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

Dermatomyositis Clinical Trial

— ALKIVIA  

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05523167
• Other study ID #: ARGX-113-2007
• Status: Recruiting
• Phase: Phase 2/Phase 3
• Start date: October 12, 2022
• Est. completion date: February 1, 2027

Study information

• Verified date: May 2024
• Source: argenx
• Contact: Sabine Coppieters, MD
• Phone: 857-350-4834
• Email: ClinicalTrials[@]argenx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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/.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 240
• Est. completion date: February 1, 2027
• Est. primary completion date: December 1, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Dermatomyositis
• Immune-Mediated Necrotizing Myopathy
• Muscular Diseases
• Myositis
• Polymyositis

Intervention

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

Locations

Country	Name	City	State
Argentina	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
Australia	The Wesley Medical Research	Auchenflower
Australia	Fiona Stanley Hospital	Murdoch
Austria	Medical University Innsbruck	Innsbruck
Belgium	AZ Sint Lucas Gent-Campus Sint Lucas	Gent
Belgium	Universitair Ziekenhuis Leuven Gasthuisberg Campus	Leuven
Belgium	Universitair Ziekenhuis Leuven Gasthuisberg Campus	Leuven
Belgium	Centre Hospitalier Universitaire Sart Tilman	Liège
Bulgaria	Medical Centre Artmed	Plovdiv
Bulgaria	Multiprofile hospital for active treatment Kaspela EOOD	Plovdiv
Canada	Genge Partners	Montréal
Canada	Ottawa Hospital Research Institute-Civic Campus	Ottawa
Cyprus	Cyprus Institute of Neurology and Genetics	Nicosia
Denmark	Copenhagen University Hospital-Rigshospitalet University Hospital	Copenhagen
France	Reference Center Neuro-Muscular Diseases - CHU Paris Group Hospitalier La Pitie Salpetriere-Charles Foix	Paris
France	Centre Hospitalier Universitaire (CHU) Hopitaux de Rouen	Rouen
France	Hopitaux Universitaire de Strasbourg-Centre de References des Maladies Autoimmunes	Strasbourg
Georgia	American Hospital Network	Tbilisi
Georgia	Aversi Clinic	Tbilisi
Georgia	LLC MediClub Georgia	Tbilisi
Georgia	LTD New Hospitals	Tbilisi
Georgia	LTD Tbilisi Heart Center	Tbilisi
Georgia	The First Medical Center	Tbilisi
Germany	Charite - Universitaetsmedizin Berlin - Campus Charite Mitte (CCM)	Berlin
Germany	Universitaetsklinikum Duesseldorf	Düsseldorf
Germany	Universitatsmedizin Gottingen - Georg-August-Universitat	Göttingen
Germany	Rheumazentrum Ruhrgebiet	Herne
Germany	Universitaetsklinikum Tuebingen (UKT)	Tuebingen
Greece	Athens Medical Center	Athens
Greece	Attikon General University Hospital	Athens
Greece	National and Kapodistrian University of Athens - Eginition Hospital	Athens
Greece	National and Kapodistrian University of Athens - School of Health Sciences - Faculty of Medicine	Athens
Greece	University of Ioannina Medical School	Ioánnina
Hungary	Semmelweis Egyetem, Institute of Genomic Medicine and Rare Disorders	Budapest
Hungary	Debreceni Egyetem - University of Debrecen	Debrecen
Ireland	Connolly Hospital Blanchardstown	Dublin
Ireland	St. Vincents University Hospital	Dublin
Ireland	North West Rheumatology Unit - Our Lady's Hospital	Manorhamilton
Italy	Azienda Ospedaliera Universitaria Policlinico Consorziale Di Bari - Rheumatology	Bari
Italy	Azienda Ospedaliero Universitaria Careggi	Firenze
Italy	Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - Ospedale San Raffaele Hospital	Milan
Italy	San Matteo Hospital	Pavia
Italy	A.O. Universitaria Pisana	Pisa
Italy	IRCCS Arcispedale Santa Maria Nuova	Reggio Emilia
Italy	Fondazione Policlinico Universitario Agostino Gemelli IRCCS - Universita Cattolica del Sacro Cuore	Roma
Italy	IRCCS Istituto Clinico Humanitas - Rheumatology	Rozzano
Korea, Republic of	Chungnam National University Hospital (CNUH)	Daejeon
Korea, Republic of	Gachon University Gil Medical Center	Incheon
Korea, Republic of	Hanyang University Seoul Hospital	Seoul
Korea, Republic of	Kyung Hee University Medical Center	Seoul
Korea, Republic of	Seoul National University Hospital	Seoul
Korea, Republic of	Ajou University Hospital	Suwon
Lithuania	Hospital of Lithuanian University of Health Sciences Kauno klinikos	Kaunas
Lithuania	Vilnius University Hospital Santaros Klinikos	Vilnius
Mexico	CITER - Centro de Investigacion y Tratamiento de las Enfermedades Reumaticas S.A. de C.V.	Mexico City
Netherlands	Amsterdam University Medical Center (Amsterdam UMC), Academic Medical Center (AMC)	Amsterdam
Netherlands	Universitair Medisch Centrum Utrecht (UMC Utrecht)	Utrecht
