A Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants With Active Idiopathic Inflammatory Myopathy.

NCT ID: NCT05523167

Last Updated: 2025-07-23

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2/PHASE3

Total Enrollment

265 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-10-12

Study Completion Date

2027-02-01

Brief Summary

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This study's purpose is to measure the treatment response from efgartigimod PH20 SC compared with placebo in participants with Idiopathic Inflammatory Myopathy (IIM). Participants with the IIM subtypes of dermatomyositis (DM), immune-mediated necrotizing myopathy (IMNM), or certain other subtypes of polymyositis (PM; including antisynthetase syndrome \[ASyS\]) will be included in the study. Treatment response will be measured by Total improvement score (TIS). Additional information can be found on https://myositis-study.com/.

Detailed Description

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Conditions

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Active Idiopathic Inflammatory Myopathy Myositis Dermatomyositis Polymyositis Immune-Mediated Necrotizing Myopathy Antisynthetase Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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EFG PH20 SC

participants receiving efgartigimod PH20 SC on top of background treatment

Group Type EXPERIMENTAL

EFG PH20 SC

Intervention Type BIOLOGICAL

Subcutaneous injection of efgartigimod coformulated with rHuPH20, a permeation enhancer

PBO PH20 SC

participants receiving placebo PH20 SC on top of background treatment

Group Type PLACEBO_COMPARATOR

PBO

Intervention Type OTHER

Subcutaneous injection of placebo coformulated with rHuPH20, a permeation enhancer

Interventions

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EFG PH20 SC

Subcutaneous injection of efgartigimod coformulated with rHuPH20, a permeation enhancer

Intervention Type BIOLOGICAL

PBO

Subcutaneous injection of placebo coformulated with rHuPH20, a permeation enhancer

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

* Ability to consent in the jurisdiction in which the study is taking place and capable of giving signed informed consent.
* A definite or probable clinical diagnosis of idiopathic inflammatory myopathy (IIM)
* One of the following medical histories: Diagnosis of dermatomyositis (DM) or juvenile dermatomyositis (JDM), Diagnosis of polymyositis (PM) (including antisynthetase syndrome (ASyS)), Diagnosis of immune-mediated necrotizing myopathy (IMNM)
* Diagnosed with active disease as defined by the presence of at least 1 of the following criteria: Abnormal levels of at least 1 of the following enzymes: creatine kinase (CK), aldolase, lactate dehydrogenase, aspartate aminotransaminase (AST), alanine aminotransferase (ALT), based on central laboratory results; Electromyography demonstrating active disease within the past 3 months; Active dermatomyositis (DM) skin rash; Muscle biopsy indicative of active idiopathic inflammatory myopathy (IIM) in the past 3 months; Magnetic resonance imaging within the past 3 months indicative of active inflammation
* Muscle weakness
* Receiving a permitted background treatment for idiopathic inflammatory myopathy.
* Contraceptive use consistent with local regulations, where available, for individuals participating in clinical studies. Women of childbearing potential must have a negative serum pregnancy test during screening and a negative urine pregnancy test at baseline before receiving investigational medicinal product (IMP).

