Clinical trial NCT06284954: A Study to Evaluate Safety and Efficacy of Empasiprubart in Adults With Dermatomyositis
Official title: A Phase 2, Randomized, Double-Blinded, Placebo-Controlled, Multicenter Study to Evaluate the Safety, Tolerability, and Efficacy of Empasiprubart in Adults With Dermatomyositis
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Dermatomyositis
Myositis
Lead sponsor: argenx
Sponsor class: INDUSTRY
First posted: 2024-02-29
Enrollment: 42
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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).


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

Primary outcomes:
measure: Incidence of adverse events (AEs)
timeFrame: Up to 90 weeks
measure: Percentage of participants discontinuing investigational medicinal product (IMP) due to an adverse event (AE)
timeFrame: Up to 25 weeks
Secondary outcomes:
measure: Mean TIS
timeFrame: Up to 25 weeks
Other outcomes:
Locations:
city: Oceanside
country: United States
facility: Profound Research LLC
state: California
city: Debary
country: United States
facility: Omega Research Debary, LLC
state: Florida
city: Homestead
country: United States
facility: Homestead Associates in Research, Inc.
state: Florida
city: Margate
country: United States
facility: Life Clinical Trials
state: Florida
city: Miami
country: United States
facility: Advance Medical Research Center
state: Florida
city: Plantation
country: United States
facility: Integral Rheumatology and Immunology Specialists (IRIS)
state: Florida
city: Tamarac
country: United States
facility: D and H Tamarac Research, LLC Center
state: Florida
city: Dayton
country: United States
facility: Wright State Physicians Health Center
state: Ohio
city: Tbilisi
country: Georgia
facility: V.Tsitlanadze Scientific Practical Reumatology Center
city: Tbilisi
country: Georgia
facility: The First University Clinic of Tbilisi State Medical University
city: Tbilisi
country: Georgia
facility: Institute of Clinical Cardiology, Ltd
city: Tbilisi
country: Georgia
facility: Jerarsi Clinic
city: Tbilisi
country: Georgia
facility: Mtskheta street Clinic
city: Tbilisi
country: Georgia
facility: The First Medical Center
city: Athens
country: Greece
facility: National and Kapodistrian University of Athens (NKUA) - University General Hospital Attikon
city: Athens
country: Greece
facility: Andreas Syggros Hospital of Skin and Venereal Diseases - University Dermatology and Venereology
city: Thessaloníki
country: Greece
facility: General Hospital of Thessaloniki Papageorgiou
city: Pisa
country: Italy
facility: Azienda Ospedaliera Universitaria Pisana
city: Rome
country: Italy
facility: Fondazione Policlinico Universitario Campus Bio-Medico
city: Chisinau
country: Moldova, Republic of
facility: Timofei Mosneaga Republican Clinical Hospital
city: Chisinau
country: Moldova, Republic of
facility: Institute of Cardiology
city: Bialystok
country: Poland
facility: Nova Reuma Domyslawska i Rusilowicz- Spolka Partnerska Lekarza Reumatologa i Fizjoterapeuty
city: Poznan
country: Poland
facility: Prywatna Praktyka Lekarska Prof. UM dr Hab. Med. Pawel Hrycaj
city: Wrocław
country: Poland
facility: Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu
city: Barcelona
country: Spain
facility: Parc de Salut Mar - Hospital del Mar
city: Sevilla
country: Spain
facility: Hospital Quironsalud Infanta Luisa
city: Valladolid
country: Spain
facility: Hospital Universitario Rio Hortega
Study officials:
References: