Study Description
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) 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.
Interventions
Active Comparator Option
Rapcabtagene autoleucel
Eligibility Criteria
Key Inclusion Criteria:
1. 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
2. Participants who had inadequate response to prior therapy
3. Diagnosed with active disease
4. Participant must meet criteria for severe myositis
Key Exclusion Criteria:
1. 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
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 ability of the participant to tolerate CD19 CAR-T cell therapy
Other protocol-defined inclusion/exclusion criteria may apply.
Novartis Investigative Site
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Rennes,35033,France
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Lyon,69003,France
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Brest,29200,France
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Paris,75013,France
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Hamburg,20246,Germany
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Cologne,North Rhine-Westphalia,50937,Germany
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Jena,Thuringia,07740,Germany
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Aachen,52074,Germany
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Leipzig,Saxony,04103,Germany
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Mainz,55131,Germany
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Nuremberg,90419,Germany
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Ulm,89081,Germany
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Ramat Gan,5265601,Israel
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Tel Aviv,6423906,Israel
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Haifa,3109601,Israel
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Monza,MB,20900,Italy
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Brescia,BS,25123,Italy
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Milan,MI,20122,Italy
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Sapporo,Hokkaido,0608648,Japan
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Kanazawa,Ishikawa-ken,920 8641,Japan
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Yokohama,Kanagawa-ku,236-0004,Japan
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Sendai,Miyagi,9808574,Japan
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Bunkyo-ku,Tokyo,113-8603,Japan
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Bunkyo-ku,Tokyo,1138519,Japan
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Shinjuku-ku,Tokyo,1608582,Japan
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Fukuoka,8128582,Japan
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Kyoto,6068507,Japan
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Izumo,Shimane,693 8501,Japan
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Suita,Osaka,565 0871,Japan
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Amsterdam,North Holland,1105 az,Netherlands
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Singapore,119074,Singapore
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Singapore,169608,Singapore
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Singapore,S308433,Singapore
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Santiago Compostela,A Coruna,15706,Spain
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Salamanca,37007,Spain
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Málaga,29010,Spain
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Madrid,28041,Spain
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Pamplona,Navarre,31008,Spain
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Córdoba,14004,Spain
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Barcelona,08035,Spain
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Taipei,10002,Taiwan
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Taichung,407219,Taiwan
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Kaohsiung City,83301,Taiwan
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Sheffield,South Yorkshire,S10 2jf,United Kingdom
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London,Nw1 2bu,United Kingdom
FL Medical Clinic Orlando Health
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Zephyrhills,Florida,33542,United States
Julio Gonzalez-Paoli
William Daily Johnston
University Of Iowa
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Iowa City,Iowa,52242,United States
Hanna Zembrzuska
University Of Alabama
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Birmingham,Alabama,35294,United States
James Andrews
LDS Hospital
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Salt Lake City,Utah,84143,United States
Dany Saad
Northwestern University
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Chicago,Illinois,60611,United States
Matthew Selle
Matthew Selle
George Georges
Worldwide Contacts
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