New drug hopes to tame rare muscle and skin disease

NCT ID NCT06284954

Disease control Sponsor: argenx Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a drug called empasiprubart in adults with dermatomyositis, a rare disease causing muscle weakness and skin rash. Three participants receive either the drug or a placebo for 25 weeks, then are followed for 65 more weeks. The goal is to see if the drug is safe and helps improve symptoms.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: empasiprubart (also called ARGX-117), given as an intravenous infusion
What this could lead to: If it works, this could point toward a new treatment option for dermatomyositis, a rare autoimmune disease that causes muscle weakness and skin rash.
What could go wrong: This is a very early Phase 2 trial with only 3 participants, so results may not apply to everyone. The drug may not work better than placebo, and side effects are still being studied.

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Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

3 people

The number who actually took part.

Started

Aug 2024

Expected to finish

Nov 2026

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * 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 Exclusion Criteria: * 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 * Inflammatory or non-inflammatory myopathies other than dermatomyositis, such as drug-induced or endocrine-induced myositis, infective myositis, polymyositis, immune-mediated necrotizing myopathy, inclusion body myositis, overlap myositis, metabolic myopathies, or muscle dystrophies * Paraneoplastic dermatomyositis secondary to malignancy * Glucocorticoid-induced myopathy * Severe muscle damage * Extensive or severe calcinosis * Interstitial lung disease with at least 1 of the following: forced vital capacity (FVC) ≤60%; supplemental oxygen therapy; rapidly progressing uncontrolled interstitial lung disease; moderate or severe interstitial lung disease

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Conditions

The condition(s) this trial relates to.

dermatomyositis juvenile dermatomyositis myositis disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Dermatomyositis Myositis

Contacts and locations

Locations

Advance Medical Research Center

Miami, Florida, 33135, United States
Andreas Syggros Hospital of Skin and Venereal Diseases - University Dermatology and Venereology

Athens, 16121, Greece
Azienda Ospedaliera Universitaria Pisana

Pisa, 56126, Italy
D and H Tamarac Research, LLC Center

Tamarac, Florida, 33321, United States
Fondazione Policlinico Universitario Campus Bio-Medico

Rome, 00128, Italy
General Hospital of Thessaloniki Papageorgiou

Thessaloniki, 56429, Greece
Homestead Associates in Research, Inc.

Homestead, Florida, 33033, United States
Hospital Quironsalud Infanta Luisa

Seville, 41010, Spain
Hospital Universitario Rio Hortega

Valladolid, 47012, Spain
Institute of Cardiology

Chisinau, MD2025, Moldova
Institute of Clinical Cardiology, Ltd

Tbilisi, 0159, Georgia
Integral Rheumatology and Immunology Specialists (IRIS)

Plantation, Florida, 33324, United States
Jerarsi Clinic

Tbilisi, 0167, Georgia
Life Clinical Trials

Margate, Florida, 33063, United States
Mtskheta street Clinic

Tbilisi, 0179, Georgia
National and Kapodistrian University of Athens (NKUA) - University General Hospital Attikon

Athens, 12462, Greece
Nova Reuma Domyslawska i Rusilowicz- Spolka Partnerska Lekarza Reumatologa i Fizjoterapeuty

Bialystok, 15707, Poland
Omega Research Debary, LLC

DeBary, Florida, 32713, United States
Parc de Salut Mar - Hospital del Mar

Barcelona, 08003, Spain
Profound Research LLC

Oceanside, California, 92056, United States
Prywatna Praktyka Lekarska Prof. UM dr Hab. Med. Pawel Hrycaj

Poznan, 61-397, Poland
The First Medical Center

Tbilisi, 0180, Georgia
The First University Clinic of Tbilisi State Medical University

Tbilisi, 0141, Georgia
Timofei Mosneaga Republican Clinical Hospital

Chisinau, 2025, Moldova
University of Florida Health (UF) - Endocrinology - Medical Specialties - Medical Plaza

Gainesville, Florida, 32610, United States
Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu

Wroclaw, 50-556, Poland
V.Tsitlanadze Scientific Practical Reumatology Center

Tbilisi, 0102, Georgia
Wright State Physicians Health Center

Dayton, Ohio, 45324, United States

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