Phase 2 Study Comparing Rapcabtagene Autoleucel and Rituximab for Severe Refractory Diffuse Cutaneous Systemic Sclerosis

What this Trial Is About

The trial investigates rapcabtagene autoleucel compared to rituximab for participants with severe refractory diffuse cutaneous systemic sclerosis dcSSc. This phase 2, randomized, open-label, assessor-blinded study aims to evaluate efficacy and safety over five years, focusing on participants with progressive and severe disease who meet specific classification criteria. The study includes two cohorts a lead-in group receiving rapcabtagene autoleucel and a randomized group receiving either rapcabtagene autoleucel or rituximab, with possible crossover to rapcabtagene autoleucel if rituximab is insufficient. Participants receive a single infusion of rapcabtagene autoleucel following lymphodepleting therapy with fludarabine and cyclophosphamide for three days, or intravenous rituximab according to protocol. After the active treatment phase, participants who received rapcabtagene autoleucel enter a long-term follow-up period described in a separate protocol. Treatment aims to address severe systemic sclerosis manifestations including lung, skin, and cardiac involvement. During the study, participants undergo assessments such as the Revised Composite Response Index in Systemic Sclerosis 50 rCRISS50 at 52 weeks, lung function tests, skin scoring, and disability questionnaires. Safety is monitored throughout the study and up to approximately five years for adverse events. The total study duration allows for detailed evaluation of treatment response, safety, and tolerability in this population aged 18 to 70 years.

Phase 2 Study Evaluating Rapcabtagene Autoleucel in Participants With Diffuse Cutaneous Systemic Sclerosis