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
Trial Snapshot
- Phase
- Phase 1
- Status
- Not yet recruiting
- Sponsor
- Enrollment
- 32
- Locations
- 11
- Primary Endpoint
- Dose-Limiting Toxicity (DLT)
Study Overview
Brief Summary
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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Sequential
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to 75 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •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);
Exclusion Criteria
- •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
Arms & Interventions
FKC289 injection
Intervention: FKC289 infusion (Drug)
Outcomes
Primary Outcomes
Dose-Limiting Toxicity (DLT)
Time Frame: Within 28 days after FKC289 infusion
Number of subjects experiencing DLT
Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame: Within 24 months after FKC289 infusion
Number of subjects experiencing AEs and SAEs
Replication Competent Lentivirus (RCL)
Time Frame: Within 24 months after FKC289 infusion
Number of subjects with positive detection of RCL
Secondary Outcomes
- 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)