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

ConditionsMedulloblastomaCentral Nervous System Embryonal TumorRefractory MedulloblastomaRecurrent MedulloblastomaPediatric Brain TumorEmbryonal Tumor With Multilayered Rosettes (ETMR)PineoblastomaAtypical Teratoid/Rhabdoid Tumor (ATRT) of the CNSCNS NeuroblastomaFOXR2-activated
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age1-30
SponsorStanford University

About this trial

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 criteria

This trial does not accept healthy volunteers

Qualifiers

Diagnosis: Histologically confirmed diagnosis of medulloblastoma or other primary CNS embryonal tumor according to 2021 CNS WHO Classification (5th edition)

Embryonal Tumor with Multilayered Rosettes (ETMR)

Pineoblastoma

Atypical Teratoid/Rhabdoid Tumor (ATRT) of the CNS

Disqualifiers

Any patient with metastatic disease OUTSIDE the CNS.

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.

Clinical evidence of active/on-going significant increased intracranial pressure (i.e., impending herniation) or uncontrolled seizures.

Prior receipt of a chimeric antigen receptor (CAR)-based therapy.

Trial design

Design model

Single group

Treatments tested in this trial

  • GPC2-CAR T cells

    Biological/Vaccine

    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.

Treatment groups

18 Participants
are divided into 1 treatment group
Group A: GPC2-CAR T Cell TherapyExperimental treatment 3 interventions

Trial outcomes

Primary outcomes

1

Manufacturing Feasibility

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.

Time frame
Up to 6 months post-leukapheresis
2

Incidence of Dose Limiting Toxicities (DLTs)

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

Time frame
Within first 28-day treatment cycle per dose level

Secondary outcomes

1

Objective Response Rate (ORR)

Percentage of subjects achieving a protocol-defined response (complete response, partial response, or stable disease) based on imaging and neurological exam.

Time frame
Up to 2 years post-infusion
2

Progression-Free Survival (PFS)

Time from enrollment to radiographic disease progression.

Time frame
Up to 2 years
3

Overall Survival (OS)

Time from initial diagnosis to death from any cause.

Time frame
Up to 2 years

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Stanford University

Lead sponsor

Source ClinicalTrials.gov
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