Phase I Clinical Trial of GPC2 Chimeric Antigen Receptor T (GPC2-CAR T) Cells for Relapsed or Refractory Medulloblastoma in Children and Young Adults

Summary

This is a single-site, open-label Phase I treatment study evaluating the manufacturing feasibility and safety of chimeric antigen receptor (CAR) T cells targeting the oncofetal protein Glypican-2 (GPC2) in pediatric and young adult patients with recurrent or refractory (R/R) medulloblastoma. Subjects with other non-medulloblastoma CNS embryonal tumors may enroll.

Eligibility

Age range

1–30 years

Sex

All

Healthy volunteers

No

Interventions

• Genetic
  GPC2-CAR T cells

  Autologous T cells transduced with a retroviral vector encoding a second-generation GPC2-targeted chimeric antigen receptor (GPC2-CAR), administered intracerebroventricularly. Up to 16 doses (cycles) are administered using an intrapatient dose-escalation strategy. Cycle 2 may occur no earlier than Day 28 following Cycle 1, and Cycles 3 through 16 may occur as early as Day 22 following the preceding cycle.

• Drug
  Fludarabine

  Participants who meet eligibility criteria for lymphodepletion chemotherapy will receive fludarabine 30 mg/m²/day administered intravenously on Days -4, -3, and -2 prior to study treatment. Participants weighing \<12 kg will receive protocol-specified dose adjustments.

• Drug
  Cyclophosphamide

  Participants who meet eligibility criteria for lymphodepletion chemotherapy will receive cyclophosphamide 500 mg/m²/day administered intravenously on Days -4, -3, and -2 prior to study treatment. Participants weighing \<12 kg will receive protocol-specified dose adjustments.

Location