Un estudio clínico para evaluar ianalumab en participantes con esclerosis sistémica cutánea difusa
12 de octubre de 2026 actualizado por: Novartis Pharmaceuticals
Un estudio multicéntrico aleatorizado, doble ciego, de grupos paralelos y controladora con placebo para evaluar la eficacia, seguridad y tolerabilidad de ianalumab en participantes con esclerosis sistémica cutánea difusa
The purpose of this study is to evaluate efficacy, safety and tolerability of s.c. ianalumab administered in participants with diffuse cutaneous systemic sclerosis relative to placebo
Descripción general del estudio
Estado
Activo, no reclutando
Condiciones
Intervención / Tratamiento
Descripción detallada
The study consists of the following periods:
- Screening Period, with a duration of up to 6 weeks;
- Treatment Period 1, with a duration of 52 weeks;
- Treatment Period 2 (Open-label treatment), with a duration of 52 weeks;
- Post-treatment Follow-up Period, with a duration of at least 20 weeks post last dose and up to 2 years.
Tipo de estudio
Intervencionista
Inscripción (Actual)
201
Fase
- Fase 2
Acceso ampliado
Disponible fuera del ensayo clínico.
Ver registro de acceso ampliado.
Contactos y Ubicaciones
Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.
Ubicaciones de estudio
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Berlin, Alemania, 13353
- Novartis Investigative Site
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Bavaria
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Würzburg, Bavaria, Alemania, 97080
- Novartis Investigative Site
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Thuringia
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Jena, Thuringia, Alemania, 07740
- Novartis Investigative Site
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Caba, Argentina, C1015ABO
- Novartis Investigative Site
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Caba, Argentina, C1427CCL
- Novartis Investigative Site
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San Miguel de Tucumán, Argentina, T4000AXL
- Novartis Investigative Site
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Buenos Aires
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CABA, Buenos Aires, Argentina, C1221ADC
- Novartis Investigative Site
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Graz, Austria, 8036
- Novartis Investigative Site
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Estado de Bahia
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Salvador, Estado de Bahia, Brasil, 40150-150
- Novartis Investigative Site
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Paraná
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Curitiba, Paraná, Brasil, 80030-110
- Novartis Investigative Site
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Rio Grande do Sul
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Porto Alegre, Rio Grande do Sul, Brasil, 90035-074
- Novartis Investigative Site
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São Paulo
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Sao Jose Rio Preto, São Paulo, Brasil, 15090-000
- Novartis Investigative Site
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São Paulo, São Paulo, Brasil, 04038-002
- Novartis Investigative Site
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Leuven, Bélgica, 3000
- Novartis Investigative Site
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Bogotá, Colombia, 110231
- Novartis Investigative Site
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Medellín, Colombia, 050034
- Novartis Investigative Site
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Antioquia
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Medellín, Antioquia, Colombia, 050001
- Novartis Investigative Site
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Cundinamarca
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Bogota, Cundinamarca, Colombia, 110111
- Novartis Investigative Site
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Bogota, Cundinamarca, Colombia, 110221
- Novartis Investigative Site
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Valle del Cauca Department
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Cali, Valle del Cauca Department, Colombia, 760012
- Novartis Investigative Site
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Seoul, Corea del Sur, 04763
- Novartis Investigative Site
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Seoul, Corea del Sur, 06273
- Novartis Investigative Site
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Seoul
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Seoul, Seoul, Corea del Sur, 03080
- Novartis Investigative Site
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Barcelona, España, 08041
- Novartis Investigative Site
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Madrid, España, 28009
- Novartis Investigative Site
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Salamanca, España, 37007
- Novartis Investigative Site
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Andalusia
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Málaga, Andalusia, España, 29009
- Novartis Investigative Site
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Arizona
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Mesa, Arizona, Estados Unidos, 85202
- Arizona Arthritis and Rheumatology Research PLLC
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California
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Los Angeles, California, Estados Unidos, 90095
- UCLA
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Newport Beach, California, Estados Unidos, 92663
- Hoag Hospital
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Florida
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Clearwater, Florida, Estados Unidos, 33765
- Clinical Res Of W Florida
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Cooper City, Florida, Estados Unidos, 33024
- GNP Research
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Plantation, Florida, Estados Unidos, 33324
- IRIS Research and Development
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Sarasota, Florida, Estados Unidos, 34239
- Sarasota Arthritis Res Ctr
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Illinois
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Chicago, Illinois, Estados Unidos, 60637
- University of Chicago Hospitals
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Michigan
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Ann Arbor, Michigan, Estados Unidos, 48109
- Uni Of Michigan Health System
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New York
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New York, New York, Estados Unidos, 10021
- Hospital for Special Surgery
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Tennessee
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Jackson, Tennessee, Estados Unidos, 38305
- West Tennessee Research Institute
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Texas
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Bellaire, Texas, Estados Unidos, 77401
- Novel Research LLC
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Frisco, Texas, Estados Unidos, 75035
- Arthritis and Rheumatology Ins
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Houston, Texas, Estados Unidos, 77054
- Prolato Clinical Research Center
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Dijon, Francia, 21000
- Novartis Investigative Site
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Le Mans, Francia, 72000
- Novartis Investigative Site
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Lille, Francia, 59037
- Novartis Investigative Site
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Montpellier, Francia, 34295
- Novartis Investigative Site
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Paris, Francia, 75014
- Novartis Investigative Site
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Rennes, Francia, 35033
- Novartis Investigative Site
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Strasbourg, Francia, 67000
- Novartis Investigative Site
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Toulouse, Francia, 31054
- Novartis Investigative Site
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Alexandroupoli, Grecia, 681 00
- Novartis Investigative Site
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Athens, Grecia, 115 27
- Novartis Investigative Site
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Athens, Grecia, 115 21
- Novartis Investigative Site
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Budapest, Hungría, 1083
- Novartis Investigative Site
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Baranya
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Pécs, Baranya, Hungría, 7623
- Novartis Investigative Site
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Hajdu Bihar Megye
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Debrecen, Hajdu Bihar Megye, Hungría, 4032
- Novartis Investigative Site
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Kerala
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Kochi, Kerala, India, 682018
- Novartis Investigative Site
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Maharashtra
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Mumbai, Maharashtra, India, 400078
- Novartis Investigative Site
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National Capital Territory of Delhi
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New Delhi, National Capital Territory of Delhi, India, 110060
- Novartis Investigative Site
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New Delhi
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New Delhi, New Delhi, India, 110029
- Novartis Investigative Site
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Rajasthan
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Jaipur, Rajasthan, India, 302004
- Novartis Investigative Site
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AN
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Ancona, AN, Italia, 60020
- Novartis Investigative Site
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MI
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Milan, MI, Italia, 20100
- Novartis Investigative Site
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Rozzano, MI, Italia, 20089
- Novartis Investigative Site
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MO
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Modena, MO, Italia, 41124
- Novartis Investigative Site
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PA
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Palermo, PA, Italia, 90127
- Novartis Investigative Site
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PV
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Pavia, PV, Italia, 27100
- Novartis Investigative Site
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RM
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Roma, RM, Italia, 00168
- Novartis Investigative Site
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VR
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Verona, VR, Italia, 37134
- Novartis Investigative Site
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Hiroshima, Japón, 7348551
- Novartis Investigative Site
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Ishikawa, Japón, 9208641
- Novartis Investigative Site
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Kyoto, Japón, 6068507
- Novartis Investigative Site
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Aichi-ken
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Nagoya, Aichi-ken, Japón, 4578510
- Novartis Investigative Site
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Toyoake, Aichi-ken, Japón, 4701192
- Novartis Investigative Site
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Kanagawa
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Yokohama, Kanagawa, Japón, 2360004
- Novartis Investigative Site
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Saitama
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Iruma-gun, Saitama, Japón, 3500495
- Novartis Investigative Site
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Tokyo
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Bunkyo-ku, Tokyo, Japón, 1138603
- Novartis Investigative Site
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Shinjuku Ku, Tokyo, Japón, 1628666
- Novartis Investigative Site
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Toyama
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Toyama, Toyama, Japón, 9300194
- Novartis Investigative Site
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Kuala Lumpur, Malasia, 59100
- Novartis Investigative Site
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Kuala Lumpur
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Cheras, Kuala Lumpur, Malasia, 56000
- Novartis Investigative Site
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Chihuahua
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Chihuahua City, Chihuahua, México, 31000
- Novartis Investigative Site
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Jalisco
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Guadalajara, Jalisco, México, 44650
- Novartis Investigative Site
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Mexico City
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Mexico City, Mexico City, México, 11850
- Novartis Investigative Site
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Nuevo León
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Monterrey, Nuevo León, México, 64460
- Novartis Investigative Site
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Bydgoszcz, Polonia, 85-168
- Novartis Investigative Site
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Warsaw, Polonia, 00-874
- Novartis Investigative Site
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Beijing, Porcelana, 100191
- Novartis Investigative Site
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Tianjin, Porcelana, 300052
- Novartis Investigative Site
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Guangxi
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Nanning, Guangxi, Porcelana, 530021
- Novartis Investigative Site
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Henan
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Zhengzhou, Henan, Porcelana, 450003
- Novartis Investigative Site
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Sichuan
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Chengdu, Sichuan, Porcelana, 610041
- Novartis Investigative Site
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Zhejiang
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Ningbo, Zhejiang, Porcelana, 315016
- Novartis Investigative Site
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Vila Nova de Gaia, Portugal, 4434-502
- Novartis Investigative Site
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Bristol, Reino Unido, BS10 5NB
- Novartis Investigative Site
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London, Reino Unido, NW3 2QG
- Novartis Investigative Site
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Bangkok, Tailandia, 10400
- Novartis Investigative Site
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THA
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Khon Kaen, THA, Tailandia, 40002
- Novartis Investigative Site
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Changhua, Taiwán, 50006
- Novartis Investigative Site
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Kaohsiung City, Taiwán, 833
- Novartis Investigative Site
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Taichung, Taiwán, 40447
- Novartis Investigative Site
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Taoyuan, Taiwán, 33305
- Novartis Investigative Site
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Fatih
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Istanbul, Fatih, Turquía (Türkiye), 34093
- Novartis Investigative Site
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Sihhiye Altindag
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Ankara, Sihhiye Altindag, Turquía (Türkiye), 06230
- Novartis Investigative Site
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Yenimahalle
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Ankara, Yenimahalle, Turquía (Türkiye), 06500
- Novartis Investigative Site
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Ho Chi Minh City, Vietnam, 700000
- Novartis Investigative Site
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VNM
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Ho Chi Minh City, VNM, Vietnam, 700000
- Novartis Investigative Site
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Criterios de participación
Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.
Criterio de elegibilidad
Edades elegibles para estudiar
18 años a 70 años (Adulto, Adulto Mayor)
Acepta Voluntarios Saludables
No
Géneros elegibles para el estudio
Todos
Descripción
Key Inclusion Criteria:
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Male and female participants >= 18 and =< 70 years (at the time of the screening visit).
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Diagnosis of systemic sclerosis, as defined by the 2013 American College of Rheumatology/ European League Against Rheumatism (ACR/EULAR) classification criteria for SSc (van den Hoogen et al 2013) and meet the dcSSc subset classification according to LeRoy (LeRoy 1988)
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Disease duration of =< 60 months (defined as time from the first non-Raynaud phenomenon manifestation, e.g., puffy hands, scleroderma, digital ulcers, arthralgia, dyspnea)
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mRSS units of >= 15 and =< 45 at the time of the screening visit
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Active disease that meets at least one of the following criteria at screening:
- Disease duration of =< 18 months defined as time from the first non-Raynaud phenomenon manifestation
- Increase in mRSS of >= 3 units compared with the most recent assessment performed within the previous 6 months
- Involvement of one new body area and an increase in mRSS of >= 2 units compared with the most recent assessment performed within the previous 6 months
- Involvement of two new body areas within the previous 6 months
- Elevated acute phase reactants (ESR) >= 30 mm/hr or high-sensitivity C-reactive protein (hsCRP) >= 6 mg/L)
- Presence of SSc-interstitial lung disease (ILD) and ATA autoantibody positivity
- Modified EUSTAR disease activity index (mDAI) ≥ 2.5
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Participant must be positive for at least one of the following autoantibodies:
- anti-topoisomerase I (ATA) (also known as anti-SCL-70)
- anti-RNA polymerase III (anti-RNAP3)
- anti-nuclear antibody (ANA) (≥ 1:80) Participants who are positive only for ANA (while being negative for both ATA /anti-RNAP3) will be limited to 30% of the overall randomized study population.
Key Exclusion Criteria:
- Rheumatic disease other than dcSSc, including limited cutaneous disease (lcSSc) or sine scleroderma at the screening visit. Secondary Sjogren's disease and scleroderma myopathy are not exclusionary.
- Positive anti-centromere antibody (ACA+) without positive ATA or anti-RNAP3 autoantibody result at the screening visit
- Previous improvement (decrease) in mRSS > 10 units
- Pulmonary disease with FVC ≤ 50% of predicted or diffusing capacity of the lung for carbon monoxide (DLCO, corrected for hemoglobin) ≤ 40% of predicted at the screening visit
- WHO Functional Class 3 or higher assessment for pulmonary arterial hypertension (PAH, as defined on right heart catheterization), receiving IV therapy for PAH or evidence of other moderately severe pulmonary disease
- Participants treated with cyclophosphamide within 12 weeks prior to Baseline.
- Prior use of a B-cell depleting therapy other than ianalumab (e.g., rituximab, other anti-CD20 mAb, anti-CD22 mAb, or anti-CD52 mAb) administered within 36 weeks prior to randomization, or as long as B cell count is less than the lower limit of normal or baseline value prior to receipt of B cell-depleting therapy (whichever is lower).
- Treatment with biologic agents, such as intravenous immunoglobulin or monoclonal antibodies, including marketed drugs, within 12 weeks or 5 half-lives (whichever is longer) prior to baseline visit, unless explicitly allowed in inclusion criteria.
- Treatment with any investigational agent within ≤ 4 weeks (or 5 half-lives of the investigational drug, whichever is longer) of the baseline visit.
- Use of anti-fibrotic agents including colchicine, D-penicillamine, pirfenidone, or tyrosine kinase inhibitors (e.g., nintedanib, nilotinib, imatinib, dasatinib) in the 4 weeks prior to baseline visit. Patients with SSc-ILD requiring antifibrotics for management of ILD during the study, as per investigator judgement, should be excluded.
- Previous treatment with chlorambucil, bone marrow transplantation or total lymphoid irradiation.
- Women of childbearing potential, defined as all women physiologically capable of becoming pregnant from menarche until becoming post-menopausal, unless they are using highly effective methods of contraception (failure rate < 1% per year) while taking study treatment and for 6 months after stopping study treatment.
Other protocol-defined inclusion/exclusion criteria may apply.
Plan de estudios
Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.
¿Cómo está diseñado el estudio?
Detalles de diseño
- Propósito principal: Tratamiento
- Asignación: Aleatorizado
- Modelo Intervencionista: Asignación paralela
- Enmascaramiento: Cuadruplicar
Armas e Intervenciones
Grupo de participantes/brazo |
Intervención / Tratamiento |
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Experimental: VAY736 (Ianalumab)
Treatment Period 1: Ianalumab subcutaneous (s.c.) injection as defined in the protocol Treatment Period 2: Open-label (OL) Ianalumab subcutaneous (s.c.) injection as defined in the protocol |
subcutaneous (s.c.) injection as defined in the protocol
Otros nombres:
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Comparador de placebos: Placebo
Treatment Period 1: Placebo to Ianalumab subcutaneous (s.c.) injection as defined in the protocol Treatment Period 2: Open-label (OL) Ianalumab subcutaneous (s.c.) injection as defined in the protocol |
subcutaneous (s.c.) injection as defined in the protocol
Otros nombres:
Ianalumab matching placebo subcutaneous (s.c.) injection as defined in the protocol |
¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
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3/5 rCRISS25 response
Periodo de tiempo: Week 52
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To demonstrate the superiority of ianalumab, compared to placebo, in achieving 3/5 Revised Composite Response Index in Systemic Sclerosis 25 (rCRISS25) response at Week 52 |
Week 52
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Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
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Change from baseline in FVC% predicted at Week 52
Periodo de tiempo: Week 52
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To demonstrate the superiority of ianalumab, compared to placebo, on change from baseline in percent-predicted forced vital capacity (FVC%) at Week 52 |
Week 52
