Actively Recruiting
An Exploratory Clinical Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Autologous BCMA-targeted CAR-T Cell Injection in Participants With Relapsed/Refractory Light Chain Amyloidosis
Led by Beijing Boren Hospital · Updated on 2026-06-04
30
Participants Needed
1
Research Sites
104 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying relapsed or refractory systemic light chain amyloidosis (AL amyloidosis), a disease where abnormal protein deposits affect organs like the kidney and heart. The trial focuses on evaluating the safety and preliminary effectiveness of a new treatment called targeted BCMA autologous CAR-T cell injection. The study also explores how the treatment behaves in the body, including its pharmacokinetics, pharmacodynamics, and immune response after infusion. Participants receive a fixed dose of BCMA-targeted CAR-T cells derived from their own immune cells. Before infusion, they undergo lymphodepleting conditioning with drugs such as fludarabine and cyclophosphamide for three days to prepare the body. The CAR-T cells are then given as a single intravenous injection on Day 0. The study includes a safety observation period after infusion and a main follow-up lasting up to two years, with possible long-term follow-up for up to 15 years. During the study, participants undergo various assessments including evaluations of safety events, treatment responses, and blood tests to monitor CAR-T cell levels and viral vector presence. Follow-up visits occur frequently after infusion and then every 90 days. Researchers measure outcomes like adverse events, response rates, survival, and pharmacokinetic parameters. The total participation can last several years with ongoing monitoring to gather detailed information about the treatment's effects and safety.
CONDITIONS
Brief Title
Autologous BCMA-targeted CAR-T Cell Injection for Relapsed/Refractory Light Chain Amyloidosis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Signed informed consent approved by ethics committee.
- Age 18 years or older.
- Pathologically confirmed light chain amyloidosis.
- Relapsed or refractory light chain amyloidosis previously treated with two or more therapy lines.
- Difference in free light chains (dFLC) greater than 50 mg/L.
- Expected survival of at least 12 weeks.
- ECOG performance status score of 2 or less.
- Diagnosis confirmed by clinical, laboratory, imaging, and biopsy criteria with immunoglobulin light chain involvement.
- Female participants of childbearing potential agree to use effective contraception from consent until 365 days after infusion.
- Adequate organ function meeting specified blood counts, liver function, oxygen saturation, coagulation, and disease stage requirements.
You will not qualify if you...
- Prior gene therapy before enrollment.
- Live vaccine within 4 weeks before enrollment.
- Other interventional study drug treatment within 12 weeks before leukapheresis.
- Central nervous system involvement or complete intestinal obstruction.
- Moderate or higher pleural effusion or ascites requiring continuous catheter drainage.
- Active malignancy in past 5 years unless cured.
- Positive tests for hepatitis B, hepatitis C, HIV, CMV, or syphilis.
- Uncontrolled active infection except mild urogenital or upper respiratory infections.
- Severe heart disease including recent heart attack, severe heart failure, or serious arrhythmias.
- Uncontrolled hypertension.
- Unrecovered toxicity from prior treatment except alopecia and minor lab abnormalities.
- Major surgery within 2 weeks before enrollment or planned major surgery during study period except local anesthesia.
- Solid organ transplantation.
- Pregnant or breastfeeding women.
- History of central nervous system disease or psychiatric disorders.
- Other unstable systemic diseases requiring medication.
- Known severe allergic reaction or intolerance to CAR-T product or components.
- Bleeding or severe thrombosis conditions or current thrombolytic/anticoagulant therapy.
- Any other conditions deemed unsuitable by the investigator.
AI-Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Approximately 1 to 2 weeks
Participants undergo PBMC leukapheresis and pre-lymphodepletion examinations to prepare for the conditioning and infusion process.
1 to 2 visits (in-person)
Duration - 3 days
Participants receive lymphodepleting chemotherapy with fludarabine plus cyclophosphamide for three consecutive days before the CAR-T cell infusion.
3 daily visits (in-person)
Duration - 1 day
Participants receive a single intravenous infusion of targeted BCMA autologous CAR-T cells on Day 0.
1 infusion visit (in-person)
Duration - Up to 2 years (Day 1 to Day 720)
Participants are monitored for safety and efficacy from the day of infusion until completion of the 2-year main follow-up period or until criteria for exiting the main follow-up are met.
Visits at Day 28, Day 60, Day 90, and every 90 days thereafter until Day 720
Duration - Up to 15 years
Participants who complete the main follow-up or exit early continue to be monitored long-term for up to 15 years after infusion.
Visits as scheduled during long-term monitoring
Trial Site Locations
Total: 1 location
1
Beijing GoBroad Boren Hospital
Beijing, Fengtai District, China, 100070
Actively Recruiting
Research Team
Y
Yajing Zhang
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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