Exclusion Criteria

* A clinically significant active infection at screening
* A COVID-19 polymerase chain reaction (PCR)-positive test before enrollment
* Any other known autoimmune disease that, in the investigator's opinion, would interfere with an accurate assessment of clinical symptoms of idiopathic inflammatory myopathy (IIM) or put the patient at undue risk
* A history of malignancy unless considered cured by adequate treatment, with no evidence of recurrence for ≥ 3 years before the first administration of the investigational medicinal product (IMP). Adequately treated participants with the following cancers can be included at any time: Basal cell or squamous cell skin cancer ; Carcinoma in situ of the cervix; Carcinoma in situ of the breast; Incidental histological finding of prostate cancer
* Severe muscle damage
* Glucocorticoid-induced myopathy that the investigator considers the primary cause of muscle weakness or permanent weakness linked to a non-idiopathic inflammatory myopathy (IIM) cause
* Juvenile myositis (JDM) diagnosed \> 5 years from screening or juvenile myositis with extensive calcinosis or severe calcinosis.
* Uncontrolled interstitial lung disease or any other uncontrolled idiopathic inflammatory myopathy (IIM) manifestation that, in the opinion of the investigator, would be likely to require treatment with prohibited medication during the study
* Other inflammatory and noninflammatory myopathies: inclusion body myositis, overlap myositis), metabolic myopathies, muscle dystrophies or a family history of muscle dystrophy, drug-induced or endocrine induced myositis, and juvenile myositis (other than juvenile dermatomyositis (JDM))
* Clinically significant disease, recent major surgery or intends to have surgery during the study, or has any other condition in the opinion of the investigator that could confound the results of the trial or put the patient at undue risk
* Known hypersensitivity reaction to investigational medicinal product (IMP) or 1 of its excipients
* Received a live or live-attenuated vaccine less than 4 weeks before screening.
* Positive serum test at screening for active viral infection with any of the following conditions: Hepatitis B virus (HBV); Hepatitis C virus (HCV); HIV
* Participant has previously participated in an efgartigimod clinical trial and received at least 1 dose of investigational medicinal product (IMP).
* Participant is concurrently participating in any other clinical study, including a noninterventional study.
* Participant has a current or history (ie, within 12 months of screening) of alcohol, drug, or medication abuse.
* Participant is pregnant or lactating or intends to become pregnant during the study.
* Participant has severe renal impairment .
* Participant is institutionalized by a court or other governmental order or is in a dependent relationship with the sponsor or investigator.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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argenx

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Neuromuscular Research Center

Phoenix, Arizona, United States

Site Status

HonorHealth Neurology - Bob Bove Neuroscience Institute - Neurology

Scottsdale, Arizona, United States

Site Status

Attune Health Research, Inc

Beverly Hills, California, United States

Site Status

University of Southern California Norris Comprehensive Cancer Center

Los Angeles, California, United States

Site Status

Profound Research LLC - Oceanside

Oceanside, California, United States

Site Status

University of California, Irvine

Orange, California, United States

Site Status

Eisenhower Medical Center

Rancho Mirage, California, United States

Site Status

Stanford Medicine Outpatient Center - Stanford Dermatology Clinic-Stanford University School of Medicine

Redwood City, California, United States

Site Status

California Pacific Medical Center - Sutter Health

San Francisco, California, United States

Site Status

Amyotrophic Lateral Sclerosis (ALS) Treatment Center,University of California San Francisco (UCSF)

San Francisco, California, United States

Site Status

Denver Arthritis Clinic

Denver, Colorado, United States

Site Status

Yale Cancer Center-Yale University School Of Medicine

New Haven, Connecticut, United States

Site Status

Georgetown University Hospital

Washington D.C., District of Columbia, United States

Site Status

UF Health Rheumatology

Gainesville, Florida, United States

Site Status

Mayo Clinic

Jacksonville, Florida, United States

Site Status

University of South Florida (USF) - Morsani Center (USF Health Carol and Frank Morsani Center for Advanced Healthcare)

Tampa, Florida, United States

Site Status

Emory University Hospital, The Emory Clinic

Atlanta, Georgia, United States

Site Status

Augusta University

Augusta, Georgia, United States

Site Status

Northwestern Memorial Hospital

Chicago, Illinois, United States

Site Status

University Of Kansas Medical Center

Kansas City, Kansas, United States

Site Status

Johns Hopkins Medicine - Johns Hopkins Myositis Center

Baltimore, Maryland, United States

Site Status

Harvard Medical School - Brigham and Women's Hospital (BWH) - The Schuster Family Transplantation Research Center (TRC)

