Phase 2 Study of Rapcabtagene Autoleucel in Myositis

NCT06665256 | Active Not Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: CYTB323L12201
• Study Type: interventional
• Target: 21
• Locations: 15 countries
• Sites: 68

Brief Summary

A Phase 2, randomized, open-label, controlled study to evaluate the efficacy and safety of rapcabtagene autoleucel versus comparator in participants with severe refractory idiopathic inflammatory myopathies (IIM)

Conditions

Idiopathic Inflammatory Myopathies

Keywords

Chimeric Antigen Receptor T cells (CAR-T); rapcabtagene autoleucel; idiopathic inflammatory myopathies (IIM); dermatomyositis; Anti-Synthetase Syndrome; Immune-Mediated Necrotizing Myopathy; Interstitial Lung Disease

Outcome Measures

Primary Outcomes (1)

• Proportion of participants achieving moderate- to-major improvement in Total Improvement Score (TIS) at Week 52

  The percentage of participants with a TIS of at least 40 at the 52nd week after the start of the study, corresponding to moderate-to-major improvement.

  Week 52

Secondary Outcomes (4)

• Adjusted annual cumulative glucocorticoid dose up to Week 52

  Week 52

• Change from baseline in percent predicted Forced Vital Capacity (FVC%) at Week 52

  Baseline, Week 52

• Proportion of participants achieving major improvement in TIS at Week 52

  Week 52

• Change from baseline in Patient-Reported-Outcome Measurement Information System (PROMIS)-Fatigue 7a at Week 52

  Baseline, Week 52

Study Arms (2)

Rapcabtagene autoleucel

EXPERIMENTAL

Single infusion of rapcabtagene autoleucel (YTB323)

Biological: Rapcabtagene autoleucel

Comparator

ACTIVE COMPARATOR

Investigator choice of treatment as per protocol. (Tacrolimus, Mycophenolate mofetil, Cyclophosphamide, or Rituximab)

Other: Active Comparator Option

Interventions

Rapcabtagene autoleucel BIOLOGICAL

Single infusion of rapcabtagene autoleucel after lymphodepleting therapy with fludarabine (adjusted based on renal impairment) and cyclophosphamide daily for 3 days.

Also known as: YTB323

Rapcabtagene autoleucel

Active Comparator Option OTHER

Investigator choice of treatment as per protocol

Comparator

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 probable or definite myositis according to American College of Rheumatology/European League Against Rheumatism 2017 (ACR/EULAR 2017) criteria
• Participants who had inadequate response to prior therapy
• Diagnosed with active disease such as presence of at least 1 of the following criteria: abnormal enzyme levels assessed as secondary to IIM, or EMG demonstrating active disease, DM skin rash, muscle biopsy demonstrating active IIM, or MRI demonstrating active inflammation.
• Participant must meet criteria for severe myositis such as presence of active muscle weakness.

You may not qualify if:

• Any condition during Screening 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
• BMI at Screening of ≤17 or ≥40 kg/m2
• Severe muscle damage at Screening
• Inadequate organ function
• Hypersensitivity and/or contraindications to any product (including its ingredients) to be given to the participant as per the study protocol
• Other inflammatory and non-inflammatory myopathies
• Any medical conditions that are not related to IIM that would jeopardize the ability of the participant to tolerate CD19 CAR-T cell therapy

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (68)

University Of Alabama

Birmingham, Alabama, 35294, United States

Location

FL Medical Clinic Orlando Health

Zephyrhills, Florida, 33542, United States

Location

Augusta University Georgia

Augusta, Georgia, 30912, United States

Location

Northwestern University

Chicago, Illinois, 60611, United States

Location

University Of Iowa

Iowa City, Iowa, 52242, United States

Location

Duke Clinical Research Institute

Durham, North Carolina, 27710, United States

Location

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15261, United States

Location

Baylor University Medical Center

Dallas, Texas, 75246, United States

Location

Houston Methodist Hospital

Houston, Texas, 77030, United States

Location

LDS Hospital

Salt Lake City, Utah, 84143, United States

Location

Novartis Investigative Site

Melbourne, Victoria, 3004, Australia

Location

Novartis Investigative Site

Barretos, São Paulo, 14784 400, Brazil

Location

Novartis Investigative Site

São Paulo, São Paulo, 01232-010, Brazil

Location

Novartis Investigative Site

São Paulo, São Paulo, 01308-050, Brazil

Location

Novartis Investigative Site

Brest, 29200, France

Location

Novartis Investigative Site

Lille, 59037, France

Location

Novartis Investigative Site

Lyon, 69003, France

Location

Novartis Investigative Site

Marseille, 13885, France

Location

Novartis Investigative Site

Paris, 75013, France

Location

Novartis Investigative Site

Rennes, 35033, France

Location

Novartis Investigative Site

Cologne, North Rhine-Westphalia, 50937, Germany

Location

Novartis Investigative Site

Leipzig, Saxony, 04103, Germany

Location

Novartis Investigative Site

Jena, Thuringia, 07740, Germany

Location

Novartis Investigative Site

Aachen, 52074, Germany

Location

Novartis Investigative Site

Hamburg, 20246, Germany

Location

Novartis Investigative Site

Mainz, 55131, Germany

Location

Novartis Investigative Site

Nuremberg, 90419, Germany

Location

Novartis Investigative Site

Ulm, 89081, Germany

Location

Novartis Investigative Site

Haifa, 3109601, Israel

Location

Novartis Investigative Site

Ramat Gan, 5265601, Israel

Location

Novartis Investigative Site

Tel Aviv, 6423906, Israel

Location

Novartis Investigative Site

Ancona, AN, 60126, Italy

Location

Novartis Investigative Site

Brescia, BS, 25123, Italy

Location

Novartis Investigative Site

Monza, MB, 20900, Italy

Location

Novartis Investigative Site

Milan, MI, 20122, Italy

Location

Novartis Investigative Site

Rozzano, MI, 20089, Italy

Location

Novartis Investigative Site

Verona, VR, 37134, Italy

Location

Novartis Investigative Site

Sapporo, Hokkaido, 060-8648, Japan

Location

Novartis Investigative Site

Yokohama, Kanagawa, 236-0004, Japan

Location

Novartis Investigative Site

Sendai, Miyagi, 9808574, Japan

Location

Novartis Investigative Site

Suita, Osaka, 565-0871, Japan

Location

Novartis Investigative Site

Izumo, Shimane, 6938501, Japan

Location

Novartis Investigative Site

Bunkyo-ku, Tokyo, 113-8603, Japan

Location

Novartis Investigative Site

Bunkyo-ku, Tokyo, 1138519, Japan

Location

Novartis Investigative Site

Shinjuku-ku, Tokyo, 1608582, Japan

Location

Novartis Investigative Site

Fukuoka, 8128582, Japan

Location

Novartis Investigative Site

Ishikawa, 9208641, Japan

Location

Novartis Investigative Site

Kyoto, 6068507, Japan

Location

Novartis Investigative Site

Amsterdam, North Holland, 1105 AZ, Netherlands

Location

Novartis Investigative Site

Riyadh, 11211, Saudi Arabia

Location

Novartis Investigative Site

Singapore, 119074, Singapore

Location

Novartis Investigative Site

Singapore, 169608, Singapore

Location

Novartis Investigative Site

Singapore, S308433, Singapore

Location

Novartis Investigative Site

Santiago Compostela, A Coruna, 15706, Spain

Location

Novartis Investigative Site

Santander, Cantabria, 39008, Spain

Location

Novartis Investigative Site

Pamplona, Navarre, 31008, Spain

Location

Novartis Investigative Site

Barcelona, 08035, Spain

Location

Novartis Investigative Site

Córdoba, 14004, Spain

Location

Novartis Investigative Site

Madrid, 28009, Spain

Location

Novartis Investigative Site

Madrid, 28041, Spain

Location

Novartis Investigative Site

Málaga, 29010, Spain

Location

Novartis Investigative Site

Salamanca, 37007, Spain

Location

Novartis Investigative Site

Geneva, 1211, Switzerland

Location

Novartis Investigative Site

Kaohsiung City, 83301, Taiwan

Location

Novartis Investigative Site

Taichung, 407219, Taiwan

Location

Novartis Investigative Site

Taipei, 10002, Taiwan

Location

Novartis Investigative Site

Sheffield, South Yorkshire, S10 2JF, United Kingdom

Location

Novartis Investigative Site

London, NW1 2BU, United Kingdom

Location

MeSH Terms

Conditions

Myositis; Dermatomyositis; Lung Diseases, Interstitial

Condition Hierarchy (Ancestors)

Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Polymyositis; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Lung Diseases; Respiratory Tract Diseases

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 29, 2024
• First Posted: October 30, 2024
• Study Start: December 17, 2024
• Primary Completion (Estimated): March 9, 2029
• Study Completion (Estimated): November 29, 2032
• Last Updated: June 23, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will share

Locations

NL (1); BR (3); IL (3); SA (1); CH (1); DE (8); FR (6); SG (3); TW (3); ES (9); US (10); GB (2); JP (11); IT (6); AU (1)