Actively Recruiting
Safety and Efficacy of CAR-T Cell Therapy for Relapsed/refractory Neuroblastoma and Desmoplastic Small Round Cell Tumors: a Single-arm, Open-label Trial.
Led by Sun Yat-Sen University Cancer Center · Updated on 2025-02-26
10
Participants Needed
3
Research Sites
156 weeks
Total Duration
On this page
Sponsors
S
Sun Yat-Sen University Cancer Center
Lead Sponsor
Y
Yake Biotechnology Ltd.
Collaborating Sponsor
AI-Summary
What this Trial Is About
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;
CONDITIONS
Official 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.
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients diagnosed with relapsed or refractory neuroblastoma or desmoplastic small round cell tumors
- Age between 1 and 50 years, any gender
- Willing to participate and able to provide written informed consent
- Expected survival of at least 12 weeks
- Karnofsky performance status (for patients 16 years or older) or Lansky performance status (for patients under 16 years) of at least 50
- Good major organ function including:
- Liver: ALT no more than 5 times the upper normal limit and bilirubin no more than 2.0 mg/dL (exceptions for Gilbert-Meulengracht syndrome apply)
- Kidney: Plasma creatinine no more than 1.5 times the upper normal limit or estimated glomerular filtration rate (eGFR) at least 60 mL/min/1.73m²
- Lung: Oxygen saturation at least 95% in room air
- Heart: Left ventricular ejection fraction at least 45%
- Steroid treatments stopped at least 2 weeks before CAR-T infusion, except physiological replacement doses
- Immunosuppressive drugs stopped at least 4 weeks before enrollment
- Anti-proliferative treatments other than lymphodepleting chemotherapy stopped at least 2 weeks before infusion
- CNS disease prophylaxis stopped 1 week before CAR-T infusion
- Use of reliable contraception for patients of childbearing potential until 12 months after infusion and until two tests confirm no CAR-T cells
- If patient cannot provide suitable T cells, donor T cells may be used
You will not qualify if you...
- Increased intracranial pressure or altered consciousness
- Radiation therapy within 2 weeks before infusion
- Active hepatitis B (HBV DNA > 500 IU/mL) or hepatitis C (HCV RNA positive)
- HIV positive or positive syphilis test
- Uncontrolled acute life-threatening infections within 72 hours before infusion
- Unstable angina or myocardial infarction within 6 months before screening
- History or presence of other malignancies except adequately treated basal cell or squamous cell carcinoma, carcinoma in situ of cervix or breast with no recurrence for at least 3 years, or completely resected primary malignant tumors in remission for at least 5 years
- Pregnant or breastfeeding women
- Uncontrolled arrhythmias not managed medically
- Need for oral anticoagulation therapy within 1 week before CAR-T infusion
- Active neuroautoimmune or inflammatory diseases such as Guillain-Barré syndrome or amyotrophic lateral sclerosis
- Other conditions considered unsuitable by the investigator for study participation
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 3 locations
1
Sun Yat-sen University Cancer Center
Guangzhou, Guangdong, China, 510060
Actively Recruiting
2
Dongguan Taixin Hospital
Dongguan, Guang, China, 523125
Actively Recruiting
3
Shanghai YaKe Biotechnology Ltd.
Shanghai, Shanghai Municipality, China, 200438
Actively Recruiting
Research Team
Y
Yizhuo Zhang, PhD
CONTACT
S
Suying Lu, PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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