A Clinical Study on Organoid-Based Drug Sensitivity-Guided Chemotherapy for High-Risk, Relapsed, and Refractory Pediatric Soft Tissue Malignant Tumors
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 发起方
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- objective response rate (ORR)
研究概览
简要总结
This is a prospective, open-label, single-arm, single-center study designed to evaluate the feasibility of using patient-derived organoid drug sensitivity analysis to guide chemotherapy for pediatric patients with refractory and relapsed malignant soft tissue tumors. The primary endpoint of the study is objective response rate (ORR), and the secondary endpoint is event-free survival (EFS).
详细描述
This is a prospective, open-label, single-arm, single-center study with a planned total enrollment of 30 pediatric patients with refractory and relapsed malignant soft tissue tumors. Patients will be enrolled in this clinical trial after providing informed consent and meeting the inclusion and exclusion criteria. As this is a single-arm study without a control group, all patients will be assigned to the experimental arm. Treatment regimens will be comprehensively evaluated and adjusted based on the results of patient-derived organoid drug sensitivity testing in combination with clinical response. Clinical efficacy will be assessed during follow-up to evaluate the feasibility of using organoid drug sensitivity analysis to guide chemotherapy for refractory and relapsed malignant soft tissue tumors in children.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Year 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age between 1 and 18 years, gender unrestricted.
- •Patients with histopathologically confirmed rhabdomyosarcoma, Ewing sarcoma, malignant rhabdoid tumor, or non-rhabdomyosarcoma soft tissue sarcoma.
- •Previous receipt of at least one line of systemic anti-tumor therapy with subsequent disease progression.
- •Presence of measurable target lesions according to RECIST 1.1 criteria.
- •Sufficient biopsy tissue can be obtained from either metastatic or primary lesions.
- •ECOG performance status score of 0 to
- •Expected overall survival ≥ 6 months.
- •Adequate bone marrow reserve: HB ≥ 90 g/L; ANC ≥ 1.5×10⁹/L; PLT ≥ 80×10⁹/L.
- •Adequate hepatic and renal function: total bilirubin ≤ 1.5 × upper limit of normal (ULN); ALT and AST ≤ 2.5 × ULN; serum creatinine ≤ ULN.
- •Voluntary participation in the study with signed written informed consent form (ICF), good compliance, and willingness to comply with follow-up procedures.
排除标准
- •Patients with peripheral nervous system disorders caused by the disease, or a history of significant psychiatric or central nervous system disorders.
- •Patients with severe infection or active peptic ulcer requiring treatment.
- •Patients currently participating in other clinical trials or who have participated within the past 3 weeks.
- •Patients deemed ineligible by the investigator for any other reason.
研究组 & 干预措施
Treatment group relying on organoid drug sensitivity results
Treatment regimens will be comprehensively evaluated and adjusted based on the results of patient-derived organoid drug sensitivity testing in combination with clinical response.
干预措施: treatment based on the results of drug sensitivity analysis (Drug)
结局指标
主要结局
objective response rate (ORR)
时间窗: from the first drug administration up to two years
ORR is the proportion of patients with best response of complete response (CR) and partial response (PR) according to Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 and iRECIST.
次要结局
- Event-Free Survival (EFS)(from the first drug administration up to two years)
研究者
Dapeng Jiang
Shanghai Jiao Tong University School of Medicine
Shanghai Jiao Tong University School of Medicine