A Phase I/II Open-label, Single-arm, Multicenter Clinical Study to Evaluate the Safety and Efficacy of FKC289 in Subjects With Relapsed/Refractory Primary AL Amyloidosis
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 32
- 试验地点
- 11
- 主要终点
- Dose-Limiting Toxicity (DLT)
研究概览
简要总结
This Phase I study is designed to evaluate the safety and efficacy of FKC289 in subjects with relapsed/refractory primary light chain (AL) amyloidosis.The primary objectives are to assess the safety of FKC289 and to establish the recommended Phase II dose (RP2D). And this Phase II study is designed to evaluate the efficacy of FKC289.The primary objectives are to assess proportion of subjects with hematologically best complete response (CR) within 6 months.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Voluntarily signed the informed consent form (ICF);
- •Aged between 18-75 years old (inclusive);
- •Confirmed clinical diagnosis of primary light chain (AL) amyloidosis;
- •Relapsed or refractory disease;
- •dFLC ≥ 40 mg/L (based on central laboratory test results);
- •ECOG performance status of 0 to 2;
- •No active systemic infection within 2 weeks prior to leukapheresis;
- •Adequate bone marrow and major organ function (including hepatic, renal, cardiac, and pulmonary);
排除标准
- •Presence of any clinically significant cardiovascular disease within 6 months prior to screening;
- •Clinically significant central nervous system (CNS) disease or pathological abnormality;
- •History of solid organ transplantation (e.g., heart, lung, kidney, liver);
- •Presence of complete intestinal obstruction;
- •Moderate to severe pleural effusion or ascites that is difficult to control with conventional treatment and requires continuous catheter drainage;
- •History of malignancy within 5 years prior to signing the ICF
研究组 & 干预措施
FKC289 injection
干预措施: FKC289 infusion (Drug)
结局指标
主要结局
Dose-Limiting Toxicity (DLT)
时间窗: Within 28 days after FKC289 infusion
Number of subjects experiencing DLT
Adverse Events (AEs) and Serious Adverse Events (SAEs)
时间窗: Within 24 months after FKC289 infusion
Number of subjects experiencing AEs and SAEs
Replication Competent Lentivirus (RCL)
时间窗: Within 24 months after FKC289 infusion
Number of subjects with positive detection of RCL
次要结局
- Hematologic complete response rate (CRR)(Within 6 months,12 months, and 24 months after FKC289 infusion)
- Hematologic objective response rate (ORR)(Within 6 months,12 months, and 24 months after FKC289 infusion)
- Pharmacokinetics (PK)(Within 24 months after FKC289 infusion)
- Pharmacodynamics (PD)(Within 24 months after FKC289 infusion)