RecruitingPhase 1NCT07087002

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


Sponsor

Stanford University

Enrollment

18 participants

Start Date

Aug 28, 2025

Study Type

INTERVENTIONAL

Conditions

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

Min Age: 1 YearMax Age: 30 Years

Plain Language Summary

Simplified for easier understanding

This study is testing a new CAR-T cell therapy called GPC2-CAR T cells — where a patient's own immune cells are genetically modified to target and destroy brain tumor cells — in children and young adults with medulloblastoma or related rare brain tumors (embryonal tumors) that have returned or progressed after prior treatment. **You may be eligible if...** - You have been diagnosed with medulloblastoma or another related embryonal brain tumor, confirmed by biopsy - Your tumor has come back or progressed after initial treatment with curative intent - You are typically under 30 years old (pediatric and young adult patients) - Your cancer cells test positive for the GPC2 protein target **You may NOT be eligible if...** - You have not had any prior treatment for the tumor - You have serious organ dysfunction (heart, liver, kidney, or lung) - You have active serious infections - Your overall health is too poor for intensive treatment Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

GENETICGPC2-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.

DRUGFludarabine

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.

DRUGCyclophosphamide

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.


Locations(1)

Lucile Packard Children's Hospital Stanford

Palo Alto, California, United States

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NCT07087002


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