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

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

This study will evaluate the safety and efficacy of empasiprubart compared with placebo in adult participants with dermatomyositis (DM).

Study Overview

• Status: Not yet recruiting
• Conditions: Myositis; Dermatomyositis
• Intervention / Treatment: Biological: Empasiprubart IV; Other: Placebo IV

• Study Type: Interventional
• Enrollment (Estimated): 80
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Sabine Coppieters, MD; Phone Number: 857-350-4834; Email: clinicaltrials@argenx.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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 at least 1 of the following: elevated levels of creatine kinase, aldolase, lactate dehydrogenase, aspartate aminotransaminase or alanine aminotransferase at screening; or electromyography ≤12 weeks before screening; or an MRI depicting active muscle inflammation ≤12 weeks before screening; or muscle biopsy demonstrating signs of active inflammation ≤12 weeks before screening
• 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
• Interstitial lung disease with 1 of the following: forced vital capacity (FVC) ≤60%; supplemental oxygen therapy; rapidly progressing uncontrolled interstitial lung disease; moderate or severe interstitial lung disease

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Dose regimen 1; Patients receiving Empasiprubart IV	Biological: Empasiprubart IV; Intravenous infusion with Empasiprubart IV; Other Names: ARGX-117 concentrate for solution
Experimental: Dose regimen 2; Patients receiving Empasiprubart IV	Biological: Empasiprubart IV; Intravenous infusion with Empasiprubart IV; Other Names: ARGX-117 concentrate for solution
Experimental: Dose regimen 3; Patients receiving Empasiprubart IV	Biological: Empasiprubart IV; Intravenous infusion with Empasiprubart IV; Other Names: ARGX-117 concentrate for solution
Placebo Comparator: Dose regimen 4; Patients receiving Placebo IV	Other: Placebo IV; Intravenous infusion with Placebo IV

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Incidence and severity of adverse events (AEs)	Up to 112 weeks
Percentage of participants discontinuing investigational medicinal product (IMP) due to an adverse event (AE)	Up to 52 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Mean TIS	The Total Improvement Score (TIS) assesses minimal, moderate, and major clinical response categorically or continuously using American College of Rheumatology and European League Against Rheumatism (ACR/EULAR) criteria.	Up to 52 weeks

Collaborators and Investigators

• Sponsor: argenx

Study record dates

Study Major Dates

• Study Start (Estimated): May 10, 2024
• Primary Completion (Estimated): July 18, 2025
• Study Completion (Estimated): January 20, 2028

Study Registration Dates

• First Submitted: February 22, 2024
• First Submitted That Met QC Criteria: February 22, 2024
• First Posted (Actual): February 29, 2024

Study Record Updates

• Last Update Posted (Actual): March 15, 2024
• Last Update Submitted That Met QC Criteria: March 13, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Skin Diseases; Musculoskeletal Diseases; Connective Tissue Diseases; Muscular Diseases; Neuromuscular Diseases; Polymyositis; Myositis; Dermatomyositis
• Other Study ID Numbers: ARGX-117-2301; 2023-508337-14 (Other Identifier: CTIS number)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No