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

NCT06868290 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: CYTB323I12201
• Study Type: interventional
• Target: 126
• Locations: 8 countries
• Sites: 31

Brief 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)

Conditions

ANCA Associated Vasculitis (AAV)

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)

Outcome Measures

Primary Outcomes (1)

• Event-free survival (EFS)

  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 Outcomes (6)

• Percentage of patients achieving complete remission

  Up to Week 13

• Adjusted annual cumulative GC dose between Randomization and analysis cutoff date

  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

  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

  Up to Week 39

• Change from baseline in symptoms of GPA/ MPA using the AAV-PRO at Week 39

  Up to Week 39

Study Arms (2)

Rapcabtagene autoleucel

EXPERIMENTAL

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

Biological: Rapcabtagene autoleucel; Drug: Glucocorticoids

Active comparator

ACTIVE COMPARATOR

Comparator and concomitant glucocorticoids as per protocol

Other: Active Comparator; Drug: Glucocorticoids

Interventions

Rapcabtagene autoleucel BIOLOGICAL

Single infusion of rapcabtagene autoleucel

Rapcabtagene autoleucel

Active Comparator OTHER

Active comparator option as per protocol

Active comparator

Glucocorticoids DRUG

Concomitant glucocorticoids as per protocol

Active comparator; Rapcabtagene autoleucel

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (31)

University Of Colorado

Aurora, Colorado, 80045, United States

RECRUITING

Mayo Clinic Jacksonville

Jacksonville, Florida, 32224, United States

RECRUITING

Northwestern University

Chicago, Illinois, 60611, United States

RECRUITING

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

RECRUITING

Mayo Clinic Rochester

Rochester, Minnesota, 55905, United States

RECRUITING

Oregon Health Sciences University

Portland, Oregon, 97239, United States

RECRUITING

Houston Methodist Hospital

Houston, Texas, 77030, United States

RECRUITING

LDS Hospital

Salt Lake City, Utah, 84143, United States

RECRUITING

Novartis Investigative Site

Salvador, Estado de Bahia, 41253-190, Brazil

RECRUITING

Novartis Investigative Site

Barretos, São Paulo, 14784 400, Brazil

RECRUITING

Novartis Investigative Site

São Paulo, São Paulo, 01308-050, Brazil

RECRUITING

Novartis Investigative Site

São Paulo, 01509-010, Brazil

RECRUITING

Novartis Investigative Site

Haifa, 3109601, Israel

RECRUITING

Novartis Investigative Site

Ramat Gan, 5265601, Israel

RECRUITING

Novartis Investigative Site

Sapporo, Hokkaido, 0608648, Japan

RECRUITING

Novartis Investigative Site

Kobe, Hyōgo, 6500047, Japan

RECRUITING

Novartis Investigative Site

Kanazawa, Ishikawa-ken, 920 8641, Japan

RECRUITING

Novartis Investigative Site

Sendai, Miyagi, 9808574, Japan

RECRUITING

Novartis Investigative Site

Suita, Osaka, 5650871, Japan

RECRUITING

Novartis Investigative Site

Bunkyo Ku, Tokyo, 1138431, Japan

RECRUITING

Novartis Investigative Site

Shinjuku-ku, Tokyo, 1608582, Japan

RECRUITING

Novartis Investigative Site

Chiba, 260 8677, Japan

RECRUITING

Novartis Investigative Site

Fukuoka, 8128582, Japan

RECRUITING

Novartis Investigative Site

Kyoto, 6068507, Japan

RECRUITING

Novartis Investigative Site

Riyadh, 11211, Saudi Arabia

RECRUITING

Novartis Investigative Site

Singapore, 119074, Singapore

RECRUITING

Novartis Investigative Site

Singapore, S308433, Singapore

RECRUITING

Novartis Investigative Site

Basel, 4031, Switzerland

RECRUITING

Novartis Investigative Site

Bern, 3010, Switzerland

RECRUITING

Novartis Investigative Site

Cambridge, CB2 0QQ, United Kingdom

RECRUITING

Novartis Investigative Site

London, W12 0HS, United Kingdom

RECRUITING

MeSH Terms

Conditions

Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis; Vasculitis; Granulomatosis with Polyangiitis; Microscopic Polyangiitis

Interventions

Glucocorticoids

Condition Hierarchy (Ancestors)

Systemic Vasculitis; Vascular Diseases; Cardiovascular Diseases; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Lung Diseases, Interstitial; Lung Diseases; Respiratory Tract Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Intervention Hierarchy (Ancestors)

Adrenal Cortex Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Physiological Effects of Drugs; Pharmacologic Actions; Chemical Actions and Uses

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

1-888-669-6682; novartis.email@novartis.com

Novartis Pharmaceuticals

CONTACT

+41613241111; novartis.email@novartis.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Masking Details: The study investigator and the participant will be unblinded to the study treatment. A blinded assessor will perform the efficacy assessments to minimize bias in data collection.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 7, 2025
• First Posted: March 10, 2025
• Study Start: March 13, 2025
• Primary Completion (Estimated): June 7, 2029
• Study Completion (Estimated): May 24, 2030
• Last Updated: February 23, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will share

Locations

CH (2); SA (1); SG (2); US (8); IL (2); JP (10); GB (2); BR (4)