NCT06665256

Phase 2 Study of Rapcabtagene Autoleucel in Myositis

A Phase 2, Randomized, Open-label, Controlled Study to Evaluate the Efficacy and Safety of Rapcabtagene Autoleucel Versus Comparator in Participants With Severe Refractory Idiopathic Inflammatory Myopathies (IIM)

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 123 (estimated)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

A Phase 2, randomized, open-label, controlled study to evaluate the efficacy and safety of rapcabtagene autoleucel versus comparator in participants with severe refractory idiopathic inflammatory myopathies (IIM)

Detailed description

This is a Phase 2, randomized, active-controlled study. This study comprises two cohorts: * A lead-in cohort enrolling participants to receive rapcabtagene autoleucel * A randomized cohort with participants receiving either rapcabtagene autoleucel or a comparator option. Participants in the comparator arm whose signs and symptoms are not fully controlled may receive rapcabtagene autoleucel treatment once the participant is confirmed to be eligible After end of study (EOS), 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

• Idiopathic Inflammatory Myopathies

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.
OTHER	Active Comparator Option	Investigator choice of treatment as per protocol

Timeline

• Start date: 2024-12-17
• Primary completion: 2029-07-17
• Completion: 2030-07-17
• First posted: 2024-10-30
• Last updated: 2026-02-05

Locations

55 sites across 13 countries: United States, France, Germany, Israel, Italy, Japan, Netherlands, Saudi Arabia, Singapore, Spain, Switzerland, Taiwan, United Kingdom

Regulatory

• FDA-regulated drug study