San Pablo

Researchers are evaluating rapcabtagene autoleucel, a biological treatment given as a single infusion after lymphodepletion, in adults with active, refractory systemic lupus erythematosus (SLE) or active, refractory lupus nephritis (LN). This Phase 2, open-label study aims to assess both the effectiveness and safety of this therapy in patients who have not responded well to at least two previous treatments. Participants must meet specific criteria including positive autoantibody tests and active disease symptoms measured by the SLEDAI-2K score. Participants will receive one infusion of rapcabtagene autoleucel following preparatory lymphodepletion therapy. The study does not mention comparator groups, focusing on monitoring responses to this single treatment. Treatment effects will be evaluated at 24 and 52 weeks after infusion to understand how well the therapy works over time. Throughout the study, participants will be closely monitored with various assessments to evaluate disease activity, safety, and response to treatment. Researchers will use laboratory tests, including autoantibody levels, and clinical evaluations to track changes in disease status. Safety monitoring will continue during the study period to identify any adverse effects. The total participation duration covers at least 52 weeks to capture both short-term and longer-term outcomes of the therapy.

Researchers are evaluating the safety, tolerability, and effectiveness of rapcabtagene autoleucel compared to rituximab in adults with severe refractory diffuse cutaneous systemic sclerosis (dcSSc). This phase 2, randomized, open-label, assessor-blinded study includes participants whose disease is progressive and difficult to treat. The study aims to assess treatment response after one year, using specific criteria to measure improvement in systemic sclerosis symptoms. The study involves two main groups: a lead-in cohort receiving rapcabtagene autoleucel and a randomized cohort receiving either rapcabtagene autoleucel or rituximab. Rapcabtagene autoleucel is given as a single infusion after a three-day lymphodepleting therapy with fludarabine and cyclophosphamide. Rituximab is administered intravenously according to protocol. Participants in the rituximab group whose disease is not controlled may later receive rapcabtagene autoleucel. After treatment, those who received rapcabtagene autoleucel enter a long-term follow-up phase detailed in a separate protocol. Participants will be monitored over five years with assessments including treatment response evaluation at week 52. Researchers will track safety, tolerability, and disease progression throughout the study. The study includes multiple centers and uses blinded assessors to ensure objective evaluation. This long-term follow-up allows for ongoing observation of participants who received the investigational treatment, with safety and efficacy measures collected during the entire study period.