Un estudio para evaluar la seguridad y eficacia a largo plazo de una formulación subcutánea de efgartigimod en adultos con miopatía inflamatoria idiopática activa (ALKIVIA+)
Una ampliación de fase 3, de un solo brazo, multicéntrica y abierta del estudio ARGX-113-2007 para investigar la seguridad, tolerabilidad y eficacia a largo plazo de efgartigimod PH20 SC en participantes de 18 años o más con miopatía inflamatoria idiopática activa
The main purpose of this study is to measure the long-term safety and tolerability of efgartigimod PH20 SC in adult participants with Idiopathic Inflammatory Myopathy (IIM) who previously participated in ARGX-113-2007.
The study consists of a treatment period where participants will receive efgartigimod PH20 SC for up to 51 months. The treatment period will be followed by a treatment-free safety follow-up period of 56 days.
Descripción general del estudio
Estado
Condiciones
Intervención / Tratamiento
Tipo de estudio
Inscripción (Estimado)
Fase
- Fase 3
Contactos y Ubicaciones
Ubicaciones de estudio
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Berlin, Alemania, 10117
- Charite Universitaetsmedizin Berlin - Campus Charite Mitte (CCM) - Rheumatology
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Hamburg, Alemania, 20246
- Universitaetsklinikum Hamburg-Eppendorf
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Herne, Alemania, 44649
- Rheumazentrum Ruhrgebiet
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Mannheim, Alemania, 68167
- Universitatsmedizin Mannheim GmbH
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Tübingen, Alemania, 72076
- Universitaetsklinikum Tuebingen (UKT)
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Ulm, Alemania, 89081
- Klinikum der Universitaet Ulm
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La Plata, Argentina, B1902COS
- Framingham Centro Medico
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San Miguel, Argentina, B1663GKT
- Centro Dermatologico Schejtman
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San Miguel de Tucumán, Argentina, 4000
- Centro Medico Privado de Reumatologia
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Innsbruck, Austria, 6020
- Tirol Kliniken - A.o. Landeskrankenhaus Innsbruck - Neurology
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Plovdiv, Bulgaria, 4002
- Multiprofile hospital for active treatment Kaspela EOOD
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Plovdiv, Bulgaria, 4002
- Medical Centre Artmed
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Ghent, Bélgica, 9000
- AZ Sint-Lucas - Campus Sint-Lucas - Neurology
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Leuven, Bélgica, 3000
- Universitair Ziekenhuis Leuven Gasthuisberg Campus
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Prague, Chequia, 12850
- Revmatologicky Ustav
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Nicosia, Chipre, 2371
- Cyprus Institute of Neurology and Genetics
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Seoul, Corea del Sur, 03080
- Seoul National University Hospital
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Copenhagen, Dinamarca, 2100
- Copenhagen University Hospital-Rigshospitalet University Hospital
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Barcelona, España, 08041
- Hospital de la Santa Creu i Sant Pau
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Madrid, España, 28041
- Hospital Universitario 12 de Octubre
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Valencia, España, 46026
- Hospital Universitari i Politecnic La Fe de Valencia - Neurology
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Arizona
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Phoenix, Arizona, Estados Unidos, 85028
- Neuromuscular Clinical and Research Center - Neurology
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California
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Beverly Hills, California, Estados Unidos, 90039
- Attune Health Research, Inc
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Orange, California, Estados Unidos, 92868
- UCI Health - ALS and Neuromuscular Center - Neurology
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San Francisco, California, Estados Unidos, 94143
- UCSF Health - ALS and Neurodegenerative Disease Center - Dermatology
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Connecticut
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New Haven, Connecticut, Estados Unidos, 06520
- Yale Cancer Center-Yale University School Of Medicine
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Florida
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Gainesville, Florida, Estados Unidos, 32610
- UF Health - Rheumatology Medical Specialties Medical Plaza
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Jacksonville, Florida, Estados Unidos, 32224
- Mayo Clinic - Florida - Neurology
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Tampa, Florida, Estados Unidos, 33616
- University of South Florida (USF) - Morsani Center (USF Health Carol and Frank Morsani Center for Advanced Healthcare)
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Georgia
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Atlanta, Georgia, Estados Unidos, 30322
