Phase 2 Study Evaluating Rapcabtagene Autoleucel in Participants With Severe Active GPA or MPA
Purpose
The purpose of this study is to evaluate the efficacy and safety of rapcabtagene autoleucel versus comparator in participants with severe active Granulomatosis with Polyangiitis (GPA) or Microscopic Polyangiitis (MPA)
Condition
- ANCA Associated Vasculitis (AAV)
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 GPA or MPA according to the American College of Rheumatology/ European League Against Rheumatism 2022 (ACR/EULAR 2022) classification criteria 2. Positive test for ANCA-autoantibodies 3. GPA and MPA participants with severe active disease
Exclusion Criteria
- Any condition 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. Hypersensitivity and/or contraindications to any product to be given to the participant as part of the study protocol 3. Other systemic autoimmune diseases requiring therapy 4. Any medical conditions that are not related to GPA/MPA that would jeopardize the ability of the participant to tolerate CD19 CAR-T cell therapy 5. Inadequate organ function
Study Design
- Phase
- Phase 2
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Single (Outcomes Assessor)
- Masking Description
- The study investigator and the participant will be unblinded to the study treatment. A blinded assessor will perform the efficacy assessments to minimize bias in data collection.
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental Rapcabtagene autoleucel |
Single infusion of rapcabtagene autoleucel (YTB323) and concomitant glucocorticoids as per protocol |
|
|
Active Comparator Active comparator |
Comparator and concomitant glucocorticoids as per protocol |
|
Recruiting Locations
Aurora, Colorado 80045
Jacksonville, Florida 32224
Chicago, Illinois 60611
Boston, Massachusetts 02114
Ann Arbor, Michigan 48109 5271
Rochester, Minnesota 55905
Dallas, Texas 75246
Houston, Texas 77030
More Details
- Status
- Recruiting
- Sponsor
- Novartis Pharmaceuticals
Detailed Description
This is a Phase 2, 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 comparator. 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.