Actively Recruiting
Phase I Clinical Trial of GPC2 Chimeric Antigen Receptor T Cells 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
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating a new approach using GPC2-targeted chimeric antigen receptor (CAR) T cells to treat children and young adults with relapsed or refractory medulloblastoma and other similar central nervous system embryonal tumors. This Phase 1 clinical trial focuses on the safety, feasibility, and initial effects of this treatment delivered directly into the brain's ventricles. The study is led by Stanford University and includes participants aged 1 to 30 years with confirmed diagnoses and specific tumor markers. Participants will first undergo leukapheresis to collect their immune cells, which are then modified to produce GPC2-CAR T cells. Before receiving these cells, patients undergo lymphodepleting chemotherapy with fludarabine and cyclophosphamide. Afterward, they receive up to eight intracerebroventricular infusions of the modified cells every 28 days, with doses gradually increased within each patient. This treatment schedule follows a set dose escalation strategy to monitor patient response and safety. During the trial, participants will be closely monitored for manufacturing success of the CAR T cells and any dose-limiting toxicities within the first treatment cycle. Additional assessments include imaging and cerebrospinal fluid tests to evaluate disease status. Researchers will also track objective response rates, progression-free survival, and overall survival for up to two years after infusion. Safety and organ function tests, performance status evaluations, and pregnancy testing are part of ongoing monitoring throughout the study period.
CONDITIONS
Brief 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 diagnosis of medulloblastoma or other primary CNS embryonal tumor as per 2021 CNS WHO Classification
- History of relapsed or refractory disease after initial curative treatment
- Tumor positive for GPC2 with H-score ≥ 100 by immunohistochemistry
- Evaluable disease by radiographic findings or positive cerebrospinal fluid cytology within 28 days
- Programmable ventriculo-peritoneal (VP) shunt if VP shunt device is present
- Stable or recovered toxicities from prior therapies to Grade 1 or less
- At least 6 weeks since craniospinal radiation or 14 days since small volume radiotherapy
- At least 21 days or 5 half-lives since prior systemic therapy, with exceptions for immune checkpoint therapy
- At least 28 days since bevacizumab treatment and 30 days since investigational drug
- At least 12 weeks since systemic immune checkpoint therapy
- Age between 1 and 30 years at enrollment; first 3 subjects must be at least 3 years old at infusion
- Karnofsky score ≥ 60% for participants 16 years or older; Lansky score ≥ 60% or ECOG ≤ 2 if younger than 16
- Adequate organ and marrow function including specified blood counts and organ tests
- Negative pregnancy test for females of childbearing potential
- Willingness to use birth control during and after treatment
- Ability to provide informed consent or have legal representative consent with assent as 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 already having a suitable device
- Active or uncontrolled increased intracranial pressure or seizures
- Prior receipt of CAR-based therapy
- Currently on anticoagulation therapy
- Known HIV infection or active hepatitis B or C infection unless viral load is undetectable
- Pregnancy or breastfeeding
- Known allergy to any agents used in the study
- 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
- Significant cardiac disease within 12 months
- Other serious medical conditions that increase risk or interfere with study compliance
- Inability to follow study procedures or attend follow-up visits
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Approximately 1 week
Participants undergo leukapheresis to collect blood cells for manufacturing CAR T cells, followed by lymphodepleting chemotherapy with fludarabine and cyclophosphamide prior to treatment.
1 visit for leukapheresis and 3 days of chemotherapy visits
Duration - Up to 8 cycles of 28 days each
Participants receive up to 8 doses of intracerebroventricular GPC2-CAR T cell infusions every 28 days using a dose escalation strategy.
Up to 8 infusion visits every 28 days
Duration - Up to 2 years
Participants are monitored for safety and treatment response after the final CAR T cell infusion, including survival and disease progression.
Regular follow-up visits scheduled over 2 years
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
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here