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GPC2-CAR T Cell Therapy for Medulloblastoma

This study is testing a new treatment called GPC2-CAR T cells for children and young adults (ages 1 to 30) with medulloblastoma or other specific brain tumors that have come back or are hard to treat. Before receiving the GPC2-CAR T cells, participants will get chemotherapy with fludarabine and cyclophosphamide. The GPC2-CAR T cells are given directly into the fluid around the brain. Researchers want to see if this treatment is safe, if it can be made successfully, and if it shows any early signs of working. The study plans to enroll 18 participants.

Study design
This is a single-site, open-label Phase 1 study, meaning everyone knows what treatment they are getting. It plans to enroll 18 participants.
What's involved
Participants will receive chemotherapy for 3 days, followed by up to 8 doses of GPC2-CAR T cells given every 28 days. The manufacturing of the GPC2-CAR T cells will be assessed for up to 6 months.
Compensation
Not stated in the trial record.
Follow-up
The study measures manufacturing feasibility for up to 6 months after a procedure called leukapheresis. Safety is assessed within the first 28-day treatment cycle for each dose level.

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NCT07087002

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

Recruiting
PHASE1Ages 1–30InterventionalTreatment
Stanford University
~18 participants
Updated 2026-01-27 on ClinicalTrials.gov
What's tested:GPC2-CAR T cellsFludarabineCyclophosphamide

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Manufacturing Feasibility
Measured over Up to 6 months post-leukapheresis
+1 more outcome measured

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

Explore related trials

Atypical Teratoid/Rhabdoid TumorCentral Nervous System Embryonal TumorEmbryonal Tumor with Multilayered RosettesMedulloblastomaNeuroblastomaPineoblastomaGPC2 Gene

Where it's being run

1 sites across 1 states
California1

Study leadership

  • Katherine Ryan, DO · PRINCIPAL_INVESTIGATOR · Stanford University

Who to contact

Mariah Duncan
[email protected]
(650) 497-8953
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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 cerebrospinal fluid cytology within 28 days of enrollment. 5. Patients with VP shunts: Patients with pre-existing ventriculo-peritoneal (VP) shunt devices must have a programmable shunt device to enroll on this study. A VP shunt is not a requirement for this study. 6. Prior therapy: No limit to the number of prior treatment regimens. Toxicities due to prior therapy must be stable or recovered to ≤ Grade 1 (except for clinically non-significant toxicities such as alopecia, nutritional support measures, electrolyte abnormalities, or those not impacting the investigator's ability to assess treatment emergent toxicities).

What this trial measures

  • Manufacturing FeasibilityUp to 6 months post-leukapheresis

    Proportion of manufacturing attempts that result in at least one dose of GPC2-CAR T cells that meet the IND release criteria and protocol-specified dose.

  • Incidence of Dose Limiting Toxicities (DLTs)Within first 28-day treatment cycle per dose level

    Number and severity of DLTs following GPC2-CAR T cell administration at each dose level using protocol-defined criteria.