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

Study Purpose

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/.

Recruitment Criteria

Accepts Healthy Volunteers Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms	No
Study Type An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes. Searching Both is inclusive of interventional and observational studies.	Interventional
Eligible Ages	18 Years and Over
Gender	All

More Inclusion & Exclusion 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.

Trial Details

Trial ID: This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.	NCT05523167
Phase Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans. Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data. Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs. Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.	Phase 2/Phase 3
Lead Sponsor The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.	argenx
Principal Investigator The person who is responsible for the scientific and technical direction of the entire clinical study.	N/A
Principal Investigator Affiliation	N/A
Agency Class Category of organization(s) involved as sponsor (and collaborator) supporting the trial.	Industry
Overall Status	Recruiting
Countries	Argentina, Australia, Austria, Belgium, Brazil, Bulgaria, Canada, China, Cyprus, Czechia, Denmark, France, Georgia, Germany, Greece, Hungary, Ireland, Israel, Italy, Japan, Korea, Republic of, Latvia, Lithuania, Mexico, Netherlands, Peru, Poland, Portugal, Romania, Russian Federation, Serbia, Slovakia, Spain, Sweden, Switzerland, Taiwan, Thailand, Turkey, Ukraine, United Kingdom, United States
Conditions The disease, disorder, syndrome, illness, or injury that is being studied.	Active Idiopathic Inflammatory Myopathy, Myositis, Dermatomyositis, Polymyositis, Immune-Mediated Necrotizing Myopathy, Antisynthetase Syndrome
Study Website:	View Trial Website

Arms & Interventions

Arms

Experimental: EFG PH20 SC

participants receiving efgartigimod PH20 SC on top of background treatment

Placebo Comparator: PBO PH20 SC

participants receiving placebo PH20 SC on top of background treatment

Interventions

Biological: - EFG PH20 SC

Subcutaneous injection of efgartigimod coformulated with rHuPH20, a permeation enhancer

Other: - PBO

Subcutaneous injection of placebo coformulated with rHuPH20, a permeation enhancer

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Neuromuscular Research Center, Phoenix, Arizona

Status

Recruiting

Address

Neuromuscular Research Center

Phoenix, Arizona, 85028

Site Contact

Kumaraswamy Sivakumar, MD

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