A Study to Evaluate Safety and Efficacy of Empasiprubart in Adults With Dermatomyositis

NCT06284954 | Active Not Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: ARGX-117-23012023-508337-14
• Study Type: interventional
• Target: 3
• Locations: 7 countries
• Sites: 28

Brief Summary

This study will evaluate the safety and efficacy of empasiprubart compared with placebo in adult participants with dermatomyositis (DM). The study duration will be approximately 92 weeks for all participants. After the screening period, eligible participants will be randomized in a 2:1 ratio to receive either empasiprubart or placebo, respectively, during the treatment period (duration of 25 weeks). At the end of the treatment period, all the participants will enter a safety follow-up period (duration of 65 weeks).

Conditions

Dermatomyositis; Myositis

Outcome Measures

Primary Outcomes (2)

• Incidence of adverse events (AEs)

  Up to 90 weeks

• Percentage of participants discontinuing investigational medicinal product (IMP) due to an adverse event (AE)

  Up to 25 weeks

Secondary Outcomes (1)

• Mean TIS

  Up to 25 weeks

Study Arms (2)

Empasiprubart

EXPERIMENTAL

Patients receiving Empasiprubart IV

Biological: Empasiprubart IV

Placebo

PLACEBO COMPARATOR

Patients receiving Placebo IV

Other: Placebo IV

Interventions

Empasiprubart IV BIOLOGICAL

Intravenous infusion with Empasiprubart IV

Also known as: ARGX-117

Empasiprubart

Placebo IV OTHER

Intravenous infusion with Placebo IV

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Is at least 18 years of age and the local legal age of consent for clinical studies when signing the Informed Consent Form
• Is capable of providing signed informed consent and complying with protocol requirements
• Agrees to use contraceptive measures consistent with local regulations and women of child-bearing potential must have a negative serum pregnancy test at screening and a negative urine pregnancy test before receiving the study drug
• Has a clinical diagnosis of dermatomyositis or juvenile dermatomyositis. The diagnosis date for juvenile dermatomyositis should be ≤5 years before screening
• Has active muscle disease associated with classic dermatomyositis or juvenile dermatomyositis at screening and before the first study drug adminisitration and at least 1 of the following: elevated levels of creatine kinase, aldolase, lactate dehydrogenase, aspartate aminotransaminase or alanine aminotransferase at screening; or electromyography ≤18 weeks before the first study drug administration; or an MRI depicting active muscle inflammation ≤18 weeks before the first study drug administration; or muscle biopsy demonstrating signs of active inflammation ≤18 weeks before the first study drug administration
• Has at least mild skin disease at screening
• Complies with the permitted background dermatomyositis treatment requirements at screening
• Has had immunization with the first meningococcal, pneumococcal, and the single Haemophilus influenza type B vaccine ≥14 days before the first study drug administration

You may not qualify if:

• Known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of dermatomyositis or puts the participant at undue risk
• Naïve to standard dermatomyositis treatment according to local recommendations
• History of malignancy unless considered cured by adequate treatment with no evidence of recurrence for ≥3 years before the first study drug administration. 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 findings of prostate cancer
• Clinically significant active infection that is not sufficiently resolved before the first study drug administration in the investigator's opinion
• Positive serum test at screening for active infection with any of the following: Hepatitis B virus, Hepatitis C virus, HIV
• Clinically significant disease, recent major surgery, or intention to have major surgery during the study; or any other medical condition that, in the investigator's opinion, would confound the results of the study or put the participant at undue risk
• Current participation in another interventional clinical study
• Known hypersensitivity to the study drug or any of its excipients
• History (within 12 months before screening) of or current alcohol, drug, or medication abuse, as assessed by the investigator
• Pregnant or lactating state or intending to become pregnant during the study
• Previous participation in an empasiprubart clinical study with at least 1 dose of study drug received
• Known complement component deficiency as assessed by the investigator
• Change in dermatomyositis physical therapy or exercise program from ≤4 weeks before screening
• Paraneoplastic dermatomyositis secondary to malignancy
• Glucocorticoid-induced myopathy

Sponsors & Collaborators

• argenx: lead

Study Sites (28)

Profound Research LLC

Oceanside, California, 92056, United States

Location

Omega Research Debary, LLC

DeBary, Florida, 32713, United States

Location

University of Florida Health (UF) - Endocrinology - Medical Specialties - Medical Plaza

Gainesville, Florida, 32610, United States

Location

Homestead Associates in Research, Inc.

Homestead, Florida, 33033, United States

Location

Life Clinical Trials

Margate, Florida, 33063, United States

Location

Advance Medical Research Center

Miami, Florida, 33135, United States

Location

Integral Rheumatology and Immunology Specialists (IRIS)

Plantation, Florida, 33324, United States

Location

D and H Tamarac Research, LLC Center

Tamarac, Florida, 33321, United States

Location

Wright State Physicians Health Center

Dayton, Ohio, 45324, United States

Location

V.Tsitlanadze Scientific Practical Reumatology Center

Tbilisi, 0102, Georgia

Location

The First University Clinic of Tbilisi State Medical University

Tbilisi, 0141, Georgia

Location

Institute of Clinical Cardiology, Ltd

Tbilisi, 0159, Georgia

Location

Jerarsi Clinic

Tbilisi, 0167, Georgia

Location

Mtskheta street Clinic

Tbilisi, 0179, Georgia

Location

The First Medical Center

Tbilisi, 0180, Georgia

Location

National and Kapodistrian University of Athens (NKUA) - University General Hospital Attikon

Athens, 12462, Greece

Location

Andreas Syggros Hospital of Skin and Venereal Diseases - University Dermatology and Venereology

Athens, 16121, Greece

Location

General Hospital of Thessaloniki Papageorgiou

Thessaloniki, 56429, Greece

Location

Azienda Ospedaliera Universitaria Pisana

Pisa, 56126, Italy

Location

Fondazione Policlinico Universitario Campus Bio-Medico

Rome, 00128, Italy

Location

Timofei Mosneaga Republican Clinical Hospital

Chisinau, 2025, Moldova

Location

Institute of Cardiology

Chisinau, MD2025, Moldova

Location

Nova Reuma Domyslawska i Rusilowicz- Spolka Partnerska Lekarza Reumatologa i Fizjoterapeuty

Bialystok, 15707, Poland

Location

Prywatna Praktyka Lekarska Prof. UM dr Hab. Med. Pawel Hrycaj

Poznan, 61-397, Poland

Location

Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu

Wroclaw, 50-556, Poland

Location

Parc de Salut Mar - Hospital del Mar

Barcelona, 08003, Spain

Location

Hospital Quironsalud Infanta Luisa

Seville, 41010, Spain

Location

Hospital Universitario Rio Hortega

Valladolid, 47012, Spain

Location

MeSH Terms

Conditions

Dermatomyositis; Myositis

Condition Hierarchy (Ancestors)

Polymyositis; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 22, 2024
• First Posted: February 29, 2024
• Study Start: August 20, 2024
• Primary Completion (Estimated): November 1, 2026
• Study Completion (Estimated): November 1, 2026
• Last Updated: September 25, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

ES (3); PL (3); GE (6); IT (2); MO (2); GR (3); US (9)