FKC288 for Relapsed or Refractory Systemic Light Chain (AL) Amyloidosis
ConditionLight Chain Amyloidosis
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18-70
SponsorNanjing University School of Medicine
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
The subject must personally sign a written informed consent form approved by the ethics committee before the start of the study;
The subject's age is ≥18 years old and <70 years old;
The subject must be diagnosed with light chain amyloidosis by pathological examination, with at least one major organ involved (heart, kidney, or liver);
The subject with recurrent/refractory light chain amyloidosis that achieved no response with conventional treatment;
Disqualifiers
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.
Subjects with central metastasis or complete intestinal obstruction.
Subject with moderate or more severe hydrothorax and ascites which are hard to control by conventional treatment and require continuous catheter drainage.
With an active malignant tumor in the past 5 years, unless it is a curable tumor and has been obviously cured.
Trial design
Design model
Single group
Treatments tested in this trial
FKC288
DrugAdministration of FKC288 Four dose groups of 0.1×10\^6 CAR-T/kg, 0.3×10\^6 CAR-T/kg, 1.0×10\^6 CAR-T/kg, and 3.0×10\^6 CAR-T/kg FKC288 are designed in this study. 3 to 6 subjects are expected to be enrolled in each dose group according to observed DLT.
Treatment groups
12 Participants
are divided into 1 treatment groupGroup A: Treatment armExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
The proportion of subjects with dose-limiting toxicity
The number of participants with dose limiting toxicity in each dose group and the type of dose limiting toxicity that occurred.
Time frame
Within 28 days after FKC288 injection infusion2
The proportion of subjects with adverse events
All adverse events were evaluated according to NCI-CTCAE v5.0 criteria.
Time frame
Within 24 weeks after FKC288 injection infusionSecondary outcomes
1
Proportion of subjects achieving hematologic response
The proportion of subjects who achieve any type of hematologic response according to the 2023 National Comprehensive Cancer Network (NCCN) systemic light-chain amyloidosis guidelines within 6 months after receiving FKC288 infusion.
Time frame
Within 6 months after FKC288 injection infusion2
Proportion of subjects achieving organ response
The proportion of subjects who achieve any type of organ response according to the 2023 National Comprehensive Cancer Network (NCCN) systemic light-chain amyloidosis guidelines within 2 years after receiving FKC288 infusion.
Time frame
Within 2 years after FKC288 injection infusion3
Progression-free survival (PFS) of all subjects
Time from the first FKC288 infusion to the first assessment of disease progression/relapse or death from any cause.
Time frame
Within 2 years after FKC288 injection infusion4
Overall survival (OS) of all subjects
Time from the first FKC288 infusion to death from any cause.
Time frame
Within 2 years after FKC288 injection infusionSponsors and contacts
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Source ClinicalTrials.gov