Study of Anti-CD19 CAR T-Cell Therapy for Adults with Severe Refractory Systemic Autoimmune Diseases Including Lupus, Scleroderma, DermatomyositisPolymyositis, and ANCA-Associated Vasculitis

What this Trial Is About

Researchers are investigating the use of anti-CD19 CAR T-cell therapy as a new treatment approach for serious systemic autoimmune diseases that are difficult to treat, including Systemic Lupus Erythematosus SLE, Systemic Sclerosis SSc, DermatomyositisPolymyositis DMPM, and ANCA Associated Vasculitis AAV. These conditions involve harmful B cell activity and often do not respond well to current treatments, leading to severe health risks. This open-label, phase I basket study aims to assess the safety, feasibility, and early effectiveness of this therapy by monitoring adverse effects, infection rates, and disease response over 24 weeks in eight participants. Participants will receive a single intravenous infusion of an investigational product called CD19-CARLenti, which consists of their own T cells modified to target CD19-positive B cells. The T cells are collected through leukapheresis and processed using specialized equipment before infusion. The study includes pre-treatment procedures such as lymphodepletion, with all treatments and visits conducted at specialized hospital departments. The therapy targets a common disease mechanism across the four autoimmune conditions included in this basket trial. During the study, participants will have regular evaluations to monitor for side effects like cytokine release syndrome and neurotoxicity within the first 4 weeks, as well as infections and blood cell changes over the full 24-week period. Researchers will also measure how long the CAR T cells and B cell depletion persist in the blood and track changes in disease-related antibodies. The primary outcomes include safety events and overall disease response at 24 weeks. Participants will be closely followed through outpatient visits and laboratory tests throughout the study period.

Anti-CD19 CAR T-Cell Therapy in Refractory Systemic Autoimmune Diseases