- Emory Healthcare - Emory Clinic - Rheumatology
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Illinois
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Chicago, Illinois, Estados Unidos, 60611
- Northwestern Medicine - Northwestern Memorial Hospital
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Maryland
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Baltimore, Maryland, Estados Unidos, 21224-2734
- John Hopkins Medicine - Johns Hopkins Myositis Center
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New York
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Great Neck, New York, Estados Unidos, 11021
- Northwell Health Physician Partners Rheumatology
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North Carolina
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Chapel Hill, North Carolina, Estados Unidos, 27514
- University of North Carolina (UNC) School of Medicine
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Ohio
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Columbus, Ohio, Estados Unidos, 43201
- The Ohio State University
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Oregon
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Portland, Oregon, Estados Unidos, 97239
- Oregon Health and Science University
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Pennsylvania
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Pittsburgh, Pennsylvania, Estados Unidos, 15261
- University of Pittsburgh Medical Center
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Texas
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Austin, Texas, Estados Unidos, 78759
- Austin Neuromuscular Center (National Neuromuscular Research Institute, PLLC)
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Houston, Texas, Estados Unidos, 77030
- Nerve And Muscle Center Of Texas
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Paris, Francia, 75013
- Reference Center Neuro-Muscular Diseases - CHU Paris Group Hospitalier La Pitie Salpetriere-Charles Foix
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Tbilisi, Georgia, 0160
- Aversi Clinic
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Tbilisi, Georgia, 0180
- The First Medical Center
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Tbilisi, Georgia, 0160
- LLC MediClub Georgia
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Tbilisi, Georgia, 0162
- New Hospitals
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Athens, Grecia, 115 27
- National and Kapodistrian University of Athens - School of Health Sciences - Faculty of Medicine
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Athens, Grecia, 115 28
- National and Kapodistrian University of Athens - Eginition Hospital
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Athens, Grecia, 12462
- ATTIKON University General Hospital - Neurology
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Dublin, Irlanda, D04 T6F4
- University College Dublin (UCD) - St. Vincent's University Hospital (SVUH)
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Bari, Italia, 70124
- Azienda Ospedaliero Universitaria Consorziale Policlinico
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Milan, Italia, 20132
- Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - Ospedale San Raffaele Hospital
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Pisa, Italia, 56126
- Azienda Ospedaliero Universitaria Pisana - Ospedale Santa Chiara - Rheumatology
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Roma, Italia, 00189
- Ospedale Sant'Andrea Hospital
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Roma, Italia, 168
- Fondazione Policlinico Universitario Agostino Gemelli - Neurology
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Rozzano, Italia, 20089
- IRCCS Istituto Clinico Humanitas
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Udine, Italia, 33100
- Azienda sanitaria universitaria Friuli Centrale - ASUFC
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Bunkyō City, Japón, 113-8431
- Juntendo University Hospital
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Bunkyō City, Japón, 113-8655
- The University of Tokyo Hospital
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Nagoya, Japón, 457-8510
- Japan Community Healthcare Organization Chukyo Hospital
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Tokyo, Japón, 113-8519
- Institute of Science Tokyo Hospital
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Wakayama, Japón, 641-8510
- Wakayama Medical University Hospital
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Yamaguchi, Japón, 755-8505
- Yamaguchi University Hospital
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Vilnius, Lituania, 08410
- Santaros University Clinic
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Mexico City, México, 06700
- Hospital Angeles Clinica Londres
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Amsterdam, Países Bajos, 1105 AZ
- Amsterdam UMC Research BV
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Arequipa, Perú, 4000
- Clinica San Juan de Dios - sede Arequipa - Rheumatology
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Lima, Perú, 33
- Investigaciones Clinicas S.A.C.
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Lima, Perú, 27
- Instituto Peruano Del Hueso Y La Articulacion Sac-Privado-Lima,Centro De Investigacion Iphar
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Lodz, Polonia, 90-549
- USK-WAM Centralny Szpital Weteranow - Szpital Kniniczny ul. Zeromskiego 113 - Rheumatology
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Szczecin, Polonia, 71-252
- Centrum Wsparcia Badan Klinicznych Pomorskiego Uniwersytetu Medycznego w Szczecinie (CWBK PUM) - Rheumatology
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Beijing, Porcelana, 100730
- Peking Union Medical College Hospital
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Beijing, Porcelana, 100191
- Peking University Third Hospital
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Chengdu, Porcelana, 610072
- Sichuan Provincial Academy of Medical Sciences and Sichuan Provincial People's Hospital
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Shanghai, Porcelana, 200040
- Huashan Hospital Affiliated to Fudan University
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Shanghai, Porcelana, 200001
- Shanghai Jiao Tong University School of Medicine - Renji Hospital - Shanghai Institute of Digestive Disease (SIDD)
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Shenyang, Porcelana, 110001
- China Medical University (CMU) - First Affiliated Hospital
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Zhengzhou, Porcelana, 450052
- First Affiliated Hospital (Henan Medical University - First Affiliated Hospital)-Zhengzhou University
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Guangdong
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Guangzhou, Guangdong, Porcelana, 510515
- Nanfang Hospital, Southern Medical University
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Jingsu
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Nanjing, Jingsu, Porcelana, 210000
- Nanjing University Medical School - Taikang Xianlin Drum Tower Hospital (Nanjing Drum Tower Hospital)
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Porto, Portugal, 4099-001
- Centro Hospitalar do Porto - Hospital Santo Antonio
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Liverpool, Reino Unido, L9 7AL
- Aintree University Hospital - Liverpool University Hospitals NHS Foundation Trust
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London, Reino Unido, NW3 2QG
- The Royal Free Hospital
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Salford, Reino Unido, M6 8HD
- Northern Care Allicance NHS Foundation Trust - Salford Royal - Rheumatology
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Belgrade, Serbia, 11000
- Clinical Center of Serbia
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Belgrade, Serbia, 11000
- Institute of Rheumatology
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Solna, Suecia, 17176
- Karolinska Universitestssjukhus Solna
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Bangkok, Tailandia, 10400
- Phramongkutklao Hospital
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Bangkok, Tailandia, 10330
- King Chulalongkorn Memorial Hospital - Neurology
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Bangkok, Tailandia, 10700
- Siriraj Hospital, Mahidol University of Internal medicine
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Bangkok, Tailandia, 10400
- Ramathibodi hospital
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Khon Kaen, Tailandia, 40002
- Faculty Of Medicine, Khon Kaen University
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Taichung, Taiwán, 402
- Chung Shan Medical University Hospital
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Taichung, Taiwán
- Taichung Veterans General Hospital
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Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
Acepta Voluntarios Saludables
Géneros elegibles para el estudio
Descripción
Inclusion Criteria:
- Has completed trial ARGX-113-2007
- Being capable of providing signed informed consent and complying with protocol requirements
- Agrees to use contraceptive measures consistent with local regulations and women of childbearing potential must have a negative urine pregnancy test at baseline before receiving the study drug
Exclusion Criteria:
- Intention to have major surgery during the study period or any other medical condition that has arisen since enrollment in study ARGX-113-2007, that in the investigator's opinion, would confound the results of the study or put the participant at undue risk
- Known hypersensitivity reaction to the study drug or 1 of its excipients
- Development of any malignancy, either new or recurrent, other than basal cell carcinoma of the skin, regardless of relatedness
Plan de estudios
¿Cómo está diseñado el estudio?
Detalles de diseño
- Propósito principal: Tratamiento
- Asignación: N / A
- Modelo Intervencionista: Asignación de un solo grupo
- Enmascaramiento: Ninguno (etiqueta abierta)
Armas e Intervenciones
Grupo de participantes/brazo |
Intervención / Tratamiento |
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Experimental: Efgartigimod PH20 SC
Participants receiving efgartigimod PH20 subcutaneously |
Subcutaneous injection of Efgartigimod PH20 given by vials or prefilled syringe (PFS) |
¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Incidence of AEs, SAEs and AESIs over time
Periodo de tiempo: Up to 53 months
|
AE : adverse event ; SAE : serious adverse event ; AESI : adverse event of special interest. |
Up to 53 months
|
Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
|
Reducción de dosis de prednisona (dosis media mensual) a lo largo del tiempo
Periodo de tiempo: Hasta 51 meses
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Hasta 51 meses
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Porcentaje de cambio desde la línea de base en los niveles totales de IgG en suero a lo largo del tiempo
Periodo de tiempo: Hasta 53 meses
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Hasta 53 meses
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Duración de la dosificación cada 2 semanas
Periodo de tiempo: Hasta 51 meses
|
Hasta 51 meses
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C-GTI comprising the AIS over time
Periodo de tiempo: Up to 51 months
|
The Composite Glucocorticoid Toxicity Index (C-GTI) serves as a primary instrument to capture toxicities likely caused by glucocorticoid exposure and enables monitoring of long-term tolerability of glucocorticoids during prolonged use. The Aggregate Improvement Score (AIS) is an analytical scores generated from the weighted C-GTI. The AIS can be used to assess whether a new therapy is effective in reducing glucocorticoid toxicity over time; higher AIS scores are associated with less toxicity. |
Up to 51 months
|
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C-GTI comprising the CWS over time
Periodo de tiempo: Up to 51 months
|
The Composite Glucocorticoid Toxicity Index (C-GTI) serves as a primary instrument to capture toxicities likely caused by glucocorticoid exposure and enables monitoring of long-term tolerability of glucocorticoids during prolonged use. The Cumulative Worsening Score (CWS) is an analytical score generated from the weighted C-GTI. The CWS is designed to assess cumulative glucocorticoid toxicity; higher scores indicate increased steroid toxicity. |
Up to 51 months
|
|
C-GTI comprising the GTI-MD over time
Periodo de tiempo: Up to 51 months
|
The Composite Glucocorticoid Toxicity Index (C-GTI) serves as a primary instrument to capture toxicities likely caused by glucocorticoid exposure and enables monitoring of long-term tolerability of glucocorticoids during prolonged use. The Glucocorticoid Toxicity Index-Metabolic Domains (GTI-MD) is an abbreviated GTI version limited to the 4 metabolic domains. A GTI-MD score of 0 indicates a low likelihood of toxicity in the assessed metabolic domains. |
Up to 51 months
|
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TIS over time
Periodo de tiempo: Up to 51 months
|
The total improvement score (TIS) assesses minimal, moderate, and major clinical response, and it is assessed using the ACR/EULAR criteria. The total score varies between 0 and 100, with higher scores indicating greater improvement. |
Up to 51 months
|
|
Proportion of TIS responders (minimal, moderate, major) over time
Periodo de tiempo: Up to 51 months
|
The total improvement score (TIS) assesses minimal, moderate, and major clinical response, and it is assessed using the ACR/EULAR criteria. The total score varies between 0 and 100, with higher scores indicating greater improvement |
Up to 51 months
|
|
Individual CSMs of the TIS over time
Periodo de tiempo: Up to 51 months
|
The total improvement score (TIS) assesses minimal, moderate, and major clinical response, and it is assessed using the ACR/EULAR criteria. The ACR/EULAR criteria calculate TIS using the 6 core set measure (CSMs): MDGA, PGA, MMT8, HAQ-DI, muscle enzymes, and extramuscular global assessment (assessed by the MDAAT). |
Up to 51 months
|
|
Percentage of participants with clinically inactive disease
Periodo de tiempo: Up to 51 months
|
