Phase 2 Study of Rapcabtagene Autoleucel in Myositis

Last updated: January 21, 2026
Sponsor: Novartis Pharmaceuticals
Overall Status: Active - Recruiting

Phase

2

Condition

Idiopathic Inflammatory Myopathies

Collagen Vascular Diseases

Lupus

Treatment

Rapcabtagene autoleucel

Active Comparator Option

Clinical Study ID

NCT06665256
CYTB323L12201
  • Ages 18-75
  • All Genders

Study 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 Criteria

Inclusion

Key Inclusion Criteria:

  1. Men and women, aged ≥ 18 and ≤75 years, with a diagnosis of probable or definitemyositis according to American College of Rheumatology/European League AgainstRheumatism 2017 (ACR/EULAR 2017) criteria

  2. Participants who had inadequate response to prior therapy

  3. Diagnosed with active disease

  4. Participant must meet criteria for severe myositis

Exclusion

Key Exclusion Criteria:

  1. Any condition during Screening that could prevent a complete washout of medicationsor could otherwise make the participant ineligible for anti-CD19 CAR-T therapy andfurther participation in the study

  2. BMI at Screening of ≤17 or ≥40 kg/m2

  3. Severe muscle damage at Screening

  4. Inadequate organ function

  5. Hypersensitivity and/or contraindications to any product (including its ingredients)to be given to the participant as per the study protocol

  6. Other inflammatory and non-inflammatory myopathies

  7. Any medical conditions that are not related to IIM that would jeopardize the abilityof the participant to tolerate CD19 CAR-T cell therapy

Other protocol-defined inclusion/exclusion criteria may apply.

Study Design

Total Participants: 123
Treatment Group(s): 2
Primary Treatment: Rapcabtagene autoleucel
Phase: 2
Study Start date:
December 17, 2024
Estimated Completion Date:
July 17, 2030

Study Description

This is a Phase 2, randomized, 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 a comparator option.

Participants in the comparator arm whose signs and symptoms are not fully controlled may receive rapcabtagene autoleucel treatment once the participant is confirmed to be eligible

After end of study (EOS), participants who received rapcabtagene autoleucel infusion will enter a long-term follow-up (LTFU) period after rapcabtagene autoleucel infusion. This LTFU will be described in a separate study protocol.

