Actively Recruiting
Phase I Study of Anti-GD2 CAR T Cells in Pediatric and Young Adult Patients with Relapsed/Refractory CNS Tumors
Led by Bambino Gesù Hospital and Research Institute · Updated on 2025-02-05
54
Participants Needed
1
Research Sites
574 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and feasibility of a new treatment called iC9-GD2-CAR T-cells in children and young adults with relapsed or refractory malignant central nervous system tumors. This Phase I study focuses on patients with different types of brain tumors, including medulloblastoma, high-grade gliomas, and other rare CNS tumors. The treatment includes a safety feature called a suicide gene to help manage possible side effects. The treatment involves collecting the patient's own T cells through leukapheresis, then modifying them to target GD2, a marker on tumor cells. Before receiving the modified cells, patients undergo chemotherapy to reduce immune cells temporarily (lymphodepleting regimen). Afterwards, patients receive a single intravenous infusion of the engineered CAR T cells at a dose ranging from 1.0 to 6.0 x 10^6 cells per kg, depending on their tumor type. The study has three groups based on tumor classification and risk, which helps test safety sequentially. Participants will be closely monitored for safety and response, including imaging studies, blood tests, and clinical assessments. They will be followed actively for 5 years to track disease status and treatment effects, with an additional 15-year follow-up to meet gene therapy regulations. Key measures include safety evaluation, CAR T cell persistence and expansion, tumor response, and survival outcomes. The study aims to define the maximum tolerated dose and recommended dose of this investigational therapy.
CONDITIONS
Brief Title
GD2-CAR T Cells for Pediatric Brain Tumours
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Imaging assessments performed within 14 days before starting treatment
- Age between 6 months and 30 years
- Measurable or evaluable disease on at least two dimensions on MRI at enrollment
- Karnofsky or Lansky performance score of 60 or higher
- Recovery from toxic effects of prior treatments to grade 2 or less; clinically stable if chronic complications present
- Placement of an implantable intraventricular access device and microdialysis probe
- Signed informed consent from patient or legal guardian; assent for children 7 years or older when appropriate
- Willingness to use birth control during study and for 4 months after preparative regimen if of childbearing potential
- Negative pregnancy test for females of childbearing potential
You will not qualify if you...
- Pregnant or lactating women
- Severe, uncontrolled active infections
- HIV, active hepatitis B or C infection
- Rapidly progressing disease with life expectancy under 6 weeks
- History of severe hypersensitivity to murine protein products
- Liver dysfunction with bilirubin over 4 times normal or transaminases over 6 times normal
- Kidney dysfunction with creatinine over 3 times normal
- Blood oxygen saturation below 90%
- Cardiac ejection fraction below 45%
- Low neutrophil count (ANC under 500/mm3) or low platelets (under 20,000 not due to transfusion)
- Congestive heart failure, arrhythmia, psychiatric illness, or social situations limiting study compliance
- Recent therapies before infusion including unstable glucocorticoid use, chemotherapy within 3 weeks, immunosuppressives within 30 days, radiation less than 6 weeks prior, or investigational anti-cancer agents within 30 days
- Failure in production of GD2-CAR T cells due to low vitality, low CAR+ cell percentage, insufficient anti-tumor activity, contamination, or other manufacturing issues
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.
Duration - Single dose infusion with lymphodepleting chemotherapy prior
Participants receive a lymphodepleting chemotherapy regimen followed by a single intravenous infusion of GD2-CAR T cells targeting their CNS tumors.
1 lymphodepleting chemotherapy period and 1 infusion visit (in-person)
Duration - Up to 5 years
Participants are actively followed for safety and disease status for up to 5 years after CAR T cell infusion, including monitoring of treatment effects and tumor response.
Regular visits for assessments during 5-year follow-up
Trial Site Locations
Total: 1 location
1
Ospedale Pediatrico Bambino Gesù
Roma, Italy, Italy, 00165
Actively Recruiting
Research Team
F
Francesca Del Bufalo, MD
A
Angela Mastronuzzi, MD, PhD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
3
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