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

Part of paid clinical trials in San Francisco, California.

Sponsor
Sabine Mueller, MD, PhD
Study ID
NCT07447076
Phase
PHASE2
Status
Not Yet Recruiting

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Conditions

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

Eligibility Criteria

Sex
ALL
Age
1 Year - 39 Years
Healthy Volunteers
Not accepted

Interventions

  • Lumber Puncture — PROCEDURE
    Cerebral Spinal Fluid (CSF) will be collected for research
  • Blood Specimen Collection — PROCEDURE
    Perform blood draw
  • Magnetic resonance imaging (MRI) — PROCEDURE
    Undergo imaging procedure
  • Gemcitabine — DRUG
    Given IV
  • Paxalisib — DRUG
    Given orally (PO)
  • Tumor Biopsy — PROCEDURE
    Undergo biopsy

Study Details

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.

Key Dates

Start date
May 1, 2026
Status verified
Feb 2026
Primary completion
Dec 31, 2035
Completion
Dec 31, 2036

Study Design

Enrollment
29 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Treatment Arm A: Phase I
    Participants will receive a combination of gemcitabine and paxalisib with starting dose, 1,200mg/m2 gemcitabine and 15mg/m2 paxalisib (phase I). If this dose level is tolerated, it will be confirmed as the RP2D. However, if the dose limiting toxicity (DLT) is not tolerated, the RP2D will be set at the highest dose level tolerated by the previous group.
  • Experimental: Treatment Arm A: Efficacy (Phase II)
    All participants will receive a combination of gemcitabine and paxalisib over a 28-day cycle, for up to 24 cycles or until they meet criteria to stop treatment, whichever comes first. Gemcitabine will be administered via infusion on days 1, 8, and 15 of each cycle, while paxalisib will be taken daily throughout each cycle.

Primary Outcome Measure

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

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
University of California, San FranciscoSan FranciscoCalifornia94143
PNOC Operations Office
[email protected]
415-502-1600
[email protected]
Sabine Mueller, MD, PhD (PRINCIPAL_INVESTIGATOR)

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By research site
University of California, San Francisco· San Francisco, CA

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