NCT06655896

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

A Phase II, Multi-part, Randomized, Open-label, Assessor-blinded, Active-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Rapcabtagene Autoleucel Versus Rituximab Treatment in Participants With Severe Refractory Diffuse Cutaneous Systemic Sclerosis

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 96 (estimated)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the efficacy, safety and tolerability of rapcabtagene autoleucel (administered once following lymphodepletion) in participants with severe refractory diffuse cutaneous systemic sclerosis relative to rituximab.

Detailed description

This is a phase 2, multi-part, five-year, randomized, open-label, assessor-blinded, multicenter study to evaluate the efficacy and safety of rapcabtagene autoleucel versus rituximab in participants with severe refractory diffuse cutaneous systemic sclerosis (dcSSc). This study comprises two cohorts: * A Lead-in Cohort enrolling participants to receive rapcabtagene autoleucel. * A Randomized Cohort enrolling participants to receive rapcabtagene autoleucel or rituximab. Participants in the rituximab arm whose disease is not fully controlled may receive rapcabtagene autoleucel treatment once the participant is confirmed to be eligible per protocol. After end of study, participants who received rapcabtagene autoleucel infusion will enter a long-term follow-up (LTFU) period after rapcabtagene autoleucel infusion. This LTFU will be described in a separate study protocol.

Conditions

• Scleroderma, Diffuse

Interventions

Type	Name	Description
BIOLOGICAL	rapcabtagene autoleucel	single infusion of rapcabtagene autoleucel after lymphodepleting therapy with fludarabine (adjusted based on renal impairment) and cyclophosphamide daily for 3 days.
BIOLOGICAL	rituximab	rituximab intravenous infusion (i.v.) as per protocol

Timeline

• Start date: 2024-10-29
• Primary completion: 2028-11-30
• Completion: 2032-08-30
• First posted: 2024-10-24
• Last updated: 2026-02-20

Locations

80 sites across 18 countries: United States, Australia, Austria, Brazil, Czechia, Denmark, France, Germany, Hungary, Israel, Italy, Japan, Netherlands, Singapore, South Korea, Spain, Switzerland, Taiwan

Regulatory

• FDA-regulated drug study