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Study of New Treatments for Recurrent/Progressive ATRT

This study is looking into new ways to treat Atypical Teratoid Rhabdoid Tumor (ATRT) in young people when the cancer has come back or gotten worse. It's testing the safety and effectiveness of different treatments, including the drugs Gemcitabine (given through a vein) and Paxalisib (taken by mouth). To join, you must be between 1 and 39 years old and have a confirmed diagnosis of ATRT in the brain or spinal cord, with a specific genetic change called SMARCB1 (INI1) loss. Researchers will be looking to see if these treatments can lead to clinical benefit, which means the treatment is helping you feel better or stopping the cancer from growing. The study plans to enroll 29 participants.

Study design
This is an interventional study with multiple treatment arms, meaning different groups will receive different treatments. The study will enroll 29 participants.
What's involved
You would undergo procedures like lumbar punctures (spinal taps) to collect spinal fluid, blood draws, and Magnetic Resonance Imaging (MRI) scans. You would receive Gemcitabine intravenously or Paxalisib orally.
Compensation
Not stated in the trial record.
Follow-up
The study will assess clinical benefit for up to 2 years (24 cycles) after treatment.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07447076

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

Recruiting
PHASE2Ages 1–39InterventionalTreatment
Sabine Mueller, MD, PhD
~29 participants
Updated 2026-06-23 on ClinicalTrials.gov
What's tested:Lumber PunctureBlood Specimen CollectionMagnetic resonance imaging (MRI)GemcitabinePaxalisibTumor Biopsy

At a glance

Recruiting sites
1 of 2 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Arm A (Phase I): Proportion of participants who experience dose-limiting toxicity (DLT)
Measured over up to 28 days
+2 more outcomes measured

Conditions

Recurrent Atypical Teratoid/Rhabdoid Tumor
Atypical Teratoid/Rhabdoid Tumor (ATRT) of the CNS

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NCT07447076

Where you'd take part

This study runs at 2 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • University of California, San Francisco

    San Francisco, Californiastudy coordinator listed

    Not yet recruiting

  • University of California, San Francisco

    San Francisco, Californiastudy coordinator listed

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Sabine Mueller, MD, PhD · PRINCIPAL_INVESTIGATOR · University of California, San Francisco
  • Ashley Margol, MD, MS · STUDY_CHAIR · Children's Hospital Los Angeles

Who to contact

PNOC Operations Office
[email protected]
415-502-1600
Email the study team

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Eligibility criteria

Inclusion

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.
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 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 radiation; investigators are reminded to review potentially eligible cases to avoid confusion with pseudo-progression.
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 non-irradiated lesion that is evaluable for response.
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 considered ambulatory for the purpose of assessing the performance score.
Organ Function Requirements.
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).
Serum creatinine ≤ 1.5 Upper Limit Normal (ULN) based on age and gender
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
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 interventional agents on the developing human fetus will be specified in each study arm's details of therapeutic agents. Unless otherwise specified, the effects of study interventions should be considered potentially teratogenic. Thus, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study treatment and four months after its completion. Should a woman become pregnant or suspect she is pregnant while she is participating in this study, or should a male participant's partners become pregnant during study participation, they should inform the treating physician immediately.
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.
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); patients with evaluable but non-measurable disease, including leptomeningeal disease or positive CSF cytology only are eligible.
Subjects 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)

Exclusion

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 throughout study treatment.
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.
Previous exposure to gemcitabine or paxalisib
Concomitant use of an antihyperglycemic agent (e.g. metformin)
Chronic diarrhea greater than Grade 2

What this trial measures

  • Arm A (Phase I): Proportion of participants who experience dose-limiting toxicity (DLT)up to 28 days

    Tolerability is defined as the proportion of participants receiving at least one dose of combination gemcitabine and paxalisib with a reported dose-limiting toxicity (DLT) during cycle 1 for all participants in Phase I.

  • Arm A (Phase I): Recommended Phase 2 Dose (RP2D) (Phase I)up to 28 days

    The confirmed RP2D of combination gemcitabine and paxalisib implemented for participants enrolled in Phase II will be reported.

  • Arm A (Phase II): Rate of Clinical Benefitup to 2 years (24 cycles)

    Assess the efficacy of combination gemcitabine and paxalisib for participants in Phase II. Clinical Benefit rate (CBR) is defined as complete response (CR) + partial response (PR) + stable disease (SD), where SD is sustained over 4 months.