Skip to main content
OpenTrials
Completed

NCT Number: NCT05523167

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

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).

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Active Idiopathic Inflammatory Myopathy Antisynthetase Syndrome Connective Tissue Diseases Dermatomyositis Immune-Mediated Necrotizing Myopathy Muscular Diseases Musculoskeletal Diseases Myositis Nervous System Diseases Neuromuscular Diseases Polymyositis Skin Diseases Skin and Connective Tissue Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Consultora Integral de Salud, Córdoba, Argentina

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

EFG PH20 SC

Biological

Subcutaneous injection of efgartigimod coformulated with rHuPH20, a permeation enhancer

PBO

Other

Subcutaneous injection of placebo coformulated with rHuPH20, a permeation enhancer

Primary outcomes

  1. Mean TIS

    Time frame: phase 2: 24 weeks; phase 3: 52 weeks

    The Total improvement score (TIS) assesses minimal, moderate, and major clinical response, and is assessed using the ACR/EULAR criteria. It is measured on a 0 to 100 scale. Higher scores represent improvement; zero indicates no improvement or worsening (from baseline).

Secondary outcomes

  1. Time to reach TIS ≥ 20 (first "minimal clinical improvement")

    Time frame: phase 2: up to 24 weeks; phase 3: up to 52 weeks

    The Total improvement score (TIS) assesses minimal, moderate, and major clinical response, and is assessed using the ACR/EULAR criteria. It is measured on a 0 to 100 scale. Higher scores represent improvement; zero indicates no improvement or worsening (from baseline).

  2. Percentage of participants with TIS ≥ 20

    Time frame: phase 2: up to 24 weeks; phase 3: up to 52 weeks

    The Total improvement score (TIS) assesses minimal, moderate, and major clinical response, and is assessed using the ACR/EULAR criteria. It is measured on a 0 to 100 scale. Higher scores represent improvement; zero indicates no improvement or worsening (from baseline).

  3. Time to reach TIS ≥ 40 (first "moderate clinical improvement")

    Time frame: phase 2: up to 24 weeks; phase 3: up to 52 weeks

    The Total improvement score (TIS) assesses minimal, moderate, and major clinical response, and is assessed using the ACR/EULAR criteria. It is measured on a 0 to 100 scale. Higher scores represent improvement; zero indicates no improvement or worsening (from baseline).

  4. Percentage of participants with TIS ≥ 40

    Time frame: phase 2: up to 24 weeks; phase 3: up to 52 weeks

    The Total improvement score (TIS) assesses minimal, moderate, and major clinical response, and is assessed using the ACR/EULAR criteria. It is measured on a 0 to 100 scale. Higher scores represent improvement; zero indicates no improvement or worsening (from baseline).

  5. Change in MMT8 score

    Time frame: phase 2: up to 24 weeks; phase 3: up to 52 weeks

    The manual muscle testing-8 (MMT8) is a physician assessment of muscle strength in a set of 8 designated muscles tested bilaterally and axially. The highest total potential MMT8 score is 150

  6. Change in Patient Global Assessment of Disease Activity (PGA)

    Time frame: phase 2: up to 24 weeks; phase 3: up to 52 weeks

    The Patient Global Assessment of Disease Activity (PGA) is a tool that measures a patient's global evaluation of their overall disease activity at the time of assessment using a 10-cm VAS. The participant rates their overall disease activity by drawing a vertical mark on a 10-cm VAS from the left end of the line (no evidence of disease activity) to the right end of the line (extremely active or severe disease activity).

  7. Change in Physician Global Assessment of Disease Activity (MDGA)

    Time frame: phase 2: up to 24 weeks; phase 3: up to 52 weeks

    The Physician Global Assessment of Disease Activity (MDGA) is a tool that measures the physician's global evaluation of the participant's overall disease activity, defined as potentially reversible pathology or physiology resulting from IIM. The physician rates disease activity on the MDGA using a 10-cm VAS. Overall disease activity is rated by drawing a vertical mark on a 10-cm VAS from the left end of the line (no evidence of disease activity), midpoint of the line (moderate disease activity), and the right end of the line (extremely active or severe disease activity).

  8. Proportion of participants who have at least moderate improvement (≥40) in TIS at week 52 and adhere to an oral prednisone dosage of ≤5 mg/day (or equivalent) from week 44 onward

    Time frame: Phase 3: up to 52 weeks

    The Total improvement score (TIS) assesses minimal, moderate, and major clinical response, and is assessed using the ACR/EULAR criteria. It is measured on a 0 to 100 scale. Higher scores represent improvement; zero indicates no improvement or worsening (from baseline).

  9. Change in CK abnormality grades at week 52

    Time frame: Phase 3: up to 52 weeks

    CK: creatine kinase

  10. Change in CDASI activity score at week 52

    Time frame: Phase 3: up to 52 weeks

    The Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) is a skin-specific outcome measure used to assess disease in patients with Dermatomyositis (DM). Disease in 15 different anatomical locations is rated using 3 activity measures (erythema, scale, erosion/ulceration) and 2 damage measures (poikiloderma, calcinosis). The resulting activity and damage scores range from 0 to 100 and 0 to 32, respectively. Higher scores indicate greater disease severity.

Sponsors and collaborators

Lead sponsor

argenx

Industry

Registry information

Official study title

A Phase 2/3, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Group, 2-Arm, Multicenter, Operationally Seamless Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacodynamics, Pharmacokinetics, and Immunogenicity of Efgartigimod PH20 SC in Participants Aged 18 Years and Older With Active Idiopathic Inflammatory Myopathy

Acronym: ALKIVIA

Important dates

Study start
2022
Primary completion
2026
Study completion
2026
First posted
Aug 31, 2022
Registry last updated
Jul 24, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.