Study of Safety and Effectiveness of Autologous BCMA-targeted CAR-T Cell Therapy for Relapsed or Refractory Light Chain Amyloidosis

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 treatments effects and safety.

Autologous BCMA-targeted CAR-T Cell Injection for Relapsed/Refractory Light Chain Amyloidosis