RecruitingPhase 2NCT06665256

Phase 2 Study of Rapcabtagene Autoleucel in Myositis

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)


Sponsor

Novartis Pharmaceuticals

Enrollment

123 participants

Start Date

Dec 17, 2024

Study Type

INTERVENTIONAL

Conditions

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)


Eligibility

Min Age: 18 YearsMax Age: 75 Years

Inclusion Criteria4

  • 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
  • Participant must meet criteria for severe myositis

Exclusion Criteria7

  • 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

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Interventions

BIOLOGICALRapcabtagene autoleucel

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

OTHERActive Comparator Option

Investigator choice of treatment as per protocol


Locations(55)

University Of Alabama

Birmingham, Alabama, United States

FL Medical Clinic Orlando Health

Zephyrhills, Florida, United States

Northwestern University

Chicago, Illinois, United States

Northwestern University

Chicago, Illinois, United States

University Of Iowa

Iowa City, Iowa, United States

LDS Hospital

Salt Lake City, Utah, United States

Novartis Investigative Site

Brest, France

Novartis Investigative Site

Lille, France

Novartis Investigative Site

Lyon, France

Novartis Investigative Site

Paris, France

Novartis Investigative Site

Rennes, France

Novartis Investigative Site

Cologne, North Rhine-Westphalia, Germany

Novartis Investigative Site

Leipzig, Saxony, Germany

Novartis Investigative Site

Jena, Thuringia, Germany

Novartis Investigative Site

Aachen, Germany

Novartis Investigative Site

Hamburg, Germany

Novartis Investigative Site

Mainz, Germany

Novartis Investigative Site

Nuremberg, Germany

Novartis Investigative Site

Ulm, Germany

Novartis Investigative Site

Haifa, Israel

Novartis Investigative Site

Ramat Gan, Israel

Novartis Investigative Site

Tel Aviv, Israel

Novartis Investigative Site

Brescia, BS, Italy

Novartis Investigative Site

Monza, MB, Italy

Novartis Investigative Site

Milan, MI, Italy

Novartis Investigative Site

Sapporo, Hokkaido, Japan

Novartis Investigative Site

Kanazawa, Ishikawa-ken, Japan

Novartis Investigative Site

Yokohama, Kanagawa-ku, Japan

Novartis Investigative Site

Sendai, Miyagi, Japan

Novartis Investigative Site

Suita, Osaka, Japan

Novartis Investigative Site

Izumo, Shimane, Japan

Novartis Investigative Site

Bunkyo-ku, Tokyo, Japan

Novartis Investigative Site

Bunkyo-ku, Tokyo, Japan

Novartis Investigative Site

Shinjuku-ku, Tokyo, Japan

Novartis Investigative Site

Fukuoka, Japan

Novartis Investigative Site

Kyoto, Japan

Novartis Investigative Site

Amsterdam, North Holland, Netherlands

Novartis Investigative Site

Riyadh, Saudi Arabia

Novartis Investigative Site

Singapore, Singapore

Novartis Investigative Site

Singapore, Singapore

Novartis Investigative Site

Singapore, Singapore

Novartis Investigative Site

Santiago Compostela, A Coruna, Spain

Novartis Investigative Site

Santander, Cantabria, Spain

Novartis Investigative Site

Pamplona, Navarre, Spain

Novartis Investigative Site

Barcelona, Spain

Novartis Investigative Site

Córdoba, Spain

Novartis Investigative Site

Madrid, Spain

Novartis Investigative Site

Málaga, Spain

Novartis Investigative Site

Salamanca, Spain

Novartis Investigative Site

Geneva, Switzerland

Novartis Investigative Site

Kaohsiung City, Taiwan

Novartis Investigative Site

Taichung, Taiwan

Novartis Investigative Site

Taipei, Taiwan

Novartis Investigative Site

Sheffield, South Yorkshire, United Kingdom

Novartis Investigative Site

London, United Kingdom

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