Actively Recruiting
GPC2-CAR T Cell Therapy for Relapsed or Refractory Medulloblastoma in Children and Young Adults
Led by Stanford University · Updated on 2026-01-27
18
Participants Needed
1
Research Sites
100 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
GPC2-CAR T Cell Therapy for Relapsed or Refractory Medulloblastoma in Children and Young Adults
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Histologically confirmed medulloblastoma or primary CNS embryonal tumor as defined by 2021 CNS WHO Classification
- Includes ETMR, Pineoblastoma, ATRT of CNS, CNS neuroblastoma FOXR2-activated, or CNS Embryonal Tumor NOS
- History of relapsed or recurrent disease after initial curative treatment or failure of standard therapy
- Tumor sample with GPC2 expression H-score ≥ 100 by immunohistochemistry
- Evaluable disease by imaging or positive cerebrospinal fluid cytology within 28 days before enrollment
- Programmable ventriculo-peritoneal shunt device if present
- No limit on prior treatments; prior toxicities must be stable or recovered to grade 1 or less (except non-significant toxicities)
- Therapy washout periods met before apheresis: at least 6 weeks from craniospinal radiation, 14 days from small volume radiotherapy, 21 days or 5 half-lives from systemic therapy (except immune checkpoint therapy requiring 5 half-lives), 28 days from bevacizumab, 30 days from investigational drugs, and 12 weeks from immune checkpoint therapy
- Age between 12 months and 30 years at enrollment; first 3 treated subjects must be at least 3 years old
- Performance status: Karnofsky ≥ 60% if ≥ 16 years old; Lansky ≥ 60% or ECOG ≤ 2 if younger than 16
- Adequate organ and marrow function with specific laboratory criteria
- Negative pregnancy test for females of childbearing potential
- Willingness to use contraception during study and for 4 months after preparative regimen
- Ability to provide informed consent or have legal representative consent; assent obtained when appropriate
You will not qualify if you...
- Metastatic disease outside the central nervous system
- Unable or unwilling to have a cerebrospinal fluid reservoir placed (unless pre-existing suitable device)
- Active significant increased intracranial pressure or uncontrolled seizures
- Prior CAR-based therapy
- Current anticoagulation therapy
- Known HIV or active hepatitis B or C infection (except undetectable viral load)
- Pregnancy or breastfeeding
- Known allergy to study agents
- History of other malignancy unless treated over 5 years ago with good prognosis
- Primary immunodeficiency or autoimmune disease causing organ injury or requiring immunosuppression within 2 years
- Recent significant cardiac disease within 12 months
- Significant uncontrolled medical conditions increasing risk, including uncontrolled diabetes, COPD, pulmonary fibrosis, inflammatory disorders, immunodeficiency, renal failure, or liver dysfunction
- Inability of subject or caregivers to comply with study procedures
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Lucile Packard Children's Hospital Stanford
Palo Alto, California, United States, 94304
Actively Recruiting
Research Team
M
Mariah Duncan
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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