NCT07447076

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

A Platform Study of Novel Therapies for Children, Adolescents and Young Adults With Recurrent/Progressive Atypical Teratoid Rhabdoid Tumor (ATRT)

• Status: Not Yet Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 29 (estimated)
• Sponsor: Sabine Mueller, MD, PhD · Academic / Other
• Sex: All
• Age: 1 Year – 39 Years
• Healthy volunteers: Not accepted

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.

Detailed description

PRIMARY OBJECTIVES: I. To assess efficacy of each treatment arm based on arm-specific endpoints. II. To evaluate the safety and tolerability of each treatment arm for patients with recurrent/progressive ATRT. EXPLORATORY OBJECTIVES: I. To collect additional treatment arm-specific safety and tolerability information as applicable. II. To determine additional treatment arm-specific time-to-event endpoints. III. To assess the correlations between methylation-based subgroups and objective response and outcome measures. IV. To collect biologic samples (tumor, blood, CSF) for future biomarker discovery. V. Additional treatment arm-specific exploratory objectives may be added in each interventional arm. OUTLINE: Participants will enroll onto a treatment arm as treatment arms open for enrollment. Participants who are taken off treatment on one trial arm for progression or toxicity may enroll on a new treatment arm, if arm-specific eligibility criteria are met. If the study is opened at the treating institution, participants will be followed under the Pediatric Neuro-oncology Consortium (PNOC) COMP protocol until death or withdrawal from study.

Conditions

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

Interventions

Type	Name	Description
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

Timeline

• Start date: 2026-05-01
• Primary completion: 2035-12-31
• Completion: 2036-12-31
• First posted: 2026-03-03
• Last updated: 2026-03-03

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study