GPC2-CAR T Cell Therapy for Relapsed or Refractory Medulloblastoma in Children and Young Adults
Trial Parameters
Brief Summary
This is a single-site, open-label Phase 1 clinical trial evaluating the feasibility, safety, and preliminary activity of autologous GPC2-targeted chimeric antigen receptor (CAR) T cells administered via intracerebroventricular (ICV) infusion in children and young adults with relapsed or refractory medulloblastoma or other eligible Central Nervous System (CNS) embryonal tumors.
Eligibility Criteria
Inclusion Criteria: 1. Diagnosis: Histologically confirmed diagnosis of medulloblastoma or other primary CNS embryonal tumor according to 2021 CNS WHO Classification (5th edition) * Other acceptable CNS embryonal tumors include: * Embryonal Tumor with Multilayered Rosettes (ETMR) * Pineoblastoma * Atypical Teratoid/Rhabdoid Tumor (ATRT) of the CNS * CNS neuroblastoma, FOXR2-activated * CNS Embryonal Tumor NOS 2. Recurrent/Refractory Disease: History of relapsed and/or recurrent disease defined as tumor progression or recurrence following initial diagnosis and upfront treatment with curative intent, or failure to achieve disease control with standard curative-intent therapy. 3. GPC2 Positive: H-score ≥ 100 by IHC staining performed on the (Prescreening Protocol IRB-78780, PI: Katherine Ryan, DO) at Stanford Clinical Anatomic Pathology Lab for GPC2 from a tumor sample any time since initial diagnosis. 4. Evaluable Disease: Evaluable disease as per radiographic findings and/or positive ce