A Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants With Active Idiopathic Inflammatory Myopathy. (ALKIVIA)

argenx

Status and phase: Completed; Phase 3; Phase 2

Conditions: Dermatomyositis; Myositis; Polymyositis; Immune-Mediated Necrotizing Myopathy; Antisynthetase Syndrome; Active Idiopathic Inflammatory Myopathy

Treatments: Biological: EFG PH20 SC; Other: PBO

Study type: Interventional

Funder types: Industry

Identifiers

NCT05523167

ARGX-113-2007

2024-512785-33-00 (EU Trial (CTIS) Number)

Details and patient eligibility

About

This study's purpose is to measure the treatment response from efgartigimod PH20 SC compared with placebo in participants with Idiopathic Inflammatory Myopathy (IIM). Participants with the IIM subtypes of dermatomyositis (DM), immune-mediated necrotizing myopathy (IMNM), or certain other subtypes of polymyositis (PM; including antisynthetase syndrome [ASyS]) will be included in the study. Treatment response will be measured by Total improvement score (TIS).

Enrollment: 265 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Ability to consent in the jurisdiction in which the study is taking place and capable of giving signed informed consent.
• A definite or probable clinical diagnosis of idiopathic inflammatory myopathy (IIM)
• One of the following medical histories: Diagnosis of dermatomyositis (DM) or juvenile dermatomyositis (JDM), Diagnosis of polymyositis (PM) (including antisynthetase syndrome (ASyS)), Diagnosis of immune-mediated necrotizing myopathy (IMNM)
• Diagnosed with active disease as defined by the presence of at least 1 of the following criteria: Abnormal levels of at least 1 of the following enzymes: creatine kinase (CK), aldolase, lactate dehydrogenase, aspartate aminotransaminase (AST), alanine aminotransferase (ALT), based on central laboratory results; Electromyography demonstrating active disease within the past 3 months; Active dermatomyositis (DM) skin rash; Muscle biopsy indicative of active idiopathic inflammatory myopathy (IIM) in the past 3 months; Magnetic resonance imaging within the past 3 months indicative of active inflammation
• Muscle weakness
• Receiving a permitted background treatment for idiopathic inflammatory myopathy.
• Contraceptive use consistent with local regulations, where available, for individuals participating in clinical studies. Women of childbearing potential must have a negative serum pregnancy test during screening and a negative urine pregnancy test at baseline before receiving investigational medicinal product (IMP).

Exclusion criteria

• Any other known autoimmune disease that, in the investigator's opinion, would interfere with an accurate assessment of clinical symptoms of idiopathic inflammatory myopathy (IIM) or put the patient at undue risk
• A history of malignancy unless considered cured by adequate treatment, with no evidence of recurrence for ≥ 3 years before the first administration of the investigational medicinal product (IMP). 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 finding of prostate cancer
• Severe muscle damage
• Glucocorticoid-induced myopathy that the investigator considers the primary cause of muscle weakness or permanent weakness linked to a non-idiopathic inflammatory myopathy (IIM) cause
• Uncontrolled interstitial lung disease or any other uncontrolled idiopathic inflammatory myopathy (IIM) manifestation that, in the opinion of the investigator, would be likely to require treatment with prohibited medication during the study
• Participant is pregnant or lactating or intends to become pregnant during the study.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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265 participants in 2 patient groups, including a placebo group

EFG PH20 SC

Experimental group

Description:

participants receiving efgartigimod PH20 SC on top of background treatment

Treatment:

Biological: EFG PH20 SC

PBO PH20 SC

Placebo Comparator group

Description:

participants receiving placebo PH20 SC on top of background treatment

Treatment:

Other: PBO