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

Novartis

Status and phase: Enrolling; Phase 2

Conditions: ANCA Associated Vasculitis (AAV)

Treatments: Drug: Glucocorticoids; Other: Active Comparator; Biological: Rapcabtagene autoleucel

Study type: Interventional

Funder types: Industry

Identifiers

NCT06868290

CYTB323I12201

Details and patient eligibility

About

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)

Full 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.

Enrollment: 126 estimated patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Single Blind

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126 participants in 2 patient groups

Rapcabtagene autoleucel

Experimental group

Description:

Single infusion of rapcabtagene autoleucel (YTB323) and concomitant glucocorticoids as per protocol

Treatment:

Biological: Rapcabtagene autoleucel

Drug: Glucocorticoids

Active comparator

Active Comparator group

Description:

Comparator and concomitant glucocorticoids as per protocol

Treatment:

Other: Active Comparator

Drug: Glucocorticoids