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NCT Number: NCT06868290

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

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Key information

Conditions

ANCA Associated Vasculitis (AAV) Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis Autoimmune Diseases Brain Diseases Cardiovascular Diseases Central Nervous System Diseases Cerebral Small Vessel Diseases Cerebrovascular Disorders Granulomatosis with Polyangiitis Immune System Diseases Lung Diseases Lung Diseases, Interstitial Microscopic Polyangiitis Nervous System Diseases Respiratory Tract Diseases Skin Diseases Skin Diseases, Vascular Skin and Connective Tissue Diseases Systemic Vasculitis Vascular Diseases Vasculitis

Age range

18 year–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Novartis Investigative Site, Salvador, Estado de Bahia, Brazil

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Key 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

Key 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

Treatment and study plan

Rapcabtagene autoleucel

Biological

Single infusion of rapcabtagene autoleucel

Active comparator

Other

Active comparator option as per protocol

Glucocorticoids

Drug

Concomitant glucocorticoids as per protocol

Primary outcomes

  1. 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.

Secondary outcomes

  1. Percentage of patients achieving complete remission

    Time frame: Up to Week 13

    Complete remission is defined by Birmingham Vasculitis Activity Score version 3 (BVASv3)

  2. 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.

  3. 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.

  4. 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.

  5. 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.

  6. 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.

Study contacts

Contact information is provided by the study sponsor or research team.

Novartis Pharmaceuticals

CONTACT

[email protected]

1-888-669-6682

Novartis Pharmaceuticals

CONTACT

[email protected]

+41613241111

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study 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)

Important dates

Study start
2025
Primary completion
2029
Study completion
2030
First posted
Mar 10, 2025
Registry last updated
May 20, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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