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

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ClinicalTrials.gov Identifier: NCT05523167
Recruitment Status : Recruiting
First Posted : August 31, 2022
Last Update Posted : April 8, 2024
Sponsor:
Information provided by (Responsible Party):
argenx

Study Description
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Brief Summary:
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). Additional information can be found on https://myositis-study.com/.

Condition or disease Intervention/treatment Phase
Active Idiopathic Inflammatory Myopathy Myositis Dermatomyositis Polymyositis Immune-Mediated Necrotizing Myopathy Antisynthetase Syndrome Biological: EFG PH20 SC Other: PBO Phase 2 Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 240 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : October 12, 2022
Estimated Primary Completion Date : December 1, 2026
Estimated Study Completion Date : February 1, 2027

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: EFG PH20 SC
participants receiving efgartigimod PH20 SC on top of background treatment
 Biological: EFG PH20 SC
Subcutaneous injection of efgartigimod coformulated with rHuPH20, a permeation enhancer
Placebo Comparator: PBO PH20 SC
participants receiving placebo PH20 SC on top of background treatment
 Other: PBO
Subcutaneous injection of placebo coformulated with rHuPH20, a permeation enhancer


Outcome Measures
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Primary Outcome Measures :
  1. Total improvement score (TIS); measured on a [0,100] scale. Higher scores represent improvement; zero indicates no improvement or worsening (from baseline). [ Time Frame: phase 2: 24 weeks; phase 3: 52 weeks ]

Secondary Outcome Measures :
  1. Time to reach TIS ≥ 20 (first "minimal clinical improvement") [ Time Frame: phase 2: up to 24 weeks; phase 3: up to 52 weeks ]
  2. Percentage of participants with TIS ≥ 20 [ Time Frame: phase 2: 24 weeks; phase 3: 52 weeks ]
  3. Time to reach TIS ≥ 40 (first "moderate clinical improvement") [ Time Frame: phase 2: up to 24 weeks; phase 3: up to 52 weeks ]
  4. Percentage of participants with TIS ≥ 40 [ Time Frame: phase 2: 24 weeks; phase 3: 52 weeks ]
  5. Change in manual muscle testing-8 (MMT8) score [ Time Frame: phase 2: 24 weeks; phase 3: 52 weeks ]
  6. Change in Patient Global Assessment of Disease Activity (PGA) [ Time Frame: phase 2: 24 weeks; phase 3: 52 weeks ]
  7. Change in Physician Global Assessment of Disease Activity (MDGA) [ Time Frame: phase 2: 24 weeks; phase 3: 52 weeks ]
  8. Proportion of participants achieving target dose of ≤ 5 mg (prednisone equivalent) [ Time Frame: phase 2: 24 weeks; phase 3: 52 weeks ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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).

The full list of inclusion criteria can be found in the protocol.

Exclusion Criteria:

  • A clinically significant active infection at screening
  • A COVID-19 polymerase chain reaction (PCR)-positive test before enrollment
  • 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
  • Juvenile myositis (JDM) diagnosed > 5 years from screening or juvenile myositis with extensive calcinosis or severe calcinosis.
  • 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
  • Other inflammatory and noninflammatory myopathies: inclusion body myositis, overlap myositis), metabolic myopathies, muscle dystrophies or a family history of muscle dystrophy, drug-induced or endocrine induced myositis, and juvenile myositis (other than juvenile dermatomyositis (JDM))
  • Clinically significant disease, recent major surgery or intends to have surgery during the study, or has any other condition in the opinion of the investigator that could confound the results of the trial or put the patient at undue risk
  • Known hypersensitivity reaction to investigational medicinal product (IMP) or 1 of its excipients
  • Received a live or live-attenuated vaccine less than 4 weeks before screening.
  • Positive serum test at screening for active viral infection with any of the following conditions: Hepatitis B virus (HBV); Hepatitis C virus (HCV); HIV
  • Participant has previously participated in an efgartigimod clinical trial and received at least 1 dose of investigational medicinal product (IMP).
  • Participant is concurrently participating in any other clinical study, including a noninterventional study.
  • Participant has a current or history (ie, within 12 months of screening) of alcohol, drug, or medication abuse.
  • Participant is pregnant or lactating or intends to become pregnant during the study.
  • Participant has severe renal impairment .
  • Participant is institutionalized by a court or other governmental order or is in a dependent relationship with the sponsor or investigator.

The full list of exclusion criteria can be found in the protocol.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05523167


Contacts
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Contact: Sabine Coppieters, MD 857-350-4834 ClinicalTrials@argenx.com

Locations
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Sponsors and Collaborators
argenx
More Information
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Additional Information:

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Responsible Party: argenx
ClinicalTrials.gov Identifier: NCT05523167    
Other Study ID Numbers: ARGX-113-2007
First Posted: August 31, 2022    Key Record Dates
Last Update Posted: April 8, 2024
Last Verified: April 2024

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Muscular Diseases
Dermatomyositis
Myositis
Polymyositis
Musculoskeletal Diseases
 Neuromuscular Diseases
Nervous System Diseases
Connective Tissue Diseases
Skin Diseases