as these patients may
have difficulty taking oral medications
• No history of hypersensitivity to olanzapine or megestrol acetate
• No COVID-19 infection in the past that, in the opinion of the treating physician, had
left patients with compromised taste, which has not resolved at the time of
registration
• Not pregnant and not nursing, because this study involves an investigational agent
whose genotoxic, mutagenic and teratogenic effects on the developing fetus and newborn
are unknown. Therefore, for women of childbearing potential only, a negative urine or
serum pregnancy test done =< 14 days prior to registration is required
• Age >= 18 years
• Eastern Cooperative Oncology Group (ECOG) performance status 0, 1 or 2
• Estimated life expectancy of 3 months or longer
• Serum creatinine =< 2.0 mg/dL
• Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) =< 3 x upper limit
of normal (ULN)
• Fasting glucose > 1410 mg/dl
• Granulocytes > 1000/hpf
• No treatment with another antipsychotic agent, such as risperidone, quetiapine,
clozapine, butyrophenone within 30 days of enrollment
• In order to complete the mandatory patient-completed measures, participants must be
able to speak and/or read English or Spanish. Sites seeking to enroll Spanish-speaking
patients should have access to Spanish speaking staff on site or through the use of a
translation service to be able to conduct the informed consent discussion in Spanish,
and to conduct the weekly phone calls
Exclusion Criteria:
• Psychiatric illness which would prevent the patient from giving informed consent
• Medical condition such as uncontrolled infection (including human immunodeficiency
virus [HIV]), uncontrolled diabetes mellitus or cardiac disease which, in the opinion
of the treating physician, would make this protocol unreasonably hazardous for the
patient
• Patients who cannot swallow oral formulations of the agents
• Patients with impaired decision-making capacity (such as with a diagnosis of dementia
or memory loss) are not eligible for this study
• No presence of a hormone-sensitive tumor, such as breast, endometrial, or prostate
cancer (this exclusion criterion is intended to circumvent any confounding
antineoplastic effects of megestrol acetate)
Evaluation of [18F]FLT PET/CT as an Early Predictor of Outcome in Pediatric Solid Tumors
The experimental [18F]FLT-PET/CT will be completed before initiation of chemotherapy and
prior to the third cycle (or month) of chemotherapy. Laboratory analysis and correlative
radiology, as directed per clinical care based on the primary diagnosis, are required within
30 days of the baseline [18F]FLT PET/CT. Follow-up will comprise 24 months of standard
practice treatment and follow up.
• Patients with histologically confirmed solid tumor malignancies with residual tumors
present that require standard of care chemotherapy for a minimum number of cycles. All
anatomical sites and all tumor histologies are eligible including central nervous
system tumors.
• Patients ages 13 •25 years
• In the opinion of the investigator, patients must be thought to be able to lie still
for imaging without sedation for 20 •30 minutes.
• Patients must have a performance status of > 50% (Lansky or Karnofsky).
• Patients of childbearing potential must have a negative urine or serum pregnancy test
as per institution's standard of care within 7 days prior to [18F]FLT PET/CT imaging
• Ability to understand and the willingness to sign a written informed consent/assent.
Exclusion Criteria:
• Patients with known allergic or hypersensitivity reactions to previously administered
radiopharmaceuticals of similar chemical or biologic composition to [18F]FLT
• Subjects who had prior chemotherapy or radiotherapy before enrollment in the study.
• Subjects for whom chemotherapy is not a standard of care primary therapy option.
• Patients who are pregnant or breast-feeding
• Patients with no residual tumor (i.e. complete resection at diagnosis)
Drug: [18F]FLT-PET/CT
Sarcoma, Solid Tumor, Brain and Nervous System, Other, Eye and Orbit, Anus, Breast - Female, Breast - Male, Carcinoid Tumor, Cervix, Colon, Corpus Uteri, Ear, Esophagus, Gall Bladder, Head and Neck, Kidney, Larynx, Lip, Oral Cavity and Pharynx, Liver, Lung/Thoracic, Melanoma, skin, Nose, Ovary, Pancreas, Prostate, Rectum, Stomach, Throat, Thyroid, Urinary Bladder, Uterine (Endometrial), Vulva, Small Intestine, Soft Tissue
Multi-Center Study on Performance of the Ponto Implant System Using Minimally Invasive Ponto Surgery (in Adult Patients)
This prospective, multi-center study funded by Oticon Medical AB will be conducted at six
clinical sites in North America. Patients with a hearing loss and that are already planned
for treatment with a percutaneous (through the skin) bone-anchored hearing system (BAHS) will
be included in the study. The total number of participants included in the study will be 64.
The purpose of this study is to investigate the rate of successful BAHS use after
implantation of the Ponto Implant system using the surgical technique Minimally Invasive
Ponto Surgery (MIPS).
Call 214-648-5005 studyfinder@utsouthwestern.edu
Joe Kutz
94228
All
18 Years and over
This study is NOT accepting healthy volunteers
NCT04279236
STU-2021-0377
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Inclusion Criteria:
• 18 years of age or older
• Patient indicated for surgical intervention with a bone anchored hearing system
according to local clinic's standard guidelines.
• Normal bone quality and bone thickness above 3 mm, where no complications during
surgery are expected.
• Skin thickness of 12mm or less at the implant site
Exclusion Criteria:
• Patients undergoing re-implantation
• Patients who are unable or unwilling to follow investigational
procedures/requirements, e.g. to complete quality of life scales.
• Diseases or treatments known to compromise the bone quality at the implant site, e.g.
radiotherapy, osteoporosis, diabetes mellitus
• Known conditions (e.g. uncontrolled diabetes) that could jeopardize skin condition and
wound healing over time as judged by the investigator.
• Any other known condition that the investigator determines could interfere with
compliance or study assessments.