Phase 2 Study Evaluating Rapcabtagene Autoleucel in Participants With Severe Active GPA or MPA
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Rapcabtagene autoleucel, Active Comparator, Glucocorticoids.
- Who it may be relevant to
- Registry conditions: ANCA Associated Vasculitis (AAV). Basic parameters: 18 years — 75 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Brazil, Israel, Japan, Saudi Arabia +3
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Phase 2, Randomized, Open-label, Controlled Study to Evaluate the Efficacy and Safety of Rapcabtagene Autoleucel Versus Comparator in Participants With Severe Active Granulomatosis With Polyangiitis (GPA) or Microscopic Polyangiitis (MPA)
Overview
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)
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.
Interventions
- Biological Rapcabtagene autoleucel
Single infusion of rapcabtagene autoleucel - Other Active Comparator
Active comparator option as per protocol - Drug Glucocorticoids
Concomitant glucocorticoids as per protocol
Primary outcome measures
- Event-free survival (EFS) [Time frame: From randomization until the occurrence of an EFS event, up to approx. 4 years after randomization]
Secondary outcome measures (6)
- Percentage of patients achieving complete remission [Time frame: Up to Week 13]
- Adjusted annual cumulative GC dose between Randomization and analysis cutoff date [Time frame: From randomization until the occurrence of an EFS event, up to approx. 4 years after randomization]
- ANCA seronegativity and sustaining ANCA seronegativity until the analysis cutoff date [Time frame: From randomization until the occurrence of an EFS event, up to approx. 4 years after randomization]
- Change from baseline in estimated glomerular filtration rate (eGFR) at Week 39 [Time frame: Up to Week 39]
- Change from baseline in symptoms of GPA/ MPA using the AAV-PRO at Week 39 [Time frame: Up to Week 39]
- Change from baseline in Patient- Reported Outcome Measurement Information System (PROMIS)- Fatigue 7a at Week 91. [Time frame: Up to Week 91]
Eligibility criteria
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
- Positive test for ANCA-autoantibodies
- 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
- Hypersensitivity and/or contraindications to any product to be given to the participant as part of the study protocol
- Other systemic autoimmune diseases requiring therapy
- 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
- Inadequate organ function
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Single blind
- Primary purpose
- Treatment
Study locations
United States · 11 centers
- University Of Colorado — Aurora
- Mayo Clinic Jacksonville — Jacksonville
- Northwestern University — Chicago
- Massachusetts General Hospital — Boston
- Michigan Med University of Michigan — Ann Arbor
- University of Minnesota — Minneapolis
- Mayo Clinic Rochester — Rochester
- Oregon Health Sciences University — Portland
- … and 3 more centers
Japan · 10 centers
- Novartis Investigative Site — Sapporo
- Novartis Investigative Site — Kobe
- Novartis Investigative Site — Sendai
- Novartis Investigative Site — Suita
- Novartis Investigative Site — Bunkyo Ku
- Novartis Investigative Site — Shinjuku-ku
- Novartis Investigative Site — Chiba
- Novartis Investigative Site — Fukuoka
- … and 2 more centers
Brazil · 4 centers
- Novartis Investigative Site — Salvador
- Novartis Investigative Site — Barretos
- Novartis Investigative Site — São Paulo
- Novartis Investigative Site — São Paulo
United Kingdom · 3 centers
- Novartis Investigative Site — Cambridge
- Novartis Investigative Site — London
- Novartis Investigative Site — Manchester
Israel · 2 centers
- Novartis Investigative Site — Haifa
- Novartis Investigative Site — Ramat Gan
Singapore · 2 centers
- Novartis Investigative Site — Singapore
- Novartis Investigative Site — Singapore
Switzerland · 2 centers
- Novartis Investigative Site — Basel
- Novartis Investigative Site — Bern
Saudi Arabia · 1 center
- Novartis Investigative Site — Riyadh
Identifiers
NCT: NCT06868290 · CYTB323I12201