MyositisPhase 3ALKIVIA+NCT05979441
A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Active Idiopathic Inflammatory Myopathy
A Phase 3 study testing Efgartigimod PH20 SC in people with Myositis and Active Idiopathic Inflammatory Myopathy, run by argenx at 92 sites in 29 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myositis, Active Idiopathic Inflammatory Myopathy, Dermatomyositis
- What is being tested?
- Efgartigimod PH20 SC
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 240 participants
- Where
- 92 sitesin 29 countries
- Study dates
- 12 Sep 2023to Sep 2028
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
92 sites in 29 countries. Contact the study team or a site near you to ask about taking part.
92 sites in 29 countries
United States18 sites
Emory Healthcare - Emory Clinic - Rheumatology
Atlanta, Georgia, 30322
Austin Neuromuscular Center (National Neuromuscular Research Institute, PLLC)
Austin, Texas, 78759
John Hopkins Medicine - Johns Hopkins Myositis Center
Baltimore, Maryland, 21224-2734
Attune Health Research, Inc
Beverly Hills, California, 90039
University of North Carolina (UNC) School of Medicine
Chapel Hill, North Carolina, 27514
Northwestern Medicine - Northwestern Memorial Hospital
Chicago, Illinois, 60611
The Ohio State University
Columbus, Ohio, 43201
UF Health - Rheumatology Medical Specialties Medical Plaza
Gainesville, Florida, 32610
Northwell Health Physician Partners Rheumatology
Great Neck, New York, 11021
Nerve And Muscle Center Of Texas
Houston, Texas, 77030
Mayo Clinic - Florida - Neurology
Jacksonville, Florida, 32224
Yale Cancer Center-Yale University School Of Medicine
New Haven, Connecticut, 06520
UCI Health - ALS and Neuromuscular Center - Neurology
Orange, California, 92868
Neuromuscular Clinical and Research Center - Neurology
Phoenix, Arizona, 85028
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15261
Oregon Health and Science University
Portland, Oregon, 97239
UCSF Health - ALS and Neurodegenerative Disease Center - Dermatology
San Francisco, California, 94143
University of South Florida (USF) - Morsani Center (USF Health Carol and Frank Morsani Center for Advanced Healthcare)
Tampa, Florida, 33616
China9 sites
Peking University Third Hospital
Beijing, 100191
Peking Union Medical College Hospital
Beijing, 100730
Sichuan Provincial Academy of Medical Sciences and Sichuan Provincial People's Hospital
Chengdu, 610072
Nanfang Hospital, Southern Medical University
Guangzhou, Guangdong, 510515
Nanjing University Medical School - Taikang Xianlin Drum Tower Hospital (Nanjing Drum Tower Hospital)
Nanjing, Jingsu, 210000
Shanghai Jiao Tong University School of Medicine - Renji Hospital - Shanghai Institute of Digestive Disease (SIDD)
Shanghai, 200001
Huashan Hospital Affiliated to Fudan University
Shanghai, 200040
China Medical University (CMU) - First Affiliated Hospital
Shenyang, 110001
First Affiliated Hospital (Henan Medical University - First Affiliated Hospital)-Zhengzhou University
Zhengzhou, 450052
Italy7 sites
Azienda Ospedaliero Universitaria Consorziale Policlinico
Bari, 70124
Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - Ospedale San Raffaele Hospital
Milan, 20132
Azienda Ospedaliero Universitaria Pisana - Ospedale Santa Chiara - Rheumatology
Pisa, 56126
Ospedale Sant'Andrea Hospital
Roma, 00189
Fondazione Policlinico Universitario Agostino Gemelli - Neurology
Roma, 168
IRCCS Istituto Clinico Humanitas
Rozzano, 20089
Azienda sanitaria universitaria Friuli Centrale - ASUFC
Udine, 33100
Germany6 sites
Charite Universitaetsmedizin Berlin - Campus Charite Mitte (CCM) - Rheumatology
Berlin, 10117
Universitaetsklinikum Hamburg-Eppendorf
Hamburg, 20246
Rheumazentrum Ruhrgebiet
Herne, 44649
Universitatsmedizin Mannheim GmbH
Mannheim, 68167
Universitaetsklinikum Tuebingen (UKT)
Tübingen, 72076
Klinikum der Universitaet Ulm
Ulm, 89081
Japan6 sites
Juntendo University Hospital
Bunkyō City, 113-8431
The University of Tokyo Hospital
Bunkyō City, 113-8655
Japan Community Healthcare Organization Chukyo Hospital
Nagoya, 457-8510
Institute of Science Tokyo Hospital
Tokyo, 113-8519
Wakayama Medical University Hospital
Wakayama, 641-8510
Yamaguchi University Hospital
Yamaguchi, 755-8505
Thailand5 sites
King Chulalongkorn Memorial Hospital - Neurology
Bangkok, 10330
Phramongkutklao Hospital
Bangkok, 10400
Ramathibodi hospital
Bangkok, 10400
Siriraj Hospital, Mahidol University of Internal medicine
Bangkok, 10700
Faculty Of Medicine, Khon Kaen University
Khon Kaen, 40002
Georgia4 sites
Aversi Clinic
Tbilisi, 0160
LLC MediClub Georgia
Tbilisi, 0160
New Hospitals
Tbilisi, 0162
The First Medical Center
Tbilisi, 0180
Argentina3 sites
Framingham Centro Medico
La Plata, B1902COS
Centro Dermatologico Schejtman
San Miguel, B1663GKT
Centro Medico Privado de Reumatologia
San Miguel de Tucumán, 4000
Greece3 sites
National and Kapodistrian University of Athens - School of Health Sciences - Faculty of Medicine
Athens, 115 27
National and Kapodistrian University of Athens - Eginition Hospital
Athens, 115 28
ATTIKON University General Hospital - Neurology
Athens, 12462
Peru3 sites
Clinica San Juan de Dios - sede Arequipa - Rheumatology
Arequipa, 4000
Instituto Peruano Del Hueso Y La Articulacion Sac-Privado-Lima,Centro De Investigacion Iphar
Lima, 27
Investigaciones Clinicas S.A.C.
