Last Update: Apr 04, 2025
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)
ClinicalTrials.gov Identifier:
Novartis Reference Number:CYTB323I12201
All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation.

Study Description

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) 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.

ANCA Associated Vasculitis (AAV)
Phase2
Recruiting
126
Mar 13, 2025
May 24, 2030
All
18 Years - 75 Years (Adult, Older Adult)

Interventions

Other

Active Comparator

Active comparator option as per protocol
Drug

Glucocorticoids

Concomitant glucocorticoids as per protocol
Biological

Rapcabtagene autoleucel

Single infusion of rapcabtagene autoleucel

Eligibility Criteria

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

Novartis Investigative Site

Recruiting

Chiba,2608677,Japan

Novartis Investigative Site

Recruiting

Singapore,119074,Singapore

Worldwide Contacts

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Novartis Pharmaceuticals

Novartis Pharmaceuticals