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

Recruiting now · Phase 1

Conditions studied: 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

In brief

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 facts

Study ID

NCT07087002

Run by

Stanford University

People needed

18

Starts

2025-08-28

Expected to finish

2027-08-01

Last updated by the study team

2026-01-27

Who can join

Age: 1 and older, up to 30. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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
• 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.
• 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.
• Evaluable Disease: Evaluable disease as per radiographic findings and/or positive cerebrospinal fluid cytology within 28 days of enrollment.
• 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.
• 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).
• At time of enrollment, subjects are on track to meet the required therapy wash out period(s) prior to apheresis.
• a. At least 6 weeks following craniospinal radiation therapy. i. At least 14 days wash-out needed following small volume radiotherapy (i.e., Stereotactic Radiosurgery (SRS)).
• b. At least 21 days or 5 half-lives (whichever is shorter) must have elapsed since any prior systemic therapy, except for systemic inhibitory/stimulatory immune checkpoint therapy, which requires 5 half-lives.
• c. At least 28 days following bevacizumab treatment. d. At least 30 days following any investigational drug. e. At least 12 weeks following systemic inhibitory or stimulatory immune checkpoint therapy.
• Age: ≥ 12 months to ≤ 30 years of age at time of enrollment The first 3 subjects treated with GPC2-CAR T cells must be ≥ 3 years old at time of infusion
• Performance Status: Subjects ≥ 16 years of age must have Karnofsky ≥ 60%. Subjects < 16 years of age must have Lansky scale 60%; or ECOG performance status ≤ 2 (see Section 11.3).
• Normal Organ and Marrow Function [supportive care is allowed per institutional standards, i.e., filgrastim, transfusion]
• Hemoglobin ≥ 8 g/dL
• Absolute Neutrophil Count (ANC) ≥ 1,000/μL
• Platelet count ≥ 75,000/μL, with no platelet transfusion within 96 hours prior to enrollment
• Absolute lymphocyte count (ALC) ≥ 150/μL
• PT/INR, PTT ≤ 1.5 x ULN for age
• Adequate renal, hepatic, cardiac, and pulmonary function defined as:

You may not qualify if…

• 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.
• Currently receiving anticoagulation therapy.
• 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.
• Pregnancy or breastfeeding in a postpartum female.
• Known sensitivity or allergy to any agents/reagents used in this study.
• 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.
• Primary immunodeficiency or history of autoimmune disease (e.g., Crohn's, rheumatoid arthritis, systemic lupus) resulting in end organ injury or requiring systemic immunosuppression/systemic disease modifying agents within the last 2 years.
• History of myocardial infarction, cardiac angioplasty or stenting, unstable angina, or other clinically significant cardiac disease within 12 months of enrollment.
• Significant medical diseases or poorly controlled conditions that, in the judgement of the investigator, put the subject at an unacceptable risk of complications, including but not limited to: uncontrolled diabetes mellitus, chronic obstructive pulmonary disease, pulmonary fibrosis, clinically significant inflammatory disorders, immunodeficiency (e.g., HIV infection), immunocompromised for reasons other than malignancy (e.g., chronic corticosteroid therapy or other immunosuppressive therapy), renal failure including patients requiring dialysis, or clinically significant liver dysfunction.
• In the Investigator's judgment, the subject or parents/caregivers (as required) will not be able to comply with the study procedures outlined in the study protocol including follow-up visits.

Where it is running

• Lucile Packard Children's Hospital Stanford — Palo Alto, California, United States (enrolling)

Full record on ClinicalTrials.gov

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