Clinically inactive disease is defined as no evidence of disease activity, based on an Physician Global Assessment of Disease Activity (MDGA) and Extramuscular Global Assessment of the Myositis Disease Activity Assessment Tool (MDAAT) of 0 and normal creatine kinase (CK) values for at least 12 weeks (at week 28 and every 24 weeks thereafter). |
Up to 51 months
|
|
Percentage of participants with remission during the study
Periodo de tiempo: Up to 51 months
|
Remission is defined as a clinically inactive disease for at least 24 weeks |
Up to 51 months
|
|
PGI-S over time
Periodo de tiempo: Up to 51 months
|
The Patient Global Impression of Severity (PGI-S) asks participants to rate the severity of their disease symptoms over the past 7 days on a 4-point Likert scale, where scores range from "no symptoms" to "severe." |
Up to 51 months
|
|
CGI-S over time
Periodo de tiempo: Up to 51 months
|
The Clinical Global Impression of Severity (CGI-S) asks the physician to rate the severity of the participant's disease on a 4-point Likert scale, where scores range from "no activity" to "severe". |
Up to 51 months
|
|
Physical Functioning subscale and Physical Component Summary scores of the SF-36v2 over time
Periodo de tiempo: Up to 51 months
|
The 36-Item Short Form Survey version 2 (SF-36v2) is a 36-item scale constructed to survey health-related quality of life in 8 domains. |
Up to 51 months
|
|
EQ-5D-5L utilities over time
Periodo de tiempo: Up to 51 months
|
The EQ-5D-5L questionnaire is a standardized test recognized by many health authorities as a generic measure of health status for clinical and economic appraisal. The descriptive system comprises 5 dimensions, each having 5 levels ranging from "No problem" to "Extreme problem". |
Up to 51 months
|
|
EQ-5D-5L VAS over time
Periodo de tiempo: Up to 51 months
|
The EQ-5D-5L questionnaire is a standardized test recognized by many health authorities as a generic measure of health status for clinical and economic appraisal. A visual analog scale (VAS) is included in the EQ-5D-5L, where participants will mark their health status from 0 (the worst health you can imagine) to 100 (the best health you can imagine). |
Up to 51 months
|
|
Incidence of ADA against efgartigimod (serum levels) over time
Periodo de tiempo: Up to 53 months
|
ADA : anti-drug antibodies. |
Up to 53 months
|
|
Proportion of participants achieving sustained low disease activity while on IMP, over time
Periodo de tiempo: Up to 51 months
|
Sustained low disease activity is defined as a CGI-S score of "mild" or "no activity" and a PGI-S score of "mild" or "no symptoms" for at least 12 weeks. |
Up to 51 months
|
|
Proportion of participants with low disease activity over time, while on IMP
Periodo de tiempo: Up to 51 months
|
Low disease activity is defined as a CGI-S score of "mild" or "no activity" and a PGI-S score of "mild" or "no symptoms" |
Up to 51 months
|
|
Proportion of participants with low disease activity who continue dosing every 2 weeks at week 52 and every 24 weeks thereafter
Periodo de tiempo: Up to 51 months
|
Low disease activity is defined as a CGI-S score of "mild" or "no activity" and a PGI-S score of "mild" or "no symptoms" |
Up to 51 months
|
|
Proportion of participants who have marked worsening during the period of dosing every 2 weeks
Periodo de tiempo: Up to 51 months
|
Marked worsening is defined as a CGI-S score of "moderate" or "severe" and a PGI-S score of "moderate" or "severe" |
Up to 51 months
|
Colaboradores e Investigadores
Patrocinador
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio (Actual)
Finalización primaria (Estimado)
Finalización del estudio (Estimado)
Fechas de registro del estudio
Enviado por primera vez
Primero enviado que cumplió con los criterios de control de calidad
Publicado por primera vez (Actual)
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
Última actualización enviada que cumplió con los criterios de control de calidad
Última verificación
Más información
Términos relacionados con este estudio
Términos MeSH relevantes adicionales
Otros números de identificación del estudio
- ARGX-113-2011
- 2022-502851-79-00 (Ctis)
Plan de datos de participantes individuales (IPD)
¿Planea compartir datos de participantes individuales (IPD)?
Información sobre medicamentos y dispositivos, documentos del estudio
Estudia un producto farmacéutico regulado por la FDA de EE. UU.
Estudia un producto de dispositivo regulado por la FDA de EE. UU.
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. .