Novel Therapies for Brain Cancer

Overseen By PNOC Operations Office

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Sabine Mueller, MD, PhD

Must not be taking: Antihyperglycemics

Disqualifiers: Synchronous tumors, Uncontrolled infection, Pregnancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 6

What You Need to Know Before You Apply

What is the purpose of 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.

Who Is on the Research Team?

Sabine Mueller, MD, PhD

Principal Investigator

University of California, San Francisco

Ashley Margol, MD, MS

Principal Investigator

Children's Hospital Los Angeles

Are You a Good Fit for This Trial?

Inclusion Criteria

Participants must meet performance score criteria.

Participants must have confirmation of methylation report, co-enrollment on PNOC-030 or sufficient tumor tissue available for methylation-based subgrouping

Participants must comply with contraception requirements.

Exclusion Criteria

I am not pregnant or breastfeeding.

Participants must not have a history of allergic reactions to compounds of similar composition as the intended treatment regimen.

I do not have any uncontrolled infections or serious illnesses.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Phase I

Participants receive a combination of gemcitabine and paxalisib to determine the recommended Phase 2 dose (RP2D).

4 weeks

3 visits (in-person) for infusion on days 1, 8, and 15

Treatment Phase II

Participants 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.

up to 2 years

3 visits (in-person) per cycle for infusion on days 1, 8, and 15

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 months

What Are the Treatments Tested in This Trial?

How Is the Trial Designed?

Group I: Treatment Arm A: Phase I Experimental Treatment 6 Interventions

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.

Group II: Treatment Arm A: Efficacy (Phase II) Experimental Treatment 6 Interventions

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.

Who Is Running the Clinical Trial?

Sabine Mueller, MD, PhD

Lead Sponsor

Trials

9

Recruited

440+

Pacific Pediatric Neuro-Oncology Consortium

Collaborator

Trials

16

Recruited

840+

Rally Foundation

Collaborator

Trials

2

Recruited

8,000+