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

ConditionsRecurrent Atypical Teratoid/Rhabdoid TumorAtypical Teratoid/Rhabdoid Tumor (ATRT) of the CNS
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age1-39
SponsorSabine Mueller, MD, PhD

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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

Trial design

Design model

Parallel

Treatments tested in this trial

  • Lumber Puncture

    Procedure/Surgery

    Cerebral Spinal Fluid (CSF) will be collected for research

  • Blood Specimen Collection

    Procedure/Surgery

    Perform blood draw

  • Magnetic resonance imaging (MRI)

    Procedure/Surgery

    Undergo imaging procedure

  • Gemcitabine

    Drug

    Given IV

  • Paxalisib

    Drug

    Given orally (PO)

  • Tumor Biopsy

    Procedure/Surgery

    Undergo biopsy

Treatment groups

29 Participants
are divided into 2 treatment groups
Group A: Treatment Arm A: Phase IExperimental treatment 6 interventions
Group B: Treatment Arm A: Efficacy (Phase II)Experimental treatment 6 interventions

Trial outcomes

Primary outcomes

1

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

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.

Time frame
up to 28 days
2

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

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

Time frame
up to 28 days
3

Arm A (Phase II): Rate of Clinical Benefit

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.

Time frame
up to 2 years (24 cycles)

Secondary outcomes

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Sabine Mueller, MD, PhD

Lead sponsor

University of California, San Francisco

Sponsor institution

Pacific Pediatric Neuro-Oncology Consortium

Collaborator

Rally Foundation

Collaborator

Source ClinicalTrials.gov
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