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)
Novartis Pharmaceuticals
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)
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 rapcabt…
Eligibility
- Age range
- 18–75 years
- Sex
- All
- Healthy volunteers
- No
Key Inclusion Criteria: 1. Men and women, aged ≥ 18 and ≤75 years, with a diagnosis of probable or definite myositis according to American College of Rheumatology/European League Against Rheumatism 2017 (ACR/EULAR 2017) criteria 2. Participants who had inadequate response to prior therapy 3. Diagnosed with active disease 4. Participant must meet criteria for severe myositis Key Exclusion Criteria: 1. Any condition during Screening that could prevent a complete washout of medications or could otherwise make the participant ineligible for anti-CD19 CAR-T therapy and further participation in t…
Interventions
- BiologicalRapcabtagene autoleucel
Single infusion of rapcabtagene autoleucel after lymphodepleting therapy with fludarabine (adjusted based on renal impairment) and cyclophosphamide daily for 3 days.
- OtherActive Comparator Option
Investigator choice of treatment as per protocol
Locations (55)
- University Of AlabamaBirmingham, Alabama
- FL Medical Clinic Orlando HealthZephyrhills, Florida
- Northwestern UniversityChicago, Illinois
- Northwestern UniversityChicago, Illinois
- University Of IowaIowa City, Iowa
- LDS HospitalSalt Lake City, Utah