Overview
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, two-year, randomized, assessor- blinded, 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.
After end of study (EOS), participants who received rapcabtagene autoleucel infusion will enter a long-term follow-up (LTFU) period lasting up to 15 years after rapcabtagene autoleucel infusion. This LTFU will be described in a separate study protocol.
Eligibility
Key Inclusion Criteria:
- Men and women, aged >18 and ≤65 years, with a diagnosis of probable or definite myositis according to American College of Rheumatology/European League Against Rheumatism 2017 (ACR/EULAR 2017) criteria
- Participants who had inadequate response to prior therapy
- Diagnosed with active disease
- Participant must meet criteria for severe myositis
Key Exclusion Criteria:
- 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 the study
- BMI at Screening of ≤18.5 or ≥35 kg/m2
- Severe muscle damage at Screening
- Inadequate organ function
- Hypersensitivity and/or contraindications to any product (including its ingredients) to be given to the participant as per the study protocol
- Other inflammatory and non-inflammatory myopathies
- Any medical conditions that are not related to IIM that would jeopardize the ability of the participant to tolerate CD19 CAR-T cell therapy
Other protocol-defined inclusion/exclusion criteria may apply.