ANCA Associated Vasculitis (AAV)Phase 2NCT06868290
Phase 2 Study Evaluating Rapcabtagene Autoleucel in Participants With Severe Active GPA or MPA
A Phase 2 study testing Rapcabtagene autoleucel, Active Comparator and Glucocorticoids in people with ANCA Associated Vasculitis (AAV), run by Novartis Pharmaceuticals at 39 sites in 8 countries.
Trial summary
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) Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- ANCA Associated Vasculitis (AAV)
- What is being tested?
- Rapcabtagene autoleucel, Active Comparator and Glucocorticoids
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 126 participants
- Where
- 39 sitesin 8 countries
- Study dates
- 13 Mar 2025to 24 May 2030
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
39 sites in 8 countries.
39 sites in 8 countries
United States14 sites
Michigan Med University of Michigan
Ann Arbor, Michigan, 48109-5271
University Of Colorado
Aurora, Colorado, 80045
Massachusetts General Hospital
Boston, Massachusetts, 02114
Northwestern University
Chicago, Illinois, 60611
Uni of Chi Medi Ctr Hema and Onco
Chicago, Illinois, 60637
Baylor University Medical Center
Dallas, Texas, 75246
Duke Clinical Research Institute
Durham, North Carolina, 27710
Houston Methodist Hospital
Houston, Texas, 77030
Mayo Clinic Jacksonville
Jacksonville, Florida, 32224
University of Minnesota
Minneapolis, Minnesota, 55455
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Oregon Health Sciences University
Portland, Oregon, 97239
Mayo Clinic Rochester
Rochester, Minnesota, 55905
LDS Hospital
Salt Lake City, Utah, 84143
Japan10 sites
Novartis Investigative Site
Bunkyo Ku, Tokyo, 1138431
Novartis Investigative Site
Chiba, Chiba, 2608677
Novartis Investigative Site
Fukuoka, 8128582
Novartis Investigative Site
Ishikawa, 9208641
Novartis Investigative Site
Kobe, Hyōgo, 6500047
Novartis Investigative Site
Kyoto, 6068507
Novartis Investigative Site
Sapporo, Hokkaido, 0608648
Novartis Investigative Site
Sendai, Miyagi, 9808574
Novartis Investigative Site
Shinjuku-ku, Tokyo, 1608582
Novartis Investigative Site
Suita, Osaka, 5650871
Brazil4 sites
Novartis Investigative Site
Barretos, São Paulo, 14784-400
Novartis Investigative Site
Salvador, Estado de Bahia, 41253-190
Novartis Investigative Site
São Paulo, São Paulo, 01308-050
Novartis Investigative Site
São Paulo, 01509-010
United Kingdom4 sites
Novartis Investigative Site
Cambridge, CB2 0QQ
Novartis Investigative Site
Glasgow, G4 0SF
Novartis Investigative Site
London, W12 0HS
Novartis Investigative Site
Manchester, M13 9WL
Israel2 sites
Novartis Investigative Site
Haifa, 3109601
Novartis Investigative Site
Ramat Gan, 5265601
Singapore2 sites
Novartis Investigative Site
Singapore, Singapore, 308433
Novartis Investigative Site
Singapore, 119074
Switzerland2 sites
Novartis Investigative Site
Basel, 4031
Novartis Investigative Site
Bern, 3010
About this study
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)
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)
Read the 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.
- Conditions
- ANCA Associated Vasculitis (AAV)
- Being tested
- Rapcabtagene autoleucel, Active Comparator, Glucocorticoids
- Keywords
- Chimeric Antigen Receptor-T (CAR-T), rapcabtagene autoleucel, anti-neutrophil cytoplasmic antibody (ANCA), ANCA-associated vasculitis/vasculitides (AAV), Granulomatosis with Polyangiitis (GPA), Microscopic Polyangiitis (MPA)
- Medical subject headings
- Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis, Vasculitis, Granulomatosis with Polyangiitis, Microscopic Polyangiitis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Rapcabtagene autoleucelBiological
Single infusion of rapcabtagene autoleucel
Active ComparatorOther
Active comparator option as per protocol
GlucocorticoidsDrug
Concomitant glucocorticoids as per protocol
| Study group | Role | What they receive |
|---|---|---|
| Rapcabtagene autoleucel | Experimental | Single infusion of rapcabtagene autoleucel (YTB323) and concomitant glucocorticoids as per protocol Receives: Biological: Rapcabtagene autoleucel; Drug: Glucocorticoids |
| Active comparator | Active comparator | Comparator and concomitant glucocorticoids as per protocol Receives: Other: Active Comparator; Drug: Glucocorticoids |
What is being measured
The primary outcome is the main question the study is designed to answer.
Event-free survival (EFS)
Event-free survival (EFS) defined as the time from Randomization to the first occurrence of as per protocol defined events.
Measured: From randomization until the occurrence of an EFS event, up to approx. 4 years after randomization
Secondary outcomes (6)
Percentage of patients achieving complete remission
Complete remission is defined by Birmingham Vasculitis Activity Score version 3 (BVASv3)
Measured: Up to Week 13
Adjusted annual cumulative GC dose between Randomization and analysis cutoff date
The adjusted annual cumulative glucocorticoid dose refers to the total amount of glucocorticoids administered to a patient over the course of a year, adjusted for any changes in dosage, and measured up to the defined week of treatment as per protocol.
Measured: 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
ANCA seronegativity means that the patient tests negative for ANCA antibodies.
Measured: 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
Change from baseline in estimated glomerular filtration rate (eGFR) at Week 39.
Measured: Up to Week 39
Change from baseline in symptoms of GPA/ MPA using the AAV-PRO at Week 39
Change from baseline in symptoms of GPA/ MPA using the AAV-PRO at Week 39.
Measured: Up to Week 39
Change from baseline in Patient- Reported Outcome Measurement Information System (PROMIS)- Fatigue 7a at Week 91.
Change from baseline in PROMIS- Fatigue 7a at Week 91.
Measured: Up to Week 91
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Mar 2025
- Study start13 Mar 2025
- Primary completion7 Jun 2029 (estimated)
- Study completion24 May 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ANCA Associated Vasculitis (AAV)Studies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 126 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06868290
- Sponsor's protocol ID
- CYTB323I12201
- Other IDs
- 2024-515854-26-00
- Status last verified
- Aug 2026
- First submitted
- 7 Mar 2025
- Last updated
- 4 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Novartis is committed to sharing access to patient-level data and supporting clinical documents from eligible studies with qualified external researchers. Requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided are anonymized to protect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com