Phase 2 Study Evaluating Rapcabtagene Autoleucel in Participants With Severe Active GPA or MPA

May 19, 2026 updated by: Novartis Pharmaceuticals

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)

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Estimated)

126

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Brazil
      • São Paulo, Brazil, 01509-010
        • Recruiting
        • Novartis Investigative Site
    • Estado de Bahia
      • Salvador, Estado de Bahia, Brazil, 41253-190
        • Recruiting
        • Novartis Investigative Site
    • São Paulo
      • Barretos, São Paulo, Brazil, 14784 400
        • Recruiting
        • Novartis Investigative Site
      • São Paulo, São Paulo, Brazil, 01308-050
        • Recruiting
        • Novartis Investigative Site
  • Israel
      • Haifa, Israel, 3109601
        • Recruiting
        • Novartis Investigative Site
      • Ramat Gan, Israel, 5265601
        • Recruiting
        • Novartis Investigative Site
  • Japan
      • Chiba, Japan, 2608677
        • Recruiting
        • Novartis Investigative Site
      • Fukuoka, Japan, 8128582
        • Recruiting
        • Novartis Investigative Site
      • Ishikawa, Japan, 9208641
        • Recruiting
        • Novartis Investigative Site
      • Kyoto, Japan, 6068507
        • Recruiting
        • Novartis Investigative Site
    • Hokkaido
      • Sapporo, Hokkaido, Japan, 060-8648
        • Recruiting
        • Novartis Investigative Site
    • Hyōgo
      • Kobe, Hyōgo, Japan, 6500047
        • Recruiting
        • Novartis Investigative Site
    • Miyagi
      • Sendai, Miyagi, Japan, 9808574
        • Recruiting
        • Novartis Investigative Site
    • Osaka
      • Suita, Osaka, Japan, 565-0871
        • Recruiting
        • Novartis Investigative Site
    • Tokyo
      • Bunkyo Ku, Tokyo, Japan, 1138431
        • Recruiting
        • Novartis Investigative Site
      • Shinjuku-ku, Tokyo, Japan, 1608582
        • Recruiting
        • Novartis Investigative Site
  • Saudi Arabia
      • Riyadh, Saudi Arabia, 11211
        • Recruiting
        • Novartis Investigative Site
  • Singapore
      • Singapore, Singapore, 119074
        • Recruiting
        • Novartis Investigative Site
      • Singapore, Singapore, S308433
        • Recruiting
        • Novartis Investigative Site
  • Switzerland
      • Basel, Switzerland, 4031
        • Recruiting
        • Novartis Investigative Site
      • Bern, Switzerland, 3010
        • Recruiting
        • Novartis Investigative Site
  • United Kingdom
      • Cambridge, United Kingdom, CB2 0QQ
        • Recruiting
        • Novartis Investigative Site
      • London, United Kingdom, W12 0HS
        • Recruiting
        • Novartis Investigative Site
      • Manchester, United Kingdom, M13 9WL
        • Recruiting
        • Novartis Investigative Site
  • United States
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Recruiting
        • University of Colorado
        • Principal Investigator:
          • Larry Moreland
        • Contact:
    • Florida
      • Jacksonville, Florida, United States, 32224
        • Recruiting
        • Mayo Clinic Jacksonville
        • Principal Investigator:
          • Vikas Majithia
        • Contact:
    • Illinois
      • Chicago, Illinois, United States, 60611
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Recruiting
        • Massachusetts General Hospital
        • Principal Investigator:
          • John Stone
        • Contact:
    • Michigan
      • Ann Arbor, Michigan, United States, 48109 5271
        • Recruiting
        • Michigan Med University of Michigan
        • Principal Investigator:
          • Monalisa Ghosh
        • Contact:
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • Recruiting
        • University of Minnesota
        • Contact:
        • Principal Investigator:
          • Patrick Nachman
      • Rochester, Minnesota, United States, 55905
        • Recruiting
        • Mayo Clinic Rochester
        • Principal Investigator:
          • Ulrich Specks
        • Contact:
    • Oregon
      • Portland, Oregon, United States, 97239
        • Recruiting
        • Oregon Health Sciences University
        • Principal Investigator:
          • Atul Deodhar
        • Contact:
    • Texas
      • Dallas, Texas, United States, 75246
      • Houston, Texas, United States, 77030
    • Utah
      • Salt Lake City, Utah, United States, 84143
        • Recruiting
        • LDS Hospital
        • Principal Investigator:
          • Catherine Jennifer Bakewell
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Key inclusion criteria:

  1. 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

Key exclusion criteria:

  1. 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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Single

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Rapcabtagene autoleucel
Single infusion of rapcabtagene autoleucel (YTB323) and concomitant glucocorticoids as per protocol
Biological: Rapcabtagene autoleucel
Single infusion of rapcabtagene autoleucel
Drug: Glucocorticoids
Concomitant glucocorticoids as per protocol
Active Comparator: Active comparator
Comparator and concomitant glucocorticoids as per protocol
Drug: Glucocorticoids
Concomitant glucocorticoids as per protocol
Other: Active Comparator
Active comparator option as per protocol

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Event-free survival (EFS)
Time Frame: From randomization until the occurrence of an EFS event, up to approx. 4 years after randomization
Event-free survival (EFS) defined as the time from Randomization to the first occurrence of as per protocol defined events.
From randomization until the occurrence of an EFS event, up to approx. 4 years after randomization

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percentage of patients achieving complete remission
Time Frame: Up to Week 13
Complete remission is defined by Birmingham Vasculitis Activity Score version 3 (BVASv3)
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
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.
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
ANCA seronegativity means that the patient tests negative for ANCA antibodies.
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 estimated glomerular filtration rate (eGFR) at Week 39.
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 symptoms of GPA/ MPA using the AAV-PRO at Week 39.
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
Change from baseline in PROMIS- Fatigue 7a at Week 91.
Up to Week 91

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Novartis Pharmaceuticals

Investigators

  • Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 13, 2025

Primary Completion (Estimated)

June 7, 2029

Study Completion (Estimated)

May 24, 2030

Study Registration Dates

First Submitted

March 7, 2025

First Submitted That Met QC Criteria

March 7, 2025

First Posted (Actual)

March 10, 2025

Study Record Updates

Last Update Posted (Actual)

May 20, 2026

Last Update Submitted That Met QC Criteria

May 19, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CYTB323I12201

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

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

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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