Study Description
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) 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.
Interventions
Active Comparator Option
Rapcabtagene autoleucel
Eligibility Criteria
Key Inclusion Criteria:
1. 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
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 the study
2. BMI at Screening of ≤18.5 or ≥35 kg/m2
3. Severe muscle damage at Screening
4. Inadequate organ function
5. Hypersensitivity and/or contraindications to any product (including its ingredients) to be given to the participant as per the study protocol
6. Other inflammatory and non-inflammatory myopathies
7. 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.
Novartis Investigative Site
Recruiting
Haifa,3109601,Israel
Novartis Investigative Site
Recruiting
Ramat Gan,52621,Israel
Novartis Investigative Site
Recruiting
Sapporo city,Hokkaido,060 8648,Japan
University Of Iowa
Recruiting
Iowa City,Iowa,52242,United States
Hanna Zembrzuska
Worldwide Contacts
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