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RECRUITINGPHASE1

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

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

NCT07087002•Stanford University

View on ClinicalTrials.gov

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

1 - 30 years

Sex

ALL

Healthy Volunteers

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 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.
+26 more criteria

Exclusion Criteria

•1. Any patient with metastatic disease OUTSIDE the CNS.
•2. Unwilling or unable, in the investigator's judgement, to have a CSF reservoir (Ommaya or Rickham) placed. Does not apply to subjects who have a pre-existing device suitable for ICV delivery of CAR T cells and ICP monitoring.
•3. Clinical evidence of active/on-going significant increased intracranial pressure (i.e., impending herniation) or uncontrolled seizures.
•4. Prior receipt of a chimeric antigen receptor (CAR)-based therapy.
•5. Currently receiving anticoagulation therapy.
•6. Known history of infection with HIV or hepatitis B (HBsAg positive) or hepatitis C virus (anti-HCV positive).
•EXCEPTION: A history of hepatitis B or hepatitis C is permitted if the viral load is undetectable per quantitative PCR and/or nucleic acid testing.
•7. Pregnancy or breastfeeding in a postpartum female.
•8. Known sensitivity or allergy to any agents/reagents used in this study.
•9. History of prior other malignancy. EXCEPTION: Previously diagnosed and definitively treated more than 5 years prior to enrollment or whose prognosis is deemed good enough to not warrant surveillance.
+4 more criteria

Locations (1)

Lucile Packard Children's Hospital Stanford

Palo Alto, California, United States

Key Dates

Start Date

August 28, 2025

Primary Completion Date

August 1, 2027

Completion Date

August 1, 2027

Last Updated

January 27, 2026

Enrollment

ESTIMATED participants

Conditions

MedulloblastomaCentral Nervous System Embryonal TumorRefractory MedulloblastomaRecurrent MedulloblastomaPediatric Brain TumorEmbryonal Tumor With Multilayered Rosettes (ETMR)PineoblastomaAtypical Teratoid/Rhabdoid Tumor (ATRT) of the CNSCNS NeuroblastomaFOXR2-activated

Interventions

GPC2-CAR T cells

BIOLOGICAL

Fludarabine

DRUG

Cyclophosphamide

DRUG

Contacts

Mariah Duncan

CONTACT

(650) 497-8953 GPC2CART@stanfordchildrens.org

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: January 27, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

Molecular and Clinical Risk-Directed Therapy for Infants and Young Children With Newly Diagnosed Medulloblastoma

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