Clinical trial NCT05979441: A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Active Idiopathic Inflammatory Myopathy
Official title: A Phase 3, Single-Arm, Multicenter, Open-label Extension of Study ARGX-113-2007 to Investigate the Long-term Safety, Tolerability, and Efficacy of Efgartigimod PH20 SC in Participants Aged 18 Years and Older With Active Idiopathic Inflammatory Myopathy
Study type: INTERVENTIONAL
Overall status: ENROLLING_BY_INVITATION
Phase: PHASE3
Conditions: Myositis
Active Idiopathic Inflammatory Myopathy
Dermatomyositis
Polymyositis
Immune-Mediated Necrotizing Myopathy
Antisynthetase Syndrome
Lead sponsor: argenx
Sponsor class: INDUSTRY
First posted: 2023-08-07
Enrollment: 240
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to measure the long-term safety and tolerability of efgartigimod PH20 SC in adult participants with IIM who previously participated in ARGX-113-2007. Secondary objectives include efficacy measures of efgartigimod PH20 SC in participants with IIM.


Eligibility criteria: Inclusion Criteria:

* Has completed trial ARGX-113-2007
* Being capable of providing signed informed consent and complying with protocol requirements
* Agrees to use contraceptive measures consistent with local regulations and women of childbearing potential must have a negative urine pregnancy test at baseline before receiving the investigational medicinal product

Exclusion Criteria:

* Intention to have major surgery during the study period; or any other medical condition that has arisen since enrollment in study ARGX-113-2007, that in the investigator's opinion, would confound the results of the study or put the participant at undue risk
* Known hypersensitivity reaction to investigational medicinal product or 1 of its excipients Development of any malignancy, either new or recurrent, other than basal cell carcinoma of the skin, regardless of relatedness
* Permanent discontinuation of IMP in ARGX-113-2007, or met the permanent discontinuation criteria at the rollover visit
* Diagnosis with a deselected subtype of myositis based on the analysis of the phase 2 stage data in ARGX-113-2007, unless the investigator determines that the participant is benefiting from IMP as defined by a score of "much better" or "moderately better" on the 'Clinical Global Impression of Change' and 'Patient Global
* Impression of Change' assessments for at least 12 weeks, and that enrolling in the study is in the participant's best interest