Connect with a study center

  • Novartis Investigative Site

    Brest, 29200
    France

    Site Not Available

  • Novartis Investigative Site

    Brest 3030300, 29200
    France

    Active - Recruiting

  • Novartis Investigative Site

    Lille 2998324, 59037
    France

    Suspended

  • Novartis Investigative Site

    Lyon, 69003
    France

    Site Not Available

  • Novartis Investigative Site

    Lyon 2996944, 69003
    France

    Active - Recruiting

  • Novartis Investigative Site

    Paris, 75013
    France

    Site Not Available

  • Novartis Investigative Site

    Paris 2988507, 75013
    France

    Active - Recruiting

  • Novartis Investigative Site

    Paris 13, 75651
    France

    Site Not Available

  • Novartis Investigative Site

    Rennes, 35000
    France

    Site Not Available

  • Novartis Investigative Site

    Rennes 2983990, 35033
    France

    Active - Recruiting

  • Novartis Investigative Site

    Cologne 2886242, North Rhine-Westphalia 2861876 50937
    Germany

    Active - Recruiting

  • Novartis Investigative Site

    Leipzig, Sachsen 04103
    Germany

    Site Not Available

  • Novartis Investigative Site

    Leipzig 2879139, Saxony 2842566 04103
    Germany

    Active - Recruiting

  • Novartis Investigative Site

    Jena 2895044, Thuringia 2822542 07740
    Germany

    Active - Recruiting

  • Novartis Investigative Site

    Aachen 3247449, 52074
    Germany

    Active - Recruiting

  • Novartis Investigative Site

    Hamburg, 20246
    Germany

    Site Not Available

  • Novartis Investigative Site

    Hamburg 2911298, 20246
    Germany

    Active - Recruiting

  • Novartis Investigative Site

    Jena, 07740
    Germany

    Site Not Available

  • Novartis Investigative Site

    Koeln, 50937
    Germany

    Site Not Available

  • Novartis Investigative Site

    Mainz 2874225, 55131
    Germany

    Active - Recruiting

  • Novartis Investigative Site

    Nuremberg 2861650, 90419
    Germany

    Active - Recruiting

  • Novartis Investigative Site

    Ulm 2820256, 89081
    Germany

    Active - Recruiting

  • Novartis Investigative Site

    Haifa, 3109601
    Israel

    Site Not Available

  • Novartis Investigative Site

    Haifa 294801, 3109601
    Israel

    Active - Recruiting

  • Novartis Investigative Site

    Ramat Gan, 5265601
    Israel

    Site Not Available

  • Novartis Investigative Site

    Ramat Gan 293788, 5265601
    Israel

    Active - Recruiting

  • Novartis Investigative Site

    Tel Aviv, 6423906
    Israel

    Site Not Available

  • Novartis Investigative Site

    Tel Aviv 293397, 6423906
    Israel

    Active - Recruiting

  • Novartis Investigative Site

    Brescia, BS 25123
    Italy

    Site Not Available

  • Novartis Investigative Site

    Brescia 3181554, BS 25123
    Italy

    Active - Recruiting

  • Novartis Investigative Site

    Monza 3172629, MB 20900
    Italy

    Active - Recruiting

  • Novartis Investigative Site

    Milan 6951411, MI 20122
    Italy

    Active - Recruiting

  • Novartis Investigative Site

    Milano, MI 20122
    Italy

    Site Not Available

  • Novartis Investigative Site

    Sapporo city, Hokkaido 060 8648
    Japan

    Site Not Available

  • Novartis Investigative Site

    Sapporo 2128295, Hokkaido 2130037 0608648
    Japan

    Active - Recruiting

  • Novartis Investigative Site

    Kanazawa, Ishikawa 920 8641
    Japan

    Site Not Available

  • Novartis Investigative Site

    Kanazawa 1860243, Ishikawa-ken 1861387 920 8641
    Japan

    Active - Recruiting

  • Novartis Investigative Site

    Yokohama 1848354, Kanagawa-ku 236-0004
    Japan

    Active - Recruiting

  • Novartis Investigative Site

    Sendai city, Miyagi 980 8574
    Japan

    Site Not Available

  • Novartis Investigative Site

    Sendai 2111149, Miyagi 2111888 9808574
    Japan

    Active - Recruiting

  • Novartis Investigative Site

    Suita, Osaka 565 0871
    Japan

    Site Not Available

  • Novartis Investigative Site

    Suita 1851483, Osaka 1853904 565 0871
    Japan

    Active - Recruiting

  • Novartis Investigative Site

    Izumo-city, Shimane 693 8501
    Japan

    Site Not Available

  • Novartis Investigative Site

    Izumo 1861084, Shimane 1852442 693 8501
    Japan

    Active - Recruiting

  • Novartis Investigative Site

    Bunkyo-ku, Tokyo 1850144 1138519
    Japan

    Active - Recruiting

  • Novartis Investigative Site

    Shinjuku-ku, Tokyo 1850144 1608582
    Japan

    Active - Recruiting

  • Novartis Investigative Site

    Fukuoka 1863967, 8128582
    Japan

    Active - Recruiting

  • Novartis Investigative Site

    Kyoto, 606 8507
    Japan

    Site Not Available

  • Novartis Investigative Site

    Kyoto 1857910, 6068507
    Japan

    Active - Recruiting

  • Novartis Investigative Site

    Amsterdam 2759794, North Holland 2749879 1105 AZ
    Netherlands

    Active - Recruiting

  • Novartis Investigative Site

    Singapore, S308433
    Singapore

    Site Not Available

  • Novartis Investigative Site

    Singapore 1880252, S308433
    Singapore

    Active - Recruiting

  • Novartis Investigative Site

    Santiago Compostela, A Coruna 15706
    Spain

    Active - Recruiting

  • Novartis Investigative Site

    Barcelona, Catalunya 08035
    Spain

    Site Not Available

  • Novartis Investigative Site

    Pamplona, Navarra 31008
    Spain

    Site Not Available

  • Novartis Investigative Site

    Pamplona 3114472, Navarre 3115609 31008
    Spain

    Active - Recruiting

  • Novartis Investigative Site

    Barcelona 3128760, 08035
    Spain

    Active - Recruiting

  • Novartis Investigative Site

    Córdoba 2519240, 14004
    Spain

    Active - Recruiting

  • Novartis Investigative Site

    Madrid 3117735, 28041
    Spain

    Active - Recruiting

  • Novartis Investigative Site

    Málaga 2514256, 29010
    Spain

    Active - Recruiting

  • Novartis Investigative Site

    Salamanca 3111108, 37007
    Spain

    Active - Recruiting

  • Novartis Investigative Site

    Kaohsiung City 1673820, 83301
    Taiwan

    Active - Recruiting

  • Novartis Investigative Site

    Taichung, 407219
    Taiwan

    Site Not Available

  • Novartis Investigative Site

    Taichung 1668399, 407219
    Taiwan

    Active - Recruiting

  • Novartis Investigative Site

    Taipei, 10002
    Taiwan

    Site Not Available

  • Novartis Investigative Site

    Taipei 1668341, 10002
    Taiwan

    Active - Recruiting

  • Novartis Investigative Site

    Sheffield 2638077, South Yorkshire S10 2JF
    United Kingdom

    Active - Recruiting

  • Novartis Investigative Site

    London 2643743, NW1 2BU
    United Kingdom

    Active - Recruiting

  • University Of Alabama

    Birmingham 4049979, Alabama 4829764 35294
    United States

    Active - Recruiting

  • FL Medical Clinic Orlando Health

    Zephyrhills 4178941, Florida 4155751 33542
    United States

    Active - Recruiting

  • Northwestern University

    Chicago, Illinois 60611
    United States

    Site Not Available

  • Northwestern University

    Chicago 4887398, Illinois 4896861 60611
    United States

    Active - Recruiting

  • University Of Iowa

    Iowa City, Iowa 52242
    United States

    Site Not Available

  • University Of Iowa

    Iowa City 4862034, Iowa 4862182 52242
    United States

    Active - Recruiting

  • LDS Hospital

    Salt Lake City, Utah 84143
    United States

    Site Not Available

  • LDS Hospital

    Salt Lake City 5780993, Utah 5549030 84143
    United States

    Active - Recruiting

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