Phase 1 Study of Zamto-cel CAR-T Therapy for Severe Refractory Autoimmune Diseases Including Lupus and Systemic Sclerosis

What this Trial Is About

Researchers are evaluating the safety and tolerability of zamtocabtagene autoleucel zamto-cel, an investigational chimeric antigen receptor T-cell CAR-T therapy, in adults with severe refractory autoimmune diseases including systemic lupus erythematosus SLE, lupus nephritis LN, and systemic sclerosis SScdcSSc who have not responded adequately to standard therapies. This Phase 1, multi-cohort, dose-finding study aims to determine safe dose levels and gather preliminary data on the therapys effects in patients with these conditions. Participants will undergo leukapheresis to collect their T cells, which are then genetically engineered to target CD19 and CD20 antigens. Before receiving the fresh CAR-T cell infusion intravenously at one of two dose levels, subjects will be treated with lymphodepleting chemotherapy using cyclophosphamide and fludarabine. The study will enroll patients in small groups to carefully assess safety before expanding to recommended Phase 2 dosing. Monitoring will continue for up to one year post-infusion, and longer-term follow-up is planned under a separate protocol. Throughout the study, participants will be assessed for adverse events, dose-limiting toxicities, and various immune-related side effects. Researchers will evaluate disease activity at multiple time points using disease-specific measures, track the persistence and behavior of the infused cells, and assess health-related quality of life. Safety and response will be closely monitored, with follow-up visits and tests scheduled up to one year after treatment to gather comprehensive data on the therapys impact and tolerability.

US Zamto-cel Autoimmune Diseases