Primary outcomes:
measure: Incidence treatment-emergent adverse events and adverse event of special interest
timeFrame: Up to 60 weeks
Secondary outcomes:
measure: Composite Glucocorticoid Toxicity Index (C-GTI) comprising the Aggregate Improvement Score (AIS) and the Cumulative Worsening Score (CWS)
timeFrame: Up to 52 weeks
measure: Prednisone dose reduction (average monthly dose)
timeFrame: Up to 52 weeks
measure: Proportion of participants who discontinue corticosteroids
timeFrame: Up to 52 weeks
measure: Total improvement score (TIS)
timeFrame: Up to 52 weeks
measure: Proportion of TIS responders (minimal, moderate, major)
timeFrame: up to 52 weeks
measure: Individual core set measures (CSMs) of the TIS
timeFrame: up to 52 weeks
measure: Percentage of participants with clinically inactive disease
timeFrame: up to 52 weeks
measure: Percentage of participants with remission, defined as a clinically inactive disease for at least 24 weeks
timeFrame: up to 52 weeks
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Neuromuscular Clinical and Research Center - Neurology
state: Arizona
city: Beverly Hills
country: United States
facility: Attune Health Research, Inc
state: California
city: Orange
country: United States
facility: UCI Health - ALS and Neuromuscular Center - Neurology
state: California
city: San Francisco
country: United States
facility: UCSF Health - ALS and Neurodegenerative Disease Center - Dermatology
state: California
city: Gainesville
country: United States
facility: UF Health - Rheumatology Medical Specialties Medical Plaza
state: Florida
city: Jacksonville
country: United States
facility: Mayo Clinic - Florida - Neurology
state: Florida
city: Atlanta
country: United States
facility: Emory Healthcare - Emory Clinic - Rheumatology
state: Georgia
city: Chicago
country: United States
facility: Northwestern Medicine - Northwestern Memorial Hospital
state: Illinois
city: Great Neck
country: United States
facility: Northwell Health Physician Partners Rheumatology
state: New York
city: Portland
country: United States
facility: Oregon Health and Science University
state: Oregon
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center
state: Pennsylvania
city: Austin
country: United States
facility: Austin Neuromuscular Center (National Neuromuscular Research Institute, PLLC)
state: Texas
city: Houston
country: United States
facility: Nerve And Muscle Center Of Texas
state: Texas
city: San Miguel De Tucumán
country: Argentina
facility: Centro Medico Privado de Reumatologia
city: San Miguel
country: Argentina
facility: Centro Dermatologico Schejtman
city: Innsbruck
country: Austria
facility: Tirol Kliniken - A.o. Landeskrankenhaus Innsbruck - Neurology
city: Gent
country: Belgium
facility: AZ Sint-Lucas - Campus Sint-Lucas - Neurology
city: Leuven
country: Belgium
facility: Universitair Ziekenhuis Leuven Gasthuisberg Campus
city: Plovdiv
country: Bulgaria
facility: Medical Centre Artmed
city: Nicosia
country: Cyprus
facility: Cyprus Institute of Neurology and Genetics
city: Praha
country: Czechia
facility: Revmatologicky Ustav
city: Copenhagen
country: Denmark
facility: Copenhagen University Hospital-Rigshospitalet University Hospital
city: Paris
country: France
facility: Reference Center Neuro-Muscular Diseases - CHU Paris Group Hospitalier La Pitie Salpetriere-Charles Foix
city: Tbilisi
country: Georgia
facility: Aversi Clinic
city: Tbilisi
country: Georgia
facility: LLC MediClub Georgia
city: Tbilisi
country: Georgia
facility: New Hospitals
city: Tbilisi
country: Georgia
facility: The First Medical Center
city: Berlin
country: Germany
facility: Charite Universitaetsmedizin Berlin - Campus Charite Mitte (CCM) - Rheumatology
city: Tuebingen
country: Germany
facility: Universitaetsklinikum Tuebingen (UKT)
city: Athens
country: Greece
facility: National and Kapodistrian University of Athens - Eginition Hospital
city: Athens
country: Greece
facility: ATTIKON University General Hospital - Neurology
city: Milan
country: Italy
facility: Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - Ospedale San Raffaele Hospital
city: Pisa
country: Italy
facility: Azienda Ospedaliero Universitaria Pisana - Ospedale Santa Chiara - Rheumatology
city: Roma
country: Italy
facility: Fondazione Policlinico Universitario Agostino Gemelli - Neurology
city: Seoul
country: Korea, Republic of
facility: Seoul National University Hospital
city: Vilnius
country: Lithuania
facility: Santaros University Clinic
city: Amsterdam
country: Netherlands
facility: Amsterdam UMC Research BV
city: Arequipa
country: Peru
facility: Clinica San Juan de Dios - sede Arequipa - Rheumatology
city: Szczecin
country: Poland
facility: Centrum Wsparcia Badan Klinicznych Pomorskiego Uniwersytetu Medycznego w Szczecinie (CWBK PUM) - Rheumatology
city: Łódź
country: Poland
facility: USK-WAM Centralny Szpital Weteranow - Szpital Kniniczny ul. Zeromskiego 113 - Rheumatology
city: Porto
country: Portugal
facility: Centro Hospitalar do Porto - Hospital Santo Antonio
city: Belgrade
country: Serbia
facility: Institute of Rheumatology
city: Madrid
country: Spain
facility: Hospital Universitario 12 de Octubre
city: Valencia
country: Spain
facility: Hospital Universitari i Politecnic La Fe de Valencia - Neurology
city: Solna
country: Sweden
facility: Karolinska Universitestssjukhus Solna
city: Taichung City
country: Taiwan
facility: Chung Shan Medical University Hospital
city: Bangkok
country: Thailand
facility: King Chulalongkorn Memorial Hospital - Neurology
city: Bangkok
country: Thailand
facility: Phramongkutklao Hospital
city: Khon Kaen
country: Thailand
facility: Faculty Of Medicine, Khon Kaen University
city: London
country: United Kingdom
facility: The Royal Free Hospital
city: Salford
country: United Kingdom
facility: Northern Care Allicance NHS Foundation Trust - Salford Royal - Rheumatology
Study officials:
References: