Resumen

El propósito de este estudio es medir la respuesta al tratamiento con efgartigimod PH20 SC en comparación con placebo en participantes con miopatía inflamatoria idiopática (MII). Se incluirán en el estudio los subtipos de MII: dermatomiositis (DM), miopatía necrotizante inmunomediada (MNIM) o ciertos otros subtipos de polimiositis (PM; incluyendo el síndrome antisintetasa [SAS]). La respuesta al tratamiento se medirá mediante la puntuación de mejoría total (PMT).

Elegibilidad

Edad mínima: 18 YearsSexo: ALL

Criterios de inclusión

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).

Criterios de exclusión

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.

Intervenciones

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

Ubicaciones

Consultora Integral de Salud

Córdoba, Argentina

Framingham Centro Medico

La Plata, Argentina

Dim Clinica Privada

Ramos Mejía, Argentina

Centro Dermatologico Schejtman

San Miguel, Argentina

Centro de Investigaciones Medicas Tucuman

San Miguel de Tucumán, Argentina

Patrocinadores

Principalargenx