Boston, Massachusetts, United States

Site Status

Michigan State University - Neurology

East Lansing, Michigan, United States

Site Status

St. Paul Rheumatology, PA

Saint Paul, Minnesota, United States

Site Status

Washington University School of Medicine

St Louis, Missouri, United States

Site Status

Northwell Health

Great Neck, New York, United States

Site Status

Hospital for Special Surgery

New York, New York, United States

Site Status

University of North Carolina (UNC) School of Medicine

Chapel Hill, North Carolina, United States

Site Status

Carolina Arthritis Associates

Wilmington, North Carolina, United States

Site Status

Cleveland Clinic - Main Campus

Cleveland, Ohio, United States

Site Status

The Ohio State University

Columbus, Ohio, United States

Site Status

Mercy Hospital - Mercy Clinic Neurology Oklahoma City - Neurology

Oklahoma City, Oklahoma, United States

Site Status

Oregon Health and Science University

Portland, Oregon, United States

Site Status

University of Pittsburg Medical Center

Pittsburgh, Pennsylvania, United States

Site Status

Vanderbilt University Medical Center (VUMC) - Vanderbilt Rheumatology Clinic

Nashville, Tennessee, United States

Site Status

Austin Neuromuscular Center

Austin, Texas, United States

Site Status

McGovern Medical School -The University of Texas Health Science Center at Houston

Houston, Texas, United States

Site Status

Nerve And Muscle Center Of Texas

Houston, Texas, United States

Site Status

University of Vermont Medical Center - Main Campus

Burlington, Vermont, United States

Site Status

University of Washington Medical Center

Seattle, Washington, United States

Site Status

Consultora Integral de Salud

Córdoba, , Argentina

Site Status

Framingham Centro Medico

La Plata, , Argentina

Site Status

Dim Clinica Privada

Ramos Mejía, , Argentina

Site Status

Centro Dermatologico Schejtman

San Miguel, , Argentina

Site Status

Centro de Investigaciones Medicas Tucuman

San Miguel de Tucumán, , Argentina

Site Status

The Wesley Medical Research

Auchenflower, , Australia

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Fiona Stanley Hospital

Murdoch, , Australia

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Medical University Innsbruck

Innsbruck, , Austria

Site Status

Allgemeines Krankenhaus der Stadt Wien (AKH Wien)

Vienna, , Austria

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Centre Hospitalier Universitaire de Charleroi

Charleroi, , Belgium

Site Status

AZ Sint Lucas Gent-Campus Sint Lucas

Ghent, , Belgium

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Universitair Ziekenhuis Leuven Gasthuisberg Campus

Leuven, , Belgium

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Centre Hospitalier Universitaire Sart Tilman

Liège, , Belgium

Site Status

Medical Centre Artmed

Plovdiv, , Bulgaria

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Multiprofile hospital for active treatment Kaspela EOOD

Plovdiv, , Bulgaria

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Genge Partners

Montreal, , Canada

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Ottawa Hospital Research Institute-Civic Campus

Ottawa, , Canada

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Peking University First Hospital

Beijing, , China

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Peking University Third Hospital

Beijing, , China

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Peking Union Medical College Hospital

Beijing, , China

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The First Hospital of Jilin University

Changchun, , China

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Xiangya Hospital Central South University

Changsha, , China

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The Second Xiangya Hospital of Central South University

Changsha, , China

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Sichuan Provincial Peoples Hospital

Chengdu, , China

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Sun Yat-Sen Memorial Hospital, Sun Yat-Sen University

Guangzhou, , China

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Nanfang Hospital, Southern Medical University

Guangzhou, , China

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The First Affiliated Hospital - School of Medicine - ZheJiang University

Hangzhou, , China

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Anhui Provincial Hospital

Hefei, , China

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The Second Affiliated Hospital Of Nanchang University

Nanchang, , China

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Nanjing Drum Tower Hospital

Nanjing, , China

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Huashan Hospital Affiliated to Fudan University

Shanghai, , China

Site Status

Ruijin Hospital, Shanghai Jiaotong University School of Medicine

Shanghai, , China

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Shanghai Renji Hospital

Shanghai, , China

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First Affiliated Hospital of Shanxi Medical College

Shanxi, , China

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China Medical University (CMU) - First Affiliated Hospital

Shenyang, , China

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Huazhong University of Science and Technology - Tongji Medical College - Tongji Hospital

Wuhan, , China

Site Status

First Affiliated Hospital (Henan Medical University - First Affiliated Hospital)-Zhengzhou University

Zhengzhou, , China

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Cyprus Institute of Neurology and Genetics

Nicosia, , Cyprus

Site Status

Revmatologicky ustav

Prague, , Czechia

Site Status

Copenhagen University Hospital-Rigshospitalet University Hospital

Copenhagen, , Denmark

Site Status

Hopital Edouard Herriot

Lyon, , France

Site Status

Reference Center Neuro-Muscular Diseases - CHU Paris Group Hospitalier La Pitie Salpetriere-Charles Foix

Paris, , France

Site Status

Centre Hospitalier Universitaire (CHU) Hopitaux de Rouen

Rouen, , France

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Hopitaux Universitaire de Strasbourg-Centre de References des Maladies Autoimmunes

Strasbourg, , France

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American Hospital Network

Tbilisi, , Georgia

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LTD New Hospitals

Tbilisi, , Georgia

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Aversi Clinic

Tbilisi, , Georgia

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LLC MediClub Georgia

Tbilisi, , Georgia

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The First Medical Center

Tbilisi, , Georgia

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LTD Tbilisi Heart Center

Tbilisi, , Georgia

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Charite - Universitaetsmedizin Berlin

Berlin, , Germany

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Universitaetsklinikum Duesseldorf

Düsseldorf, , Germany

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Universitatsmedizin Gottingen - Georg-August-Universitat

Göttingen, , Germany

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Universitaetsklinikum Hamburg-Eppendorf

Hamburg, , Germany

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Rheumazentrum Ruhrgebiet

Herne, , Germany

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Universitatsmedizin Mannheim

Mannheim, , Germany

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Ludwig-Maximilians-Universitat Munchen Friedrich-Baur-Institut Neurologische Klinik und Poliklinik

München, , Germany

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St. Josef-Stift Sendenhorst

Sendenhorst, , Germany

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Universitaetsklinikum Tuebingen (UKT)

Tübingen, , Germany

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Universitatsklinikum Ulm - Standort Oberer Eselsberg

Ulm, , Germany

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National and Kapodistrian University of Athens - School of Health Sciences - Faculty of Medicine

Athens, , Greece

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National and Kapodistrian University of Athens - Eginition Hospital

Athens, , Greece

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Attikon General University Hospital

Athens, , Greece

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Athens Medical Center

Athens, , Greece

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University of Ioannina Medical School

Ioannina, , Greece

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Semmelweis Egyetem, Institute of Genomic Medicine and Rare Disorders

Budapest, , Hungary

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Debreceni Egyetem - University of Debrecen

Debrecen, , Hungary

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St. Vincents University Hospital

Dublin, , Ireland

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Connolly Hospital Blanchardstown

Dublin, , Ireland

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North West Rheumatology Unit - Our Lady's Hospital

Manorhamilton, , Ireland

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Bnai Zion Medical Center

Haifa, , Israel

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Rambam Health Care Campus

Haifa, , Israel

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Meir Medical Center

Kfar Saba, , Israel

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Rabin Medical Center - Beilinson Campus (Beilinson and Hasharon Hospital)

Petah Tikva, , Israel

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Sheba Medical Center - Rheumatology

Ramat Gan, , Israel

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Azienda Ospedaliera Universitaria Policlinico Consorziale Di Bari - Rheumatology

Bari, , Italy

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Azienda Ospedaliero Universitaria Careggi

Florence, , Italy

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Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - Ospedale San Raffaele Hospital

Milan, , Italy

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San Matteo Hospital

Pavia, , Italy

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A.O. Universitaria Pisana

Pisa, , Italy

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IRCCS Arcispedale Santa Maria Nuova

Reggio Emilia, , Italy

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Fondazione Policlinico Universitario Agostino Gemelli IRCCS - Universita Cattolica del Sacro Cuore