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Change from baseline in mRSS at Week 52
Periodo de tiempo: Week 52
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To demonstrate the superiority of ianalumab, compared to placebo, on change from baseline in modified Rodnan Skin Score (mRSS) at Week 52 |
Week 52
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Change from baseline in HAQ-DI at Week 52
Periodo de tiempo: Week 52
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To demonstrate the superiority of ianalumab, compared to placebo, on change from baseline in Health Assessment Questionnaire Disability Index (HAQ-DI) at Week 52 |
Week 52
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Ianalumab concentrations in serum during treatment and Follow-up Period
Periodo de tiempo: Baseline, Week 4, Week 8, Week 12, Week 24, Week 36, Week 52, Week 64, Week 76, Week 88, Week 104, Week 108, Week 112, Week 116, Week 120, Week 124 and Week 208
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To assess the pharmacokinetics of ianalumab |
Baseline, Week 4, Week 8, Week 12, Week 24, Week 36, Week 52, Week 64, Week 76, Week 88, Week 104, Week 108, Week 112, Week 116, Week 120, Week 124 and Week 208
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Incidence and titer of anti-drug (ianalumab) antibodies (ADAs) in serum over time
Periodo de tiempo: Baseline, Week 4, Week 8, Week 12, Week 24, Week 36, Week 52, Week 64, Week 76, Week 88, Week 104, Week 124, Week 208
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To evaluate the immunogenicity of ianalumab |
Baseline, Week 4, Week 8, Week 12, Week 24, Week 36, Week 52, Week 64, Week 76, Week 88, Week 104, Week 124, Week 208
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Incidence and severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Periodo de tiempo: Up to Week 208
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The distribution of adverse events for ianalumab will be done via the analysis of frequencies for Adverse Event (AEs) and Serious Adverse Event (SAEs) through the monitoring of relevant clinical and laboratory safety parameters. |
Up to Week 208
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Colaboradores e Investigadores
Aquí es donde encontrará personas y organizaciones involucradas en este estudio.
Patrocinador
Investigadores
- Director de estudio: Novartis Pharmaceuticals, Novartis Pharmaceuticals
Publicaciones y enlaces útiles
La persona responsable de ingresar información sobre el estudio proporciona voluntariamente estas publicaciones. Estos pueden ser sobre cualquier cosa relacionada con el estudio.
Fechas de registro del estudio
Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.
Fechas importantes del estudio
Inicio del estudio (Actual)
9 de octubre de 2024
Finalización primaria (Estimado)
30 de julio de 2027
Finalización del estudio (Estimado)
31 de julio de 2034
Fechas de registro del estudio
Enviado por primera vez
17 de junio de 2024
Primero enviado que cumplió con los criterios de control de calidad
17 de junio de 2024
Publicado por primera vez (Actual)
24 de junio de 2024
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
13 de octubre de 2026
Última actualización enviada que cumplió con los criterios de control de calidad
12 de octubre de 2026
Última verificación
1 de octubre de 2026
Más información
Términos relacionados con este estudio
Palabras clave
- Agotamiento de células B
- Esclerosis sistémica difusa
- Esclerodermia Difusa
- capacidad vital forzada
- Esclerodermia difusa
- Esclerosis sistémica cutánea difusa (dcSSc)
- Esclerodermia progresiva
- Esclerosis Sistémica Progresiva
- Esclerodermia de aparición repentina
- Índice de respuesta compuesto revisado en esclerosis sistémica 25 (rCRISS25)
- puntuación cutánea de Rodnan modificada
Términos MeSH relevantes adicionales
Otros números de identificación del estudio
- CVAY736S12201
- 2024-511933-36-00 (Identificador de registro: EU CT number)
Plan de datos de participantes individuales (IPD)
¿Planea compartir datos de participantes individuales (IPD)?
SÍ
Descripción del plan IPD
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.
This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
Información sobre medicamentos y dispositivos, documentos del estudio
Estudia un producto farmacéutico regulado por la FDA de EE. UU.
Sí
Estudia un producto de dispositivo regulado por la FDA de EE. UU.
No
Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .