Sabine Mueller, MD, PhD
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.
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 d…
Inclusion Criteria: * 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 molecul…
Cerebral Spinal Fluid (CSF) will be collected for research
Perform blood draw
Undergo imaging procedure
Given IV
Given orally (PO)
Undergo biopsy
Arctuva estimate
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