Roma, , Italy

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Azienda Ospedaliero Universitaria Sant'Andrea

Roma, , Italy

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IRCCS Istituto Clinico Humanitas - Rheumatology

Rozzano, , Italy

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Azienda Ospedaliero Universitaria Citta della Salute e della Scienza di Torino - Presidio Molinette

Torino, , Italy

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ASU FC - Presidio Ospedaliero Universitario Santa Maria della Misericordia

Udine, , Italy

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Azienda Ospedaliera Universitaria Integrata di Verona - Ospedale Borgo Roma

Verona, , Italy

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Tokyo Medical And Dental University Hospital, Faculty of Medicine

Bunkyō City, , Japan

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Nippon Medical School - Nippon Medical School Hospital

Bunkyō City, , Japan

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St. Marianna University School of Medicine Hospital

Kawasaki, , Japan

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Kurume University Hospital

Kurume, , Japan

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Chiba-Nishi General Hospital

Matsudo-shi, , Japan

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Japan Community Healthcare Organization Chukyo Hospital

Nagoya, , Japan

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Kitasato University Hospital (KUH)

Sagamihara-shi, , Japan

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Sapporo City Hospital

Sapporo, , Japan

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Hokkaido University Hospital - Rheumatology

Sapporo, , Japan

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Sasebo Chuo Hospital

Sasebo-shi, , Japan

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Tohoku University Hospital

Sendai, , Japan

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Tokushima University Hospital

Tokushima, , Japan

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Juntendo University Hospital - Rheumatology

Tokyo, , Japan

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The University of Tokyo Hospital

Tokyo, , Japan

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Keio University Hospital

Tokyo, , Japan

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University of Tsukuba Hospital - Rheumatology

Tsukuba, , Japan

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Yamaguchi University Hospital

Ube-shi, , Japan

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Wakayama Medical University Hospital - Dermatology

Wakayama, , Japan

Site Status

Hospital of Lithuanian University of Health Sciences Kauno klinikos

Kaunas, , Lithuania

Site Status

Vilnius University Hospital Santaros Klinikos

Vilnius, , Lithuania

Site Status

CITER - Centro de Investigacion y Tratamiento de las Enfermedades Reumaticas S.A. de C.V.

Mexico City, , Mexico

Site Status

Centro Peninsular De Investigacion Clinica

Mérida, , Mexico

Site Status

Amsterdam University Medical Center (Amsterdam UMC), Academic Medical Center (AMC)

Amsterdam, , Netherlands

Site Status

Universitair Medisch Centrum Utrecht (UMC Utrecht)

Utrecht, , Netherlands

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Hogar Clinica San Juan de Dios

Arequipa, , Peru

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Investigaciones Clinicas S.A.C.

Lima, , Peru

Site Status

Instituto Peruano Del Hueso Y La Articulacion Sac-Privado-Lima, Centro De Investigacion Iphar

Lima, , Peru

Site Status

Hospital Nacional Cayetano Heredia

Lima, , Peru

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Szpital Uniwersytecki nr 2 im. Dr Jana Biziela w Bydgoszczy

Bydgoszcz, , Poland

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USK-WAM Centralny Szpital Weteranow - Szpital Kniniczny ul. Zeromskiego 113

Lodz, , Poland

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Centrum Wsparcia Badan Klinicznych Pomorski Uniwersytet Medyczny w Szczecinie

Szczecin, , Poland

Site Status

Centro Hospitalar Lisboa Norte, E.P.E- Hospital Santa Maria

Lisbon, , Portugal

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Centro Hospitalar Universitario de Santo Antonio

Porto, , Portugal

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Clinical Center of Serbia

Belgrade, , Serbia

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Institute of Rheumatology

Belgrade, , Serbia

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Narodny ustav reumatickych chorob

Piešťany, , Slovakia

Site Status

Chungnam National University Hospital (CNUH)

