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GPC2-CAR T Cell Therapy for Relapsed or Refractory Medulloblastoma in Children and Young Adults

NCT07087002 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 18

Last updated 2026-01-27

No results posted yet for this study

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.

Conditions

  • Medulloblastoma
  • Central Nervous System Embryonal Tumor
  • Refractory Medulloblastoma
  • Recurrent Medulloblastoma
  • Pediatric Brain Tumor
  • Embryonal Tumor With Multilayered Rosettes (ETMR)
  • Pineoblastoma
  • Atypical Teratoid/Rhabdoid Tumor (ATRT) of the CNS
  • CNS Neuroblastoma
  • FOXR2-activated

Interventions

BIOLOGICAL

GPC2-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.

DRUG

Fludarabine

Administered as part of a lymphodepleting chemotherapy regimen prior to GPC2-CAR T cell infusion. Dose: 30 mg/m²/day for 3 days.

DRUG

Cyclophosphamide

Administered with fludarabine for lymphodepletion. Dose: 500 mg/m²/day for 3 days.

Sponsors & Collaborators

Principal Investigators

  • Katherine Ryan, DO · Stanford University

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Year
Max Age
30 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-08-28
Primary Completion
2027-08-31
Completion
2027-08-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07087002 on ClinicalTrials.gov