Overview

FKC288 for Relapsed or Refractory Systemic Light Chain (AL) Amyloidosis

Status:
Recruiting
Trial end date:
2026-06-01
Target enrollment:
0
Participant gender:
All
Summary
This study is a single-center exploratory clinical trial. It is estimated that 6-12 subjects will be enrolled. The "BOIN" dose escalation design is adopted. The main purpose is to evaluate the safety of FKC288 in the treatment of subjects with relapsed or refractory AL amyloidosis and explore the recommended phase II dose of FKC288 in the treatment of patients with relapsed/refractory systemic Light Chain (AL) amyloidosis.
Phase:
Phase 1
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Nanjing University School of Medicine
Criteria
Inclusion Criteria:

1. The subject must personally sign a written informed consent form approved by the
ethics committee before the start of the study;

2. The subject's age is ≥18 years old and <70 years old;

3. The subject must be diagnosed with light chain amyloidosis by pathological
examination, with at least one major organ involved (heart, kidney, or liver);

4. The subject with recurrent/refractory light chain amyloidosis that achieved no
response with conventional treatment;

5. dFLC > 50mg/L;

6. Expected survival ≥ 12 weeks;

7. ECOG score ≤ 2 points;

8. Female subjects with fertility should agree to practice an effective method of
contraception from the day of signing the ICF 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. Before enrollment, the subject must have appropriate organ function and meet all the
following criteria:

1) Absolute neutrophil count ≥ 1.0×109/L (use of granulocyte colony-stimulating factor
(G-CSF) support is allowed, but must be without supportive treatment within 7 days before
the examination); 2) Platelet count ≥ 75×109/L (no transfusion support [including component
transfusion] or treatments aimed at raising platelets such as thrombopoietin [TPO] should
be received within 7 days before the examination); 3) Hemoglobin ≥ 9 g/dl (no transfusion
support [including component transfusion] should be received within 7 days before the
examination); 4) Bilirubin value ≤ 1.5× upper limit of normal (ULN) (except bile duct
obstruction caused by tumor compression); 5) Creatinine clearance rate ≥ 40 ml/min; 6) ALT
or AST ≤ 2.5× ULN (≤5 times the upper limit of normal in patients with liver involvement);
7) Echocardiography results indicate left ventricular ejection fraction ≥ 50% with no
significant pericardial effusion; 8) NTproBNP < 1800pg/ml, TNT < 0.06ng/ml; 9) Stable
coagulation function: INR ≤ 1.5, APTT ≤ 1.2× ULN (excluding tumor-related anticoagulant
therapy); 10) >95% basic blood oxygen saturation in the natural indoor air environments.

Exclusion Criteria:

1. Subjects who have received any of the following treatments prior to enrollment: 1)
Subjects who have received gene therapy before enrollment; 2) Subjects who have
received live vaccines within 4 weeks prior to enrollment; 3) Subjects has received
other interventional clinical research drugs within 12 weeks before apheresis.

2. Subjects with central metastasis or complete intestinal obstruction.

3. Subject with moderate or more severe hydrothorax and ascites which are hard to control
by conventional treatment and require continuous catheter drainage.

4. With an active malignant tumor in the past 5 years, unless it is a curable tumor and
has been obviously cured.

5. Subjects who are positive for hepatitis B surface antigen (HBsAg) or hepatitis B core
antibody (HBcAb) and have abnormal peripheral blood HBV DNA test results (HBV DNA test
abnormality is defined as HBV DNA quantitative detection is higher than the detection
center's detection lower limit or higher than the detection center's normal reference
range or HBV DNA qualitative detection is positive); hepatitis C virus (HCV) antibody
positive and peripheral blood hepatitis C virus (HCV) RNA positive; human
immunodeficiency virus (HIV) antibody positive; the cytomegalovirus (CMV) DNA
positive; syphilis testing RPR positive.

6. Presence of uncontrollable active infections (excluding respiratory tract infections).

7. Severe cardiovascular diseases, including but not limited to unstable angina pectoris,
myocardial infarction (within 6 months prior to screening), congestive heart failure
(New York Heart Association [NYHA] classification ≥ III), and severe arrhythmias.

8. Subjects with hypertension that cannot be controlled by medication.

9. Toxicity reactions that have not been relieved to baseline or ≤ grade 1 (NCI-CTCAE
version 5.0, except for hair loss and laboratory test abnormalities without clinical
significance) from past treatments.

10. Major surgery within 2 weeks before enrollment, or has a surgery planned during the
time the subject is expected to be infused with FKC288 or within 12 weeks after FKC288
infusion (except planned surgery under local anesthesia).

11. Subject who has a solid organ transplant.

12. Women who are pregnant or breastfeeding.

13. Subjects with previous central nervous system diseases (such as cerebral aneurysm,
epilepsy, stroke, dementia, psychosis, etc.) or conscious disorders.

14. Other systemic diseases that the investigator judges as unstable, including but not
limited to severe liver, kidney, or metabolic diseases that require medication.

15. Known life-threatening allergic reactions, hypersensitivity reactions, or intolerance
to FKC288 cell preparations or their components.

16. Subjects judged by the investigator to have bleeding or severe thrombosis, or have
inherited/acquired bleeding and severe thrombosis (including hemophilia, coagulation
dysfunction, thrombocytopenia, splenomegaly, etc.), or are receiving thrombolysis or
anticoagulation therapy.

17. Other situations deemed inappropriate for inclusion by the investigator.