Safety and Efficacy of CAR-T Cell Therapy for Relapsed/refractory Neuroblastoma and Desmoplastic … (NCT06836505) | Clinical Trial Compass
RecruitingPhase 1/2
Safety and Efficacy of CAR-T Cell Therapy for Relapsed/refractory Neuroblastoma and Desmoplastic Small Round Cell Tumors: a Single-arm, Open-label Trial.
China10 participantsStarted 2024-12-12
Plain-language summary
Title: Safety and efficacy of CAR-T cell therapy for relapsed/refractory neuroblastoma and desmoplastic small round cell tumors: a single-arm, open-label trial.
The CART used in this study will be provided by Shanghai YaKe Biotechnology Ltd.
Aims:
1. To evaluate the safety and efficacy of GD2/B7H3 CAR-T therapy for relapsed/refractory neuroblastoma, and observe its pharmacokinetic/pharmacodynamic characteristics and the survival of CAR-T cells in relapsed/refractory neuroblastoma patients.
2. To evaluate the safety and efficacy of GD2/B7H3 CAR-T therapy for relapsed/refractory desmoplastic small round cell tumor, and observe its pharmacokinetic/pharmacodynamic characteristics and the survival of CAR-T cells in desmoplastic small round cell tumor patients.
Patients: Relapsed/refractory neuroblastoma; Relapsed/refractory desmoplastic small round cell tumor.
CAR-T therapy: Lymphodepletion treatment will be performed within 14 days prior to CAR-T cell infusion: intravenous chemotherapy based on fludarabine 25mg/m² and cyclophosphamide 500mg/m² for 1 to 3 days. CAR-T cells will then be infused intravenously, with a dosage of 1.00 to 10.00 × 10⁶/kg of CAR-positive T cells.
Research period: CAR-T cell infusion will be followed up for one year, or until adverse events resolve, progression occurs, or the patient transitions to other treatments.
Outcome measures:
Incidence of adverse events related to CAR-T therapy, as well as their intensity and duration; Pharmacokinetic/pharmacodynamic characteristics of CAR-T in patients and the survival of CAR-T cells.
Overall response rate (ORR) after CAR-T cell infusion, including complete response (CR) and partial response (PR); Overall survival (OS), progression-free survival (PFS), event-free survival (EFS), time to progression (TTP), and duration of response (DOR) after CAR-T cell infusion;
Who can participate
Age range1 Year – 50 Years
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. Patients who are diagnosed as relapsed/refractory neuroblastoma or relapsed/refractory desmoplastic small round cell tumors;
✓. Age 1-50 years, any gender;
✓. Agree to participate in the trial and sign a written informed consent form;
✓. Expected survival of ≥12 weeks;
✓. Karnofsky performance status (for patients ≥16 years) or Lansky performance status (for patients \<16 years) (Appendix 1) must be at least 50;
✓. Good function of major organs:
✓. Liver function: ALT ≤ 5 times the upper limit of normal for the corresponding age, and bilirubin ≤ 2.0 mg/dL, except for patients with Gilbert-Meulengracht syndrome. Patients with Gilbert-Meulengracht syndrome who have bilirubin ≤ 3.0 times the upper limit of normal and direct bilirubin ≤ 1.5 times the upper limit of normal may be included;
✓. Renal function: Plasma creatinine ≤ 1.5 times the upper limit of normal, or estimated glomerular filtration rate (eGFR) ≥ 60 mL/min/1.73m²;
Exclusion criteria
What they're measuring
1
To evaluate the safety of CAR-T cells in relapsed/refractory neuroblastoma and desmoplastic small round cell tumor patients.
Timeframe: From enrollment to the end of treatment at 1 year
✕. Patients with increased intracranial pressure or altered consciousness;
✕. Patients who have received radiation therapy within 2 weeks prior to infusion;
✕. Patients with active hepatitis B (defined as HBV DNA \> 500 IU/mL) or hepatitis C (HCV RNA positive);
✕. HIV-positive patients or patients with a positive syphilis test;
✕. Patients with uncontrolled acute life-threatening bacterial, viral, or fungal infections (e.g., positive blood cultures within ≤72 hours before infusion);
✕. Patients with unstable angina and/or myocardial infarction within 6 months prior to screening;
✕. Patients with a history of or concurrent malignancies, except for the following conditions:
✕. Basal cell carcinoma or squamous cell carcinoma that has been adequately treated (sufficient wound healing required before study enrollment);