RecruitingPhase 2

Study of Novel Therapies for Young People With Recurrent/Progressive Atypical Teratoid Rhabdoid Tumor (ATRT)

United States29 participantsStarted 2026-06-17

Plain-language summary

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.

Who can participate

Age range

1 Year – 39 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * In addition to the below, investigators are to refer to arm-specific inclusion criteria 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) loss by immunohistochemistry (IHC) and/or biallelic loss of function of SMARCB1 by molecular report. Loss of SWI/SNF Related, Matrix Associated, Actin Dependent Regulator of Chromatin, Subfamily A, Member 4 (SMARCA4) as confirmed by IHC or molecular report is also acceptable but requires study chair approval. * 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 inclusion criteria 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…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Arm A (Phase I): Proportion of participants who experience dose-limiting toxicity (DLT)

Timeframe: up to 28 days

Arm A (Phase I): Recommended Phase 2 Dose (RP2D) (Phase I)

Timeframe: up to 28 days

Arm A (Phase II): Rate of Clinical Benefit

Timeframe: up to 2 years (24 cycles)

Trial details

NCT IDNCT07447076

SponsorSabine Mueller, MD, PhD

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2035-12-31

Contact for this trial

PNOC Operations Office

415-502-1600 PNOC035@ucsf.edu

View on ClinicalTrials.gov More Recurrent Atypical Teratoid/Rhabdoid Tumor trials

Who can participate

Age range

1 Year – 39 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.