Lima, 33
Spain3 sites
Hospital de la Santa Creu i Sant Pau
Barcelona, 08041
Hospital Universitario 12 de Octubre
Madrid, 28041
Hospital Universitari i Politecnic La Fe de Valencia - Neurology
Valencia, 46026
United Kingdom3 sites
Aintree University Hospital - Liverpool University Hospitals NHS Foundation Trust
Liverpool, L9 7AL
The Royal Free Hospital
London, NW3 2QG
Northern Care Allicance NHS Foundation Trust - Salford Royal - Rheumatology
Salford, M6 8HD
Belgium2 sites
AZ Sint-Lucas - Campus Sint-Lucas - Neurology
Ghent, 9000
Universitair Ziekenhuis Leuven Gasthuisberg Campus
Leuven, 3000
Bulgaria2 sites
Medical Centre Artmed
Plovdiv, 4002
Multiprofile hospital for active treatment Kaspela EOOD
Plovdiv, 4002
Poland2 sites
USK-WAM Centralny Szpital Weteranow - Szpital Kniniczny ul. Zeromskiego 113 - Rheumatology
Lodz, 90-549
Centrum Wsparcia Badan Klinicznych Pomorskiego Uniwersytetu Medycznego w Szczecinie (CWBK PUM) - Rheumatology
Szczecin, 71-252
Serbia2 sites
Clinical Center of Serbia
Belgrade, 11000
Institute of Rheumatology
Belgrade, 11000
Taiwan2 sites
Chung Shan Medical University Hospital
Taichung, 402
Taichung Veterans General Hospital
Taichung
Austria1 site
Tirol Kliniken - A.o. Landeskrankenhaus Innsbruck - Neurology
Innsbruck, 6020
Denmark1 site
Copenhagen University Hospital-Rigshospitalet University Hospital
Copenhagen, 2100
France1 site
Reference Center Neuro-Muscular Diseases - CHU Paris Group Hospitalier La Pitie Salpetriere-Charles Foix
Paris, 75013
Ireland1 site
University College Dublin (UCD) - St. Vincent's University Hospital (SVUH)
Dublin, D04 T6F4
Portugal1 site
Centro Hospitalar do Porto - Hospital Santo Antonio
Porto, 4099-001
About this study
Official title: A Phase 3, Single-Arm, Multicenter, Open-label Extension of Study ARGX-113-2007 to Investigate the Long-term Safety, Tolerability, and Efficacy of Efgartigimod PH20 SC in Participants Aged 18 Years and Older With Active Idiopathic Inflammatory Myopathy
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.
- Conditions
- Myositis, Active Idiopathic Inflammatory Myopathy, Dermatomyositis, Polymyositis, Immune-Mediated Necrotizing Myopathy, Antisynthetase Syndrome
- Being tested
- Efgartigimod PH20 SC
- Medical subject headings
- Myositis, Dermatomyositis, Polymyositis, Antisynthetase syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Efgartigimod PH20 SCBiological
Subcutaneous injection of Efgartigimod PH20 given by vials or prefilled syringe (PFS)
| Study group | Role | What they receive |
|---|---|---|
| Efgartigimod PH20 SC | Experimental | Participants receiving efgartigimod PH20 subcutaneously Receives: Biological: Efgartigimod PH20 SC |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of AEs, SAEs and AESIs over time
AE : adverse event ; SAE : serious adverse event ; AESI : adverse event of special interest.
Measured: Up to 53 months
Secondary outcomes (21)
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.
Measured: 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.
Measured: 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.
Measured: Up to 51 months
Prednisone dose reduction (average monthly dose) over time
Measured: 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.
Measured: 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
Measured: 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).
Measured: 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).
Measured: 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
Measured: 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."
Measured: 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".
Measured: 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.
Measured: 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".
Measured: 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).
Measured: Up to 51 months
Percent change from baseline in total IgG levels in serum over time
Measured: Up to 53 months
Incidence of ADA against efgartigimod (serum levels) over time
ADA : anti-drug antibodies.
Measured: 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.
Measured: 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"
Measured: 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"
Measured: 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"
Measured: Up to 51 months
Duration of dosing every 2 weeks
Measured: Up to 51 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Aug 2023
- Study start12 Sep 2023
- Primary completionSep 2028 (estimated)
- Study completionSep 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MyositisStudies of the same condition, open and under way.
- More trials run by argenxThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 240 participants (estimated)
Sponsor and investigators
- Lead sponsor
- argenx
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05979441
- Sponsor's protocol ID
- ARGX-113-2011
- Other IDs
- 2022-502851-79-00
- Acronym
- ALKIVIA+
- Status last verified
- Jul 2026
- First submitted
- 28 Jul 2023
- Last updated
- 16 Oct 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided