Study of New Treatments for Young People with Recurrent or Worsening Atypical Teratoid Rhabdoid Tumor (ATRT)

Sponsor: Sabine Mueller, MD, PhD

Sponsor score: 0

ClinicalTrials ID:NCT07447076

Provider View

This clinical trial is exploring new treatment options for young patients with ATRT, a rare brain tumor, that has returned or is getting worse. The study aims to test the safety and effectiveness of different treatment combinations.

Patient Parameters

Program Overview

This study is part of the Pacific Pediatric Neuro-oncology Consortium (PNOC) and involves multiple treatment options for young people with ATRT that has come back or is progressing. The goal is to evaluate how safe and effective these new treatments are. Participants may switch to different treatment options if their current one is not working or causes side effects, as long as they meet the specific requirements for the new treatment.

Description

The main goals of this study are to see how well each treatment option works and how safe they are for patients with ATRT that has returned or is getting worse. Researchers will also collect samples like tumor tissue, blood, and spinal fluid to help discover new biomarkers for the disease.

Participants will join a specific treatment group as they become available. If a treatment is not effective or causes side effects, participants may switch to another group if they meet the criteria. The study will continue to follow participants until they leave the study or pass away.

The study will also look at how different genetic subgroups of the tumor respond to treatment and collect additional safety information. Participants will be monitored under the PNOC COMP protocol if it is available at their treatment center.

Eligibility Criteria

Inclusion Criteria

  • Must have a confirmed diagnosis of ATRT in the central nervous system, with specific genetic markers.
  • Must have a report confirming the tumor's genetic subgroup or enough tumor tissue for testing.
  • Must have ATRT that has returned or is getting worse.
  • Age between 1 and 39 years old at the time of joining the study.
  • Must have recovered from previous cancer treatments and meet specific waiting periods after those treatments.
  • Must have a performance score of at least 50, meaning they can perform daily activities with some help.
  • Must have stable or decreasing doses of corticosteroids for at least one week before joining.
  • Must have adequate bone marrow, kidney, liver, and neurological function.
  • Women of childbearing age and men must agree to use effective birth control during and after the study.
  • Must be able to understand and sign a consent form, or have a legal guardian do so.
  • Must join the PNOC COMP study if it is available at their treatment center.

Exclusion Criteria

  • Cannot have other types of tumors or cancers outside the central nervous system.
  • Cannot be taking other experimental or anti-cancer drugs.
  • Cannot have uncontrolled infections or other serious illnesses.
  • Women who are pregnant or breastfeeding cannot participate.
  • Cannot have allergies to similar treatments being tested.

Arm A Specific:

  • Must meet all general study criteria.
  • Must have measurable disease according to specific criteria, even if only in the spinal fluid.
  • Must be able to swallow capsules.
  • Must have normal blood sugar and cholesterol levels, with or without medication.
  • Must have normal heart function as shown by an ECG.

Arm A Exclusions:

  • Cannot have had previous treatment with gemcitabine or paxalisib.
  • Cannot be taking medications for high blood sugar.
  • Cannot have chronic diarrhea worse than Grade 2.

Locations

FACILITYZIPCITYSTATE
University of California, San Francisco94143San FranciscoCalifornia
University of California, San Francisco94143San FranciscoCalifornia

Get This Program In Your Inbox

Receive detailed information about this treatment opportunity to review with your healthcare provider.