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

Part of paid clinical trials in Palo Alto, California.

Sponsor
Stanford University
Study ID
NCT07087002
Phase
PHASE1
Status
Recruiting
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Conditions

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

Eligibility Criteria

Sex
ALL
Age
1 Year - 30 Years
Healthy Volunteers
Not accepted

Interventions

  • GPC2-CAR T cells — BIOLOGICAL
    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.
  • Fludarabine — DRUG
    Administered as part of a lymphodepleting chemotherapy regimen prior to GPC2-CAR T cell infusion. Dose: 30 mg/m²/day for 3 days.
  • Cyclophosphamide — DRUG
    Administered with fludarabine for lymphodepletion. Dose: 500 mg/m²/day for 3 days.

Study Details

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.

Key Dates

Start date
Aug 28, 2025
Status verified
Jan 2026
Primary completion
Aug 31, 2027
Completion
Aug 31, 2027

Study Design

Enrollment
18 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: GPC2-CAR T Cell Therapy
    Participants will undergo leukapheresis for collection of peripheral blood mononuclear cells, which will be used to manufacture autologous T cells transduced with a retroviral vector encoding a GPC2-targeted chimeric antigen receptor (GPC2-CAR T cells). After lymphodepleting chemotherapy with fludarabine and cyclophosphamide, participants will receive up to 8 cycles of intracerebroventricular (ICV) GPC2-CAR T cell infusions using an intrapatient dose escalation strategy.

Primary Outcome Measure

Manufacturing Feasibility [ Time Frame: Up to 6 months post-leukapheresis ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Lucile Packard Children's Hospital StanfordPalo AltoCalifornia94304
Mariah Duncan
[email protected]
650-497-8953
Katherine Ryan, DO (PRINCIPAL_INVESTIGATOR)
Sabine Heitzeneder, MD (SUB_INVESTIGATOR)

Find similar trials in Palo Alto, CA

By condition
Brain cancer
By specialty
OncologyNeuro-oncology
By research site
Lucile Packard Children's Hospital Stanford· Palo Alto, CA

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