Actively Recruiting
Phase 1 Trial of Autologous GD2 CAR T Cells for H3K27M-mutant Diffuse Midline Glioma
Led by Stanford University · Updated on 2026-01-26
97
Participants Needed
1
Research Sites
782 weeks
Total Duration
On this page
Sponsors
S
Stanford University
Lead Sponsor
C
California Institute for Regenerative Medicine (CIRM)
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating the use of GD2 chimeric antigen receptor (CAR) T cells (GD2CART) in children and adults with H3K27M-mutant diffuse midline glioma (DMG), including diffuse intrinsic pontine glioma (DIPG) and tumors in the spinal cord or other brain regions. This phase 1 clinical trial aims to test whether these specially modified immune cells can be successfully made and safely given to patients with this aggressive brain tumor. The study also seeks to identify the best dose, route, and schedule for administering GD2CART, along with lymphodepleting chemotherapy. Participants receive GD2CART through different methods depending on the study group. Some receive the treatment intravenously after chemotherapy with cyclophosphamide and fludarabine, while others receive it directly into the brain's ventricles (intracerebroventricularly) with or without prior chemotherapy. Dose levels vary by group and are carefully escalated to find the maximum tolerated dose. The chemotherapy regimens may also include rituximab for certain groups. Treatment is given on Day 0 in a hospital setting. Throughout the trial, participants undergo various assessments including safety monitoring up to 28 days after infusion, measurement of successful production of GD2CART cells, and evaluation of tumor response through imaging at multiple time points up to 24 months post-treatment. Researchers also measure overall survival, progression-free survival, and patient-reported outcomes. If serious side effects occur, a special agent (AP1903) may be used to clear the engineered cells. The study requires ongoing follow-up and monitoring to assess long-term effects and treatment impact.
CONDITIONS
Brief Title
GD2 CAR T Cells in Diffuse Intrinsic Pontine Gliomas (DIPG) & Spinal Diffuse Midline Glioma(DMG)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of H3K27M mutant diffuse midline glioma confirmed by CLIA test
- Age between 2 and 60 years
- At least 4 weeks since completion of standard radiation therapy
- At least 3 weeks since last chemotherapy or 5 half-lives, except immune checkpoint therapy requires 3 months washout
- Karnofsky score ≥ 60% (if >16 years) or Lansky score ≥ 60% (if ≤16 years)
- Adequate organ and marrow function including ANC ≥ 1000/uL, platelets ≥ 100,000/uL, hemoglobin ≥ 8 g/dL, normal kidney, liver, heart, and lung function
- Negative pregnancy test for females of childbearing potential
- Willingness to use contraception during and 4 months after treatment
- Ability to give informed consent or have a legal guardian consent
You will not qualify if you...
- Bulky tumor involvement in cerebellar vermis or hemispheres or thalamic lesions posing high herniation risk
- Severe swallowing or medullary dysfunction or high risk of respiratory compromise
- Current systemic corticosteroid therapy above replacement levels
- Use of unapproved dietary supplements, alternative therapies, or medications
- Prior CAR T cell therapy or recent immunomodulatory therapy except checkpoint inhibitors with 3 months washout
- Uncontrolled infections including HIV, active Hepatitis B or C
- Significant systemic illness interfering with safety or efficacy assessment
- Pregnant or breastfeeding women
- Known allergy to agents used in the study
- Primary immunodeficiency or autoimmune disease requiring recent systemic immunosuppression
- Inability to comply with study visits or procedures and follow-up
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 - Single treatment day with pre-treatment chemotherapy days -6 to 0
Participants receive lymphodepleting chemotherapy followed by infusion of autologous GD2 CAR T cells administered intravenously or intracerebroventricularly depending on study arm.
Hospitalization for treatment on Day 0 with chemotherapy visits on Days -6 to -2
Duration - Up to 24 months post-infusion
Participants are monitored for safety, clinical benefit, and patient-reported outcomes after GD2 CAR T cell infusion.
Visits on Day 28, 3, 6, 9, 12, and 24 months post-infusion
Trial Site Locations
Total: 1 location
1
Lucile Packard Children's Hospital (LPCH)
Stanford, California, United States, 94304
Actively Recruiting
Research Team
A
Ashley Jacobs, RN, BSN
M
Monica Reddy
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
4
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