Autologous BCMA-targeted CAR-T Cell Injection for Relapsed/Refractory Light Chain Amyloidosis
China30 participantsStarted 2024-12-16
Plain-language summary
Systemic light chain amyloidosis (AL amyloidosis) is the most common type of systemic amyloidosis, with diverse clinical manifestations and difficulties in diagnosis and treatment. AL amyloidosis may involve multiple organs; the kidney and heart are the most commonly involved organs. The treatment goal is to reduce monoclonal immunoglobulin light-chain levels, prevent further amyloid deposition in important organs, and alleviate or reverse organ dysfunction caused by amyloid deposition. The principal approach to achieve this goal is to eliminate the plasma-cell or B-cell clones producing abnormal light chains. For patients with relapsed/refractory AL amyloidosis, the protocol states that there is currently no suitable treatment method and that participation in clinical trials is recommended.
This study evaluates targeted BCMA autologous CART cell injection in participants with relapsed/refractory light chain amyloidosis. The main purpose is to evaluate safety, preliminarily verify efficacy, and explore in vivo pharmacokinetics, pharmacodynamics, immunogenicity and related characteristics after infusion.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* The participant must personally sign an ethics-committee-approved informed consent form before study start.
* Age ≥18 years.
* Pathologically confirmed light chain amyloidosis.
* Relapsed/refractory light chain amyloidosis previously treated with 2 or more lines of therapy.
* dFLC \>50 mg/L.
* Expected survival ≥12 weeks.
* ECOG score ≤2.
* Diagnosis of AL amyloidosis must meet the following conditions: (1) clinical manifestations, physical examination, laboratory or imaging examinations confirm tissue or organ involvement; (2) tissue biopsy pathology confirms amyloid deposition, and the precursor protein of amyloid protein is immunoglobulin light chain or heavy and light chain; and the participant is relapsed/refractory.
* Female participants of childbearing potential should agree to use effective contraception from the date of signing informed consent until 365 days after infusion. Effective contraception is defined as abstinence or contraception using methods specified in the protocol with an annual failure rate \<1%.
* Adequate organ function before enrollment, meeting all of the following:
* Absolute neutrophil count ≥1.0×10\^9/L; granulocyte colony-stimulating factor (G-CSF) support is permitted.
* Platelet count ≥50×10\^9/L.
* Hemoglobin ≥8 g/dL.
* Bilirubin ≤1.5×upper limit of normal (ULN), except biliary obstruction caused by tumor compression.
* ALT or AST ≤2.5×ULN; for participants with liver involvement, ≤5×ULN.
* Mayo 2004 stag…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of Participants With Adverse Events, Serious Adverse Events, and Adverse Events of Special Interest
Timeframe: From signing of the informed consent form through withdrawal from the study or 2 years after BCMA-targeted autologous CAR-T cell infusion, whichever occurs first.