Daejeon, , South Korea

Site Status

Gachon University Gil Medical Center

Incheon, , South Korea

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Kyung Hee University Medical Center

Seoul, , South Korea

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Seoul National University Hospital

Seoul, , South Korea

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Hanyang University Seoul Hospital

Seoul, , South Korea

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Ajou University Hospital

Suwon, , South Korea

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Hospital General Universitario de Alicante

Alicante, , Spain

Site Status

Hospital Clinic de Barcelona - Sede Villarroel - Rheumatology

Barcelona, , Spain

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Hospital de la Santa Creu i Sant Pau

Barcelona, , Spain

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Hospital Universitario Ramon y Cajal

Madrid, , Spain

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Universidad Autonoma de Madrid (UAM) - Hospital Universitario La Paz

Madrid, , Spain

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Hospital Universitario 12 de Octubre

Madrid, , Spain

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Hospital De Valme

Seville, , Spain

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Hospital Universitario y Politecnico La Fe

Valencia, , Spain

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Hospital Do Meixoeiro

Vigo, , Spain

Site Status

Karolinska Universitestssjukhus Solna

Stockholm, , Sweden

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Neurocenter of Southern Switzerland

Lugano, , Switzerland

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Chung Shan Medical University Hospital

Taichung, , Taiwan

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China Medical University and Hospital

Taichung, , Taiwan

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Taichung Veterans General Hospital

Taichung, , Taiwan

Site Status

Chulalongkorn University - Faculty of Medicine - King Chulalongkorn Memorial Hospital (KCMH)

Bangkok, , Thailand

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Phramongkutklao Hospital

Bangkok, , Thailand

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Ramathibodi hospital

Bangkok, , Thailand

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Siriraj Hospital, Mahidol University of Internal medicine

Bangkok, , Thailand

Site Status

Maharaj Nakorn Chiang Mai Hospital, Chiang Mai University

Chiang Mai, , Thailand

Site Status

Khon Kaen University

Khon Kaen, , Thailand

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Thammasat University Hospital

Pathum Thani, , Thailand

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Ankara University Medical Faculty

Ankara, , Turkey (Türkiye)

Site Status

Ankara City Hospital

Ankara, , Turkey (Türkiye)

Site Status

Necmettin Erbakan University Meram Medical Faculty

Konya, , Turkey (Türkiye)

Site Status

Royal National Hospital for Rheumatic Diseases - Royal United Hospitals Bath NHS Foundation Trust

Bath, , United Kingdom

Site Status

Doncaster Royal Infirmary - Doncaster And Bassetlaw Hospitals Nhs Foundation Trust

Doncaster, , United Kingdom

Site Status

Leeds Institute of Rheumatic & Musculoskeletal Medicine - University of Leeds

Leeds, , United Kingdom

Site Status

Aintree University Hospital - Liverpool University Hospitals NHS Foundation Trust

Liverpool, , United Kingdom

Site Status

The Royal Free Hospital - Royal Free London NHS Foundation Trust

London, , United Kingdom

Site Status

Kings College Hospital NHS Foundation Trust

London, , United Kingdom

Site Status

The National Hospital for Neurology and Neurosurgery

London, , United Kingdom

Site Status

James Cook University Hospital - South Tees Hospitals NHS Foundation Trust

Middlesbrough, , United Kingdom

Site Status

Salford Royal Hospital, Northern Care Alliance NHS Foundation Trust

Salford, , United Kingdom

Site Status

Countries

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United States Argentina Australia Austria Belgium Bulgaria Canada China Cyprus Czechia Denmark France Georgia Germany Greece Hungary Ireland Israel Italy Japan Lithuania Mexico Netherlands Peru Poland Portugal Serbia Slovakia South Korea Spain Sweden Switzerland Taiwan Thailand Turkey (Türkiye) United Kingdom

Related Links

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Other Identifiers

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2024-512785-33-00

Identifier Type: CTIS

Identifier Source: secondary_id

ARGX-113-2007

Identifier Type: -

Identifier Source: org_study_id

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