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| ID | Type | Description | Link |
|---|---|---|---|
| 2022-502851-79-00 | EU Trial (CTIS) Number |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Efgartigimod PH20 SC | Experimental | Participants receiving efgartigimod PH20 subcutaneously |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Efgartigimod PH20 SC | Biological | Subcutaneous injection of Efgartigimod PH20 given by vials or prefilled syringe (PFS) |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of AEs, SAEs and AESIs over time | AE : adverse event ; SAE : serious adverse event ; AESI : adverse event of special interest. | Up to 53 months |
| Measure | Description | Time Frame |
|---|---|---|
| C-GTI comprising the AIS over time | 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Neuromuscular Clinical and Research Center - Neurology | Phoenix | Arizona | 85028 | United States | ||
| Attune Health Research, Inc |
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| Up to 51 months |
| C-GTI comprising the CWS over time | 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 | 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 |
| Prednisone dose reduction (average monthly dose) over time | Up to 51 months |
| TIS over time | 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 | 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 | 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 | 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 | Remission is defined as a clinically inactive disease for at least 24 weeks | Up to 51 months |
| PGI-S over time | 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 | 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 | 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 | 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 | 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 |
| Percent change from baseline in total IgG levels in serum over time | Up to 53 months |
| Incidence of ADA against efgartigimod (serum levels) over time | ADA : anti-drug antibodies. | Up to 53 months |
| Proportion of participants achieving sustained low disease activity while on IMP, over time | 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 | 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 | 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 | 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 |
| Duration of dosing every 2 weeks | Up to 51 months |
| Beverly Hills |
| California |
| 90039 |
| United States |
| UCI Health - ALS and Neuromuscular Center - Neurology | Orange | California | 92868 | United States |
| UCSF Health - ALS and Neurodegenerative Disease Center - Dermatology | San Francisco | California | 94143 | United States |
| Yale Cancer Center-Yale University School Of Medicine | New Haven | Connecticut | 06520 | United States |
| UF Health - Rheumatology Medical Specialties Medical Plaza | Gainesville | Florida | 32610 | United States |
| Mayo Clinic - Florida - Neurology | Jacksonville | Florida | 32224 | United States |
| University of South Florida (USF) - Morsani Center (USF Health Carol and Frank Morsani Center for Advanced Healthcare) | Tampa | Florida | 33616 | United States |
| Emory Healthcare - Emory Clinic - Rheumatology | Atlanta | Georgia | 30322 | United States |
| Northwestern Medicine - Northwestern Memorial Hospital | Chicago | Illinois | 60611 | United States |
| John Hopkins Medicine - Johns Hopkins Myositis Center | Baltimore | Maryland | 21224-2734 | United States |
| Northwell Health Physician Partners Rheumatology | Great Neck | New York | 11021 | United States |
| University of North Carolina (UNC) School of Medicine | Chapel Hill | North Carolina | 27514 | United States |
| The Ohio State University | Columbus | Ohio | 43201 | United States |
| Oregon Health and Science University | Portland | Oregon | 97239 | United States |
| University of Pittsburgh Medical Center | Pittsburgh | Pennsylvania | 15261 | United States |
| Austin Neuromuscular Center (National Neuromuscular Research Institute, PLLC) | Austin | Texas | 78759 | United States |
| Nerve And Muscle Center Of Texas | Houston | Texas | 77030 | United States |
| Framingham Centro Medico | La Plata | B1902COS | Argentina |
| Centro Dermatologico Schejtman | San Miguel | B1663GKT | Argentina |
| Centro Medico Privado de Reumatologia | San Miguel de Tucumán | 4000 | Argentina |
| Tirol Kliniken - A.o. Landeskrankenhaus Innsbruck - Neurology | Innsbruck | 6020 | Austria |
| AZ Sint-Lucas - Campus Sint-Lucas - Neurology | Ghent | 9000 | Belgium |
| Universitair Ziekenhuis Leuven Gasthuisberg Campus | Leuven | 3000 | Belgium |
| Medical Centre Artmed | Plovdiv | 4002 | Bulgaria |
| Multiprofile hospital for active treatment Kaspela EOOD | Plovdiv | 4002 | Bulgaria |
| Nanfang Hospital, Southern Medical University | Guangzhou | Guangdong | 510515 | China |
| Nanjing University Medical School - Taikang Xianlin Drum Tower Hospital (Nanjing Drum Tower Hospital) | Nanjing | Jingsu | 210000 | China |
| Peking University Third Hospital | Beijing | 100191 | China |
| Peking Union Medical College Hospital | Beijing | 100730 | China |
