A Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants with Active Idiopathic Inflammatory Myopathy.
a study on Myopathy Myositis Dermatomyositis Polymyositis Antisynthetase Syndrome
Summary
- Eligibility
- for people ages 18 years and up (full criteria)
- Location
- at UC Irvine UCLA UCSF
- Dates
- study startedcompletion around
Description
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/.
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
Keywords
Active Idiopathic Inflammatory Myopathy, Myositis, Dermatomyositis, Polymyositis, Immune-Mediated Necrotizing Myopathy, Antisynthetase Syndrome, Muscular Diseases, EFG PH20 SC, PBO, PBO PH20 SC
Eligibility
You can join if…
Open to people ages 18 years and up
- 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.
You CAN'T join if...
- 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.
Locations
- University Of California - Los Angeles
accepting new patients
Los Angeles California 90095-1670 United States - University of California, Irvine
accepting new patients
Orange California 92868 United States - Amyotrophic Lateral Sclerosis (ALS) Treatment Center,University of California San Francisco (UCSF)
accepting new patients
San Francisco California 94143-2202 United States - California Pacific Medical Center - Sutter Health
accepting new patients
San Francisco California 94109 United States - Attune Health Research, Inc
accepting new patients
Beverly Hills California 90039 United States - University of Southern California Norris Comprehensive Cancer Center
accepting new patients
Los Angeles California 90033 United States - Profound Research LLC - Oceanside
accepting new patients
Oceanside California 92056 United States - Stanford Medicine Outpatient Center - Stanford Dermatology Clinic-Stanford University School of Medicine
accepting new patients
Redwood City California 94063-3132 United States - Stanford Neuroscience Health Center
accepting new patients
Palo Alto California 94304 United States
Details
- Status
- accepting new patients
- Start Date
- Completion Date
- (estimated)
- Sponsor
- argenx
- Links
- Study website
- ID
- NCT05523167
- Phase
- Phase 2/3 research study
- Study Type
- Interventional
- Participants
- Expecting 240 study participants
- Last Updated