Phase 2 Study of Rapcabtagene Autoleucel in Myositis
Purpose
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)
Condition
- Idiopathic Inflammatory Myopathies
Eligibility
- Eligible Ages
- Between 18 Years and 75 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- 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
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 2. BMI at Screening of ≤17 or ≥40 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.
Study Design
- Phase
- Phase 2
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental Rapcabtagene autoleucel |
Single infusion of rapcabtagene autoleucel (YTB323) |
|
|
Active Comparator Comparator |
Investigator choice of treatment as per protocol |
|
Recruiting Locations
Birmingham 4049979, Alabama 4829764 35294
More Details
- Status
- Recruiting
- Sponsor
- Novartis Pharmaceuticals
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.