Using GD2-CAR T Cells to Treat Neuroblastoma

An Open Clinical Study on the Safety and Efficacy of Autologous GD2-CAR T Cells in the Treatment of Relapsed Refractory Neuroblastoma

This study is testing whether a new treatment using GD2-CAR T cells can help patients with neuroblastoma, especially those whose cancer has come back or hasn’t responded to other treatments.

Quick facts

• Phase: Early Phase 1
• Study type: Interventional
• Enrollment: 30 (estimated)
• Ages: 1 Year to 18 Years
• Sex: All
• Sponsor: The General Hospital of Western Theater Command (Academic / other)
• Drugs / interventions: chemotherapy, CAR-T, immunotherapy, CAR T
• Locations: 1 site (Chengdu, Sichuan)
• Trial ID: NCT06684639 on ClinicalTrials.gov

What this trial studies

This study investigates the efficacy of GD2-CAR T cell therapy in patients with neuroblastoma, particularly those with relapsed or refractory cases. Participants will receive escalating doses of GD2-CAR T cells, starting from a low dose and increasing based on tolerance. The study aims to evaluate the complete and partial response rates, as well as long-term outcomes such as progression-free survival and overall survival. Adverse events will be monitored for up to 24 months post-infusion to assess safety and tolerability.

Who should consider this trial

Good fit: Ideal candidates are children aged 1 to 18 years diagnosed with recurrent or refractory neuroblastoma that expresses the GD2 antigen.

Not a fit: Patients with active infections or those whose health conditions do not meet the study's eligibility criteria may not benefit from this study.

Why it matters

Potential benefit: If successful, this therapy could provide a new treatment option for patients with high-risk neuroblastoma who have limited alternatives.

How similar studies have performed: Other studies utilizing CAR T cell therapy for various cancers have shown promising results, suggesting potential success for this novel approach in neuroblastoma.

Eligibility criteria

Inclusion Criteria:

1. All cases were diagnosed as neuroblastoma with positive expression of GD2 antigen in tumor cells. Informed consent of patient or guardian.
2. Diagnosis of recurrent/refractory neuroblastoma.
3. At least 2 weeks or 5 half-lives (whichever is shorter) from the beginning of preconditioning chemotherapy after prior systemic treatment.
4. Toxic reactions caused by previous antitumor therapy must be stabilized and restored to ≤ grade 1.
5. Over 1 years old, under 18 years old.
6. Physical strength score 0-3 (ECOG standard).
7. No obvious active infection.
8. Expected survival ≥3 months
9. Adequate kidney, liver, lung and heart function, defined as creatinine clearance (estimated by the Cockcroft Gault formula) \> 60 mL/min; Serum ALT/AST ≤ 2.5 ULN; Total bilirubin ≤1.5 ULN, excluding subjects with Gilbert's syndrome; Cardiac ejection fraction ≥ 50%, echocardiography confirmed centropericardial effusion, and ECG showed no clinically significant abnormal findings. There was no clinically significant pleural effusion. Baseline blood oxygen saturation under indoor ventilation was \> 92%.
10. The serum pregnancy test results of fertile women must be negative (women who have undergone surgical sterilization or at least 2 years after menopause are considered to be infertile).

Exclusion Criteria:

1. The subject has had other malignancies, non-melanoma skin tumors, carcinoma in situ (e.g. Cervix, bladder, breast), unless disease-free survival of at least 3 years.
2. There is an uncontrollable infection, including fungal, bacterial, viral or other.
3. Known human immunodeficiency virus (HIV) infection.
4. Known history of hepatitis B (HBsAg positive) or hepatitis C (HCV antibody positive). Subjects with latent or prehepatitis B infection (defined as HBcAb positive and HBsAg negative) can be enrolled only if PCR tests for HBV DNA are negative. In addition, these subjects were required to undergo a monthly PCR test for HBV DNA. Participants who are serologically positive for HCV antibodies can also be enrolled if their PCR test results for HCV RNA are negative.
5. Existing or past CNS disease, such as seizures, cerebrovascular ischemia/bleeding, dementia, cerebellar disease, or any CNS-related autoimmune disease.
6. Serious heart disease, such as uncontrolled or symptomatic arrhythmia, congestive heart failure, or myocardial infarction within 6 months prior to screening, or any grade 3 (moderate) or 4 (severe) heart disease (according to the New York Heart Society Functional Grading Method NYHA).
7. A history of myocardial infarction, angioplasty or stent placement, unstable angina pectoris, or other clinically significant heart disease in the 12 months prior to enrollment.
8. Any medical condition that may affect the evaluation of safety or efficacy.
9. Have had severe rapid hypersensitivity reactions to any of the drugs to be used in this study.
10. Live vaccine should be administered within ≤6 weeks before starting the pretreatment regimen.
11. Pregnant or lactating female subjects.
12. Male or female subjects who do not consent to effective contraception from the time they sign informed consent until 6 months after completing immune cell therapy.
13. Subjects judged by the investigator had difficulty in completing all visits or procedures required by the study protocol (including follow-up visits), or were not compliant enough to participate in the study.

Where this trial is running

Chengdu, Sichuan

• The General Hospital of Western Theater Command — Chengdu, Sichuan, China (Recruiting)

Study contacts

• Principal investigator: Hai Yi, Ph.D — The General Hospital of Western Theater Command
• Study coordinator: JunXia Wang, Ph.D

Conditions Neuroblastoma; Relapsed; Refractory