| Sichuan Provincial Academy of Medical Sciences and Sichuan Provincial People's Hospital | Chengdu | 610072 | China |
| Shanghai Jiao Tong University School of Medicine - Renji Hospital - Shanghai Institute of Digestive Disease (SIDD) | Shanghai | 200001 | China |
| Huashan Hospital Affiliated to Fudan University | Shanghai | 200040 | China |
| China Medical University (CMU) - First Affiliated Hospital | Shenyang | 110001 | China |
| First Affiliated Hospital (Henan Medical University - First Affiliated Hospital)-Zhengzhou University | Zhengzhou | 450052 | China |
| Cyprus Institute of Neurology and Genetics | Nicosia | 2371 | Cyprus |
| Revmatologicky Ustav | Prague | 12850 | Czechia |
| Copenhagen University Hospital-Rigshospitalet University Hospital | Copenhagen | 2100 | Denmark |
| Reference Center Neuro-Muscular Diseases - CHU Paris Group Hospitalier La Pitie Salpetriere-Charles Foix | Paris | 75013 | France |
| Aversi Clinic | Tbilisi | 0160 | Georgia |
| LLC MediClub Georgia | Tbilisi | 0160 | Georgia |
| New Hospitals | Tbilisi | 0162 | Georgia |
| The First Medical Center | Tbilisi | 0180 | Georgia |
| Charite Universitaetsmedizin Berlin - Campus Charite Mitte (CCM) - Rheumatology | Berlin | 10117 | Germany |
| Universitaetsklinikum Hamburg-Eppendorf | Hamburg | 20246 | Germany |
| Rheumazentrum Ruhrgebiet | Herne | 44649 | Germany |
| Universitatsmedizin Mannheim GmbH | Mannheim | 68167 | Germany |
| Universitaetsklinikum Tuebingen (UKT) | Tübingen | 72076 | Germany |
| Klinikum der Universitaet Ulm | Ulm | 89081 | Germany |
| National and Kapodistrian University of Athens - School of Health Sciences - Faculty of Medicine | Athens | 115 27 | Greece |
| National and Kapodistrian University of Athens - Eginition Hospital | Athens | 115 28 | Greece |
| ATTIKON University General Hospital - Neurology | Athens | 12462 | Greece |
| University College Dublin (UCD) - St. Vincent's University Hospital (SVUH) | Dublin | D04 T6F4 | Ireland |
| Azienda Ospedaliero Universitaria Consorziale Policlinico | Bari | 70124 | Italy |
| Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - Ospedale San Raffaele Hospital | Milan | 20132 | Italy |
| Azienda Ospedaliero Universitaria Pisana - Ospedale Santa Chiara - Rheumatology | Pisa | 56126 | Italy |
| Ospedale Sant'Andrea Hospital | Roma | 00189 | Italy |
| Fondazione Policlinico Universitario Agostino Gemelli - Neurology | Roma | 168 | Italy |
| Azienda sanitaria universitaria Friuli Centrale - ASUFC | Udine | 33100 | Italy |
| Juntendo University Hospital | Bunkyō City | 113-8431 | Japan |
| The University of Tokyo Hospital | Bunkyō City | 113-8655 | Japan |
| Japan Community Healthcare Organization Chukyo Hospital | Nagoya | 457-8510 | Japan |
| Institute of Science Tokyo Hospital | Tokyo | 113-8519 | Japan |
| Wakayama Medical University Hospital | Wakayama | 641-8510 | Japan |
| Santaros University Clinic | Vilnius | 08410 | Lithuania |
| Hospital Angeles Clinica Londres | Mexico City | 06700 | Mexico |
| Amsterdam UMC Research BV | Amsterdam | 1105 AZ | Netherlands |
| Clinica San Juan de Dios - sede Arequipa - Rheumatology | Arequipa | 4000 | Peru |
| Instituto Peruano Del Hueso Y La Articulacion Sac-Privado-Lima,Centro De Investigacion Iphar | Lima | 27 | Peru |
| Investigaciones Clinicas S.A.C. | Lima | 33 | Peru |
| USK-WAM Centralny Szpital Weteranow - Szpital Kniniczny ul. Zeromskiego 113 - Rheumatology | Lodz | 90-549 | Poland |
| Centrum Wsparcia Badan Klinicznych Pomorskiego Uniwersytetu Medycznego w Szczecinie (CWBK PUM) - Rheumatology | Szczecin | 71-252 | Poland |
| Centro Hospitalar do Porto - Hospital Santo Antonio | Porto | 4099-001 | Portugal |
| Clinical Center of Serbia | Belgrade | 11000 | Serbia |
| Institute of Rheumatology | Belgrade | 11000 | Serbia |
| Seoul National University Hospital | Seoul | 03080 | South Korea |
| Hospital de la Santa Creu i Sant Pau | Barcelona | 08041 | Spain |
| Hospital Universitario 12 de Octubre | Madrid | 28041 | Spain |
| Hospital Universitari i Politecnic La Fe de Valencia - Neurology | Valencia | 46026 | Spain |
| Karolinska Universitestssjukhus Solna | Solna | 17176 | Sweden |
| Chung Shan Medical University Hospital | Taichung | 402 | Taiwan |
| Taichung Veterans General Hospital | Taichung | Taiwan |
| King Chulalongkorn Memorial Hospital - Neurology | Bangkok | 10330 | Thailand |
| Phramongkutklao Hospital | Bangkok | 10400 | Thailand |
| Ramathibodi hospital | Bangkok | 10400 | Thailand |
| Siriraj Hospital, Mahidol University of Internal medicine | Bangkok | 10700 | Thailand |
| Faculty Of Medicine, Khon Kaen University | Khon Kaen | 40002 | Thailand |
| Aintree University Hospital - Liverpool University Hospitals NHS Foundation Trust | Liverpool | L9 7AL | United Kingdom |
| The Royal Free Hospital | London | NW3 2QG | United Kingdom |
| Northern Care Allicance NHS Foundation Trust - Salford Royal - Rheumatology | Salford | M6 8HD | United Kingdom |
| ID | Term |
|---|---|
| D009220 | Myositis |
| D003882 | Dermatomyositis |
| D017285 | Polymyositis |
| C537778 | Antisynthetase syndrome |
| ID | Term |
|---|---|
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D012871 | Skin Diseases |
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