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New treatment option for Recurrent Atypical Teratoid/Rhabdoid Tumor
Official title Study of Novel Therapies for Young People With Recurrent/Progressive Atypical Teratoid Rhabdoid Tumor (ATRT)
ClinicalTrials.gov ID: NCT07447076
What this study is testing
What is Gemcitabine?
Gemcitabine is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for recurrent atypical teratoid/rhabdoid tumor.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This is a multi-treatment arm study that will be conducted through the Pacific Pediatric Neuro-oncology Consortium (PNOC).The study will assess the safety and efficacy of novel therapies and combinatorial strategies for participants with recurrent or progressive ATRT.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 1 to 39
You may be able to join if
- In addition to the below, investigators are to refer to arm-specific in the appendix.
- Participants must have a pathologic diagnosis of central nervous system (CNS) ATRT, with confirmation of SWI/SNF-related, matrix-associated...
- Participants must have confirmation of methylation report, co-enrollment on PNOC-030 or sufficient tumor tissue available for methylation-based...
- Participants must have recurrent or progressive ATRT.
- Participants age must be ≥1 and ≤ 39 years at the time of study enrollment. Please refer to arm specific for potential variations in lower age...
You likely can't join if
- Evidence of synchronous tumors or other extra-CNS malignancy
- Participants who are receiving any other investigational agents
- Participants who are currently receiving other anti-cancer agents
- Participants with uncontrolled infection or other uncontrolled systemic illness
- Female participants of childbearing potential who are pregnant or breast-feeding. Female participants of childbearing potential must have a negative...
- History of allergic reactions attributed to compounds of similar chemical or biologic composition as the intended treatment regimen, as detailed in...
See the full eligibility criteria
Who can join
- In addition to the below, investigators are to refer to arm-specific in the appendix.
- Participants must have a pathologic diagnosis of central nervous system (CNS) ATRT, with confirmation of SWI/SNF-related, matrix-associated, actin-dependent regulator of chromatin, subfamily b, member 1 (SMARCB1) (INI1)...
- Participants must have confirmation of methylation report, co-enrollment on PNOC-030 or sufficient tumor tissue available for methylation-based subgrouping
- Participants must have recurrent or progressive ATRT.
- Participants age must be ≥1 and ≤ 39 years at the time of study enrollment. Please refer to arm specific for potential variations in lower age eligibility limit.
- Prior Therapy: Participants must have fully recovered from the acute effects of prior anti-cancer therapy, and the following wash-out periods need to be observed prior to enrollment:
- Systemic myelosuppressive therapy: ≥ 21 days after the last dose (42 days for nitrosoureas or mitomycin C).
- Intrathecal/intraventricular chemotherapy: \> 7 days after the last dose.
- Small molecule/targeted/biologic agent: ≥ 7 days after the last dose.
- Monoclonal antibodies: ≥ 21 days after the last dose. Other non-myelosuppressive anti-cancer agents: ≥ 3 drug half-lives after the last dose.
- CAR-T cell therapy (systemic or intraventricular): \> 21 days.
- Previous radiotherapy. Participants will be eligible following radiotherapy, if they meet the following criteria:
- Previous craniospinal or total body radiotherapy: Participants must have received their last fraction ≥ 12 weeks prior to enrollment and have evidence of progressive/recurrent evaluable disease post radiation.
- Previous focal radiotherapy to target lesions: Participants must have received their last fraction to target lesions ≥12 weeks prior to enrollment and have evidence of progressive/recurrent evaluable disease post...
- Focal radiotherapy to non-target lesions: Participants may have received radiotherapy to nontarget lesions as long as the last fraction was \> 14 days prior to enrollment. Participants must have at least one...
- Performance Score: Karnofsky ≥ 50 for participants \> 16 years of age and Lansky ≥ 50 for participants ≤ 16 years of age. Participants who are unable to walk because of paralysis, but who are up in a wheelchair, will be...
- Corticosteroids: Participants who are receiving corticosteroids must be on a stable or decreasing dose for at least 1 week prior to enrollment. Please also refer to arm-specific for potential variations in steroid...
- Organ Function Requirements. Adequate Bone Marrow Function Defined as:
- Peripheral absolute neutrophil count (ANC) ≥ 750/mm3
- Platelet count ≥ 75,000/cubic millimeters (mm3) (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment). Adequate Renal Function Defined as:
- Serum creatinine ≤ 1.5 Upper Limit Normal (ULN) based on age and gender Adequate Liver Function Defined as:
- Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age; in presence of Gilbert's syndrome, total bilirubin \< 3 x ULN or direct bilirubin \< 1.5 x ULN
- Alanine aminotransferase (ALT) ≤ 3 x ULN
- Aspartate aminotransferase (AST) ≤ 3 x ULN Adequate Neurologic Function Defined as:
- Participants with seizure disorder may be enrolled if well controlled. See arm-specific recommendations for potential interactions between anticonvulsant agent(s) with study drug.
- Effect on the developing human fetus Recommendations on the potential effect of treatment agents on the developing human fetus will be specified in each study arm's details of therapeutic agents. Unless otherwise...
- A legal parent/guardian or patient must be able to understand, and willing to sign, a written informed consent and assent document, as appropriate.
- Participants must enroll on Pediatric Neuro-oncology Consortium (PNOC) COMP if PNOC COMP is open to accrual at the enrolling institution.
What rules you out
- Evidence of synchronous tumors or other extra-CNS malignancy
- Participants who are receiving any other investigational agents
- Participants who are currently receiving other anti-cancer agents
- Participants with uncontrolled infection or other uncontrolled systemic illness
- Female participants of childbearing potential who are pregnant or breast-feeding. Female participants of childbearing potential must have a negative serum or urine pregnancy test prior to the start of therapy and...
- History of allergic reactions attributed to compounds of similar chemical or biologic composition as the intended treatment regimen, as detailed in that arm's treatment description. Arm A Inclusion Criteria:
- people must meet all inclusion criteria for the overall study.
- Patients must be evaluable per Response Assessment in Pediatric Neuro-Oncology (RAPNO) criteria for medulloblastoma and other leptomeningeal seeding tumors to be evaluated for the primary endpoint (Warren et al. 2018)...
- people must be able to swallow intact capsules.
- Adequate Metabolic Function Defined as:
- Non-fasting glucose ≤ 140 milligrams per deciliter (mg/dL) without the use of antihyperglycemic agents.
- If non-fasting glucose \> 140 mg/dL, a fasting glucose should be done. If fasting glucose ≤ 125 mg/dL without the use of antihyperglycemic agents, participant will meet adequate metabolic function criteria.
- Triglycerides of \< 300 mg/dl and total cholesterol of \< 300 mg/dl - can be on lipid lowering medications as needed to achieve.
- Adequate Cardiac Function Defined as:
- Electrocardiogram (ECG) must be obtained to verify the Corrected QT Interval (QTC). If an abnormal reading is obtained, the ECG should be repeated in triplicate.
- QTC \< 470 millisecond (msec) Arm A Exclusion Criteria: In addition to not meeting any of the exclusion criteria of the overall study, participation on arm A will also require that people do not meet any of the...
- Previous exposure to gemcitabine or paxalisib
- Concomitant use of an antihyperglycemic agent (e.g. metformin)
- Chronic diarrhea greater than Grade 2
The study team makes the final eligibility decision.
Where it's taking place
- San Francisco, California, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 1 year to 39 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include San Francisco, California, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.