Phase I Clinical Trial of GPC2 Chimeric Antigen Receptor T (GPC2-CAR T) Cells for Relapsed or Refractory Medulloblastoma in Children and Young Adults
Stanford University
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
- Age range
- 1–30 years
- Sex
- All
- Healthy volunteers
- No
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 treatm…
Interventions
- BiologicalGPC2-CAR T cells
Autologous T cells transduced with retroviral vector encoding a second-generation GPC2-targeted chimeric antigen receptor (GPC2-CAR), administered intracerebroventricularly. Up to 8 doses are given every 28 days, following an intrapatient dose escalation schema.
- DrugFludarabine
Administered as part of a lymphodepleting chemotherapy regimen prior to GPC2-CAR T cell infusion. Dose: 30 mg/m²/day for 3 days.
- DrugCyclophosphamide
Administered with fludarabine for lymphodepletion. Dose: 500 mg/m²/day for 3 days.
Location
- Lucile Packard Children's Hospital StanfordPalo Alto, California