Phase I Trial of GD2.CART Cells Augmented With Constitutive Interleukin-7 Receptor for Treatment of High-Grade Pediatric CNS Tumors.
Frank Y Lin, Austin Stuckert, Candise Tat...
https://pubmed.ncbi.nlm.nih.gov/38771986Actively Recruiting
Phase 1
Age: 12Months - 25Years
All Genders
ID04099797
Phase I Study of Autologous T Lymphocytes Expressing GD2-specific Chimeric Antigen and Constitutively Active IL-7 Receptors for Treatment of GD2-expressing Brain Tumors (GAIL-B)
Led by Baylor College of Medicine · Updated on 2026-04-27
56
Participants Needed
1
Research Sites
730 weeks
Total Duration
On this page
Sponsors
B
Baylor College of Medicine
Lead Sponsor
C
Center for Cell and Gene Therapy, Baylor College of Medicine
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating a new gene therapy for patients aged 12 months to 25 years with certain aggressive brain tumors that express a protein called GD2. These tumors include diffuse midline glioma, diffuse intrinsic pontine glioma, medulloblastoma, and other rare high-grade gliomas. The study aims to find the highest safe dose of genetically modified immune cells, called GD2-C7R CAR T cells, that can be given both intravenously and directly into the brain. This gene therapy builds on previous research that combined antibodies and T cells to better target and kill cancer cells, especially by improving how long the T cells survive in the body. The treatment involves collecting the patient’s own T cells and genetically modifying them with two genes: one to help the cells recognize and attack GD2-positive cancer cells and another to help the cells survive longer. Patients first receive chemotherapy to prepare their body for the T cells, followed by an intravenous infusion of GD2-C7R T cells. Subsequent treatments involve infusions directly into the brain through a catheter called an Ommaya reservoir or a programmable VP shunt. The dosage for brain infusions is adjusted through multiple cycles up to 24 times, with monitoring after each infusion to ensure safety. Participants will undergo regular medical exams, blood tests, brain imaging, and spinal fluid collection to monitor the treatment’s effects and how long modified T cells remain in the body. Follow-up visits continue for up to 15 years to track any long-term effects of the gene therapy. Additional tumor samples may be collected if available to support research. Patients will be closely observed for side effects, including during hospital stays after each infusion, and supportive care will be provided as needed throughout the study.
CONDITIONS
Brief Title
C7R-GD2.CAR T Cells for Patients With GD2-expressing Brain Tumors (GAIL-B)
Who Can Participate
Age: 12Months - 25Years
All Genders
Eligibility Criteria
You may qualify if you...
- Histologically confirmed GD2-expressing brain tumors including diffuse midline glioma, diffuse intrinsic pontine glioma, medulloblastoma, or other high-grade CNS tumors
- Tumors less than 5 cm in maximum dimension or surgically reduced if between 5 and 5.5 cm
- Measurable disease on at least two dimensions on MRI
- Age between 12 months and 25 years
- Karnofsky/Lansky functional score of 50 or higher (60 or higher for pontine glioma)
- Central line and Ommaya reservoir or programmable VP shunt in place or planned (central line may be omitted for cycles without lymphodepletion)
- Completed standard radiation therapy at least 4 weeks prior
- Stable neurological exam for 7 days prior to enrollment
- Stable or decreasing steroid dose with maximum dexamethasone 0.1 mg/kg/day
- Adequate organ function including ANC > 1000 cells/ul, platelet count > 100,000 cells/ul, bilirubin < 1.5x upper limit, ALT and AST < 5x upper limit, and creatinine within 2x upper limit for age
You will not qualify if you...
- Pregnancy or breastfeeding
- Received other immunotherapy within 42 days before investigational agent
- Received colony-stimulating factors within 14 days before lymphodepletion
- Receiving any concurrent anti-cancer therapy within three half-lives prior
- Any condition deemed by investigator to pose risk with investigational agent administration
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - Up to 24 cycles with each cycle separated by at least 4 weeks
Participants receive chemotherapy drugs cyclophosphamide and fludarabine (or clofarabine) by IV for 3 days to prepare the body for T cell infusion. They then receive an intravenous infusion of GD2-C7R T cells, followed by intracerebroventricular (ICV) infusions of GD2-C7R T cells through an Ommaya reservoir or programmable VP shunt. Participants are monitored in the hospital for at least 3 to 5 days after each infusion to watch for side effects and may receive up to 24 additional treatment cycles if eligible.
Hospital stays of at least 3 to 5 days after each infusion, with follow-up visits during treatment
Follow-up
Duration - 15 years
Participants are followed for a total of 15 years after treatment to monitor for long-term side effects and disease status. Follow-up includes regular visits for physical exams, blood tests, MRI imaging, spinal fluid tests, and neurological evaluations.
Visits at weeks 1, 2, 3, 4, 6, 8; months 3, 6, 9, 12; twice a year for 4 years; then annually for 10 years
Trial Site Locations
Total: 1 location
1
Texas Children's Hospital
Houston, Texas, United States, 77030
Actively Recruiting
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Research Team
J
Jasia Mahdi, MD
D
David Allen
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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