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

NCT07447076 · Status: NOT_YET_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 29

Last updated 2026-03-03

No results posted yet for this study

Summary

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.

Conditions

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

Interventions

PROCEDURE

Lumber Puncture

Cerebral Spinal Fluid (CSF) will be collected for research

PROCEDURE

Blood Specimen Collection

Perform blood draw

PROCEDURE

Magnetic resonance imaging (MRI)

Undergo imaging procedure

DRUG

Gemcitabine

Given IV

DRUG

Paxalisib

Given orally (PO)

PROCEDURE

Tumor Biopsy

Undergo biopsy

Sponsors & Collaborators

  • Pacific Pediatric Neuro-Oncology Consortium

    collaborator OTHER

  • Rally Foundation

    collaborator UNKNOWN

  • Sabine Mueller, MD, PhD

    lead OTHER

Principal Investigators

  • Sabine Mueller, MD, PhD · University of California, San Francisco

  • Ashley Margol, MD, MS · Children's Hospital Los Angeles

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
1 Year
Max Age
39 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-05-01
Primary Completion
2035-12-31
Completion
2036-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07447076 on ClinicalTrials.gov