Evaluating a new treatment for adults with dermatomyositis

A Phase 2, Randomized, Double-Blinded, Placebo-Controlled, Multicenter Study to Evaluate the Safety, Tolerability, and Efficacy of Empasiprubart in Adults With Dermatomyositis

This study is testing a new treatment called empasiprubart to see if it can help adults with dermatomyositis feel better compared to a placebo.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 42 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: argenx (Industry-sponsored)
• Locations: 24 sites (Oceanside, California and 23 other locations)
• Trial ID: NCT06284954 on ClinicalTrials.gov

What this trial studies

This study evaluates the safety and efficacy of empasiprubart compared to a placebo in adults diagnosed with dermatomyositis. Participants will be randomly assigned to receive either the treatment or placebo over a 25-week period, followed by a 65-week safety follow-up. The study aims to determine if empasiprubart can effectively manage the symptoms and underlying issues associated with this condition. The total duration of the study for participants is approximately 92 weeks.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older with a clinical diagnosis of dermatomyositis or juvenile dermatomyositis and active muscle disease.

Not a fit: Patients without a diagnosis of dermatomyositis or those who do not have active muscle disease may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve the management of dermatomyositis in affected adults.

How similar studies have performed: Other studies have explored treatments for dermatomyositis, but the specific approach of empasiprubart is novel and has not been widely tested.

Eligibility criteria

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

Where this trial is running

Oceanside, California and 23 other locations

• Profound Research LLC — Oceanside, California, United States (Recruiting)
• Omega Research Debary, LLC — Debary, Florida, United States (Recruiting)
• Homestead Associates in Research, Inc. — Homestead, Florida, United States (Recruiting)
• Life Clinical Trials — Margate, Florida, United States (Recruiting)
• Advance Medical Research Center — Miami, Florida, United States (Recruiting)
• Integral Rheumatology and Immunology Specialists (IRIS) — Plantation, Florida, United States (Recruiting)
• D and H Tamarac Research, LLC Center — Tamarac, Florida, United States (Recruiting)
• Wright State Physicians Health Center — Dayton, Ohio, United States (Recruiting)
• V.Tsitlanadze Scientific Practical Reumatology Center — Tbilisi, Georgia (Recruiting)
• The First University Clinic of Tbilisi State Medical University — Tbilisi, Georgia (Recruiting)
• Institute of Clinical Cardiology, Ltd — Tbilisi, Georgia (Recruiting)
• Jerarsi Clinic — Tbilisi, Georgia (Recruiting)
• Mtskheta street Clinic — Tbilisi, Georgia (Recruiting)
• The First Medical Center — Tbilisi, Georgia (Recruiting)
• National and Kapodistrian University of Athens (NKUA) - University General Hospital Attikon — Athens, Greece (Recruiting)
• Andreas Syggros Hospital of Skin and Venereal Diseases - University Dermatology and Venereology — Athens, Greece (Recruiting)
• Fondazione Policlinico Universitario Campus Bio-Medico — Rome, Italy (Recruiting)
• Timofei Mosneaga Republican Clinical Hospital — Chisinau, Moldova, Republic of (Recruiting)
• Institute of Cardiology — Chisinau, Moldova, Republic of (Recruiting)
• Nova Reuma Domyslawska i Rusilowicz- Spolka Partnerska Lekarza Reumatologa i Fizjoterapeuty — Bialystok, Poland (Recruiting)
• Prywatna Praktyka Lekarska Prof. UM dr Hab. Med. Pawel Hrycaj — Poznan, Poland (Recruiting)
• Parc de Salut Mar - Hospital del Mar — Barcelona, Spain (Recruiting)
• Hospital Quironsalud Infanta Luisa — Sevilla, Spain (Recruiting)
• Hospital Universitario Rio Hortega — Valladolid, Spain (Recruiting)

Study contacts

• Study coordinator: Sabine Coppieters, MD

Conditions Dermatomyositis; Myositis