Relapsed or Refractory Systemic Light Chain (AL) Amyloidosis
Conditions
Interventions
Single arm:FKC289 Injection
Sponsors
ZhongShan Hospital Fudan University
Eligibility
Sex/Gender
All
Age
18 Years to 75 Years
Inclusion criteria
Inclusion criteria: Main Inclusion Criteria: 1. The subject must personally sign the written informed consent form approved by the Ethics Committee before the study begins. 2. The subject‘s age is >= 18 years old and = 40 mg/L 6. Expected survival>= 12 weeks 7. ECOG score<= 2 points 8. Female subjects of childbearing potential must agree to use effective contraception from the date of signing the informed consent form until 365 days after the infusion. An effective method of contraception is defined as abstinence or contraceptive methods with an annual failure rate of <1% specified in the plan. 9. Subjects must have adequate organ function at screening.
Exclusion criteria
Exclusion criteria: Main Exclusion Criteria: 1.Subjects with the following treatments prior to enrollment: 1.1Previous gene therapy prior to enrollment. 1.2Administration of live vaccines within 4 weeks before enrollment. 1.3Participation in another interventional clinical trial with investigational drugs within 12 weeks before apheresis. 2.Subjects with central nervous system metastasis or complete intestinal obstruction. 3.Moderate or severe pleural/ascites effusion requiring persistent indwelling catheter drainage despite conventional treatment. 4.With an active malignancy within the past 5 years, unless it is a curable tumor and has been obviously cured. 5.Subjects who are hepatitis B surface antigen (HBsAg)-positive or hepatitis B core antibody (HBcAb)-positive with abnormal peripheral blood HBV DNA (abnormal HBV DNA is defined as: quantitative HBV DNA above the lower limit of detection or above the normal reference range of the testing center, or qualitative HBV DNA-positive); hepatitis C virus (HCV) antibody-positive with detectable peripheral blood HCV RNA; human immunodeficiency virus (HIV) antibody-positive; cytomegalovirus (CMV) DNA-positive; or syphilis RPR-positive. 6.Uncontrolled active infections(except forIII - Severe arrhythmias (excluding those with pacemakers or ICDs) 8.Uncontrolled hypertension despite medical therapy. 9.Prior treatment-related toxicities that have not resolved to baseline or <= Grade 1 (per NCI-CTCAE v5.0, except for alopecia and clinically insignificant lab abnormalities). 10.Major surgery within 2 weeks before enrollment, or planned surgery during the waiting period before infusion or within 12 weeks after study treatment (except planned surgery under local anesthesia). 11.Subjects with a history of solid organ transplantation. 12. Women who are pregnant or breastfeeding. 13.Subjects with a history of central nervous system (CNS) disorders (e.g., cerebral aneurysm, epilepsy, stroke, dementia, psychiatric disorders) or impaired consciousness. 14.Other unstable systemic diseases as judged by the investigator, including but not limited to severe liver, kidney, or metabolic disorders requiring medical intervention. 15.Known life-threatening allergic reactions, hypersensitivity, or intolerance to FKC289 cell therapy or its components. 16.Subjects judged by the investigator to have active bleeding or severe thrombosis, or have inherited/acquired bleeding and severe thrombosis (including hemophilia, coagulation dysfunction, thrombocytopenia, hypersplenism, etc.), or those receiving thrombolytic or anticoagulant therapy. 17.Any other conditions considered by the investigator to make the subject unsuitable for participation.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Types and incidence rates of dose-limiting toxicities (DLT) in each dose cohort;Types and incidence rates of adverse events(AEs) and serious adverse events(SAEs) each dose cohort; | — |
Secondary
| Measure | Time frame |
|---|---|
| Safety Endpoints: Abnormalities in safety assessments, including laboratory tests, vital signs, physical examinations, electrocardiograms (ECG), etc.;Efficacy Endpoints-Objective Response Rate (ORR):The proportion of subjects achieving a best overall response of partial response (PR) or complete response (CR) within 6 months after FKC289 infusion, as assessed per the 2023 National Comprehensive Cancer Network (NCCN) Guidelines and Response Criteria for Systemic Light Chain (AL) Amyloidosis.; | — |
Countries
China
Contacts
Public ContactPeng Liu
ZhongShan Hospital Fudan University
Outcome results
None listed