Peru	Hogar Clinica San Juan de Dios	Arequipa
Peru	Instituto Peruano Del Hueso Y La Articulacion Sac-Privado-Lima, Centro De Investigacion Iphar	Lima
Peru	Investigaciones Clinicas S.A.C.	Lima
Poland	Szpital Uniwersytecki nr 2 im. Dr Jana Biziela w Bydgoszczy	Bydgoszcz
Poland	Centrum Wsparcia Badan Klinicznych Pomorski Uniwersytet Medyczny w Szczecinie	Szczecin
Portugal	Centro Hospitalar Lisboa Norte, E.P.E- Hospital Santa Maria	Lisboa
Serbia	Institute of Rheumatology	Belgrad
Serbia	Clinical Center of Serbia	Belgrade
Spain	Hospital General Universitario de Alicante	Alicante
Spain	Hospital Clinic de Barcelona - Sede Villarroel - Rheumatology	Barcelona
Spain	Hospital Universitario 12 de Octubre	Madrid
Spain	Hospital Universitario La Paz	Madrid
Spain	Hospital Universitario Ramon y Cajal	Madrid
Spain	Hospital De Valme	Sevilla
Spain	Hospital Universitario y Politecnico La Fe	Valencia
Spain	Hospital Do Meixoeiro	Vigo
Sweden	Karolinska Universitestssjukhus Solna	Stockholm
Switzerland	Neurocenter of Southern Switzerland	Lugano
Taiwan	China Medical University and Hospital	Taichung
Taiwan	Chung Shan Medical University Hospital	Taichung city
Thailand	Chulalongkorn University - Faculty of Medicine - King Chulalongkorn Memorial Hospital (KCMH)	Bangkok
Thailand	Phramongkutklao Hospital	Bangkok
Thailand	Ramathibodi hospital	Bangkok
Thailand	Siriraj Hospital, Mahidol University of Internal medicine	Bangkok
Thailand	Khon Kaen University	Khon Kaen
Thailand	Thammasat University Hospital	Pathum Thani
Turkey	Ankara City Hospital	Ankara
Turkey	Ankara University Medical Faculty	Ankara
Turkey	Necmettin Erbakan University Meram Medical Faculty	Konya
United Kingdom	Aintree University Hospital - Liverpool University Hospitals NHS Foundation Trust	Liverpool
United Kingdom	Kings College Hospital NHS Foundation Trust	London
United Kingdom	The National Hospital for Neurology and Neurosurgery	London
United Kingdom	The Royal Free Hospital - Royal Free London NHS Foundation Trust	London
United Kingdom	James Cook University Hospital - South Tees Hospitals NHS Foundation Trust	Middlesbrough
United Kingdom	Salford Royal Hospital, Northern Care Alliance NHS Foundation Trust	Salford
United States	Augusta University	Augusta	Georgia
United States	Austin Neuromuscular Center	Austin	Texas
United States	Johns Hopkins Medicine - Johns Hopkins Myositis Center	Baltimore	Maryland
United States	Attune Health Research, Inc	Beverly Hills	California
United States	Harvard Medical School - Brigham and Women's Hospital (BWH) - The Schuster Family Transplantation Research Center (TRC)	Boston	Massachusetts
United States	University of North Carolina (UNC) School of Medicine	Chapel Hill	North Carolina
United States	Northwestern Memorial Hospital	Chicago	Illinois
United States	The Ohio State University	Columbus	Ohio
United States	Denver Arthritis Clinic	Denver	Colorado
United States	Michigan State University - Neurology	East Lansing	Michigan
United States	Northwell Health	Great Neck	New York
United States	McGovern Medical School -The University of Texas Health Science Center at Houston	Houston	Texas
United States	Nerve And Muscle Center Of Texas	Houston	Texas
United States	University Of Kansas Medical Center	Kansas City	Kansas
United States	Vanderbilt University Medical Center (VUMC) - Vanderbilt Rheumatology Clinic	Nashville	Tennessee
United States	Yale Cancer Center-Yale University School Of Medicine	New Haven	Connecticut
United States	Hospital for Special Surgery	New York	New York
United States	Profound Research LLC - Oceanside	Oceanside	California
United States	Mercy Hospital - Mercy Clinic Neurology Oklahoma City - Neurology	Oklahoma City	Oklahoma
United States	University of California, Irvine	Orange	California
United States	Neuromuscular Research Center	Phoenix	Arizona
United States	University of Pittsburg Medical Center	Pittsburgh	Pennsylvania
United States	Oregon Health and Science University	Portland	Oregon
United States	Stanford Medicine Outpatient Center - Stanford Dermatology Clinic-Stanford University School of Medicine	Redwood City	California
United States	Washington University School of Medicine	Saint Louis	Missouri
United States	St. Paul Rheumatology, PA	Saint Paul	Minnesota
United States	California Pacific Medical Center - Sutter Health	San Francisco	California
United States	HonorHealth Neurology - Bob Bove Neuroscience Institute - Neurology	Scottsdale	Arizona
United States	University of Washington Medical Center	Seattle	Washington
United States	University of South Florida (USF) - Morsani Center (USF Health Carol and Frank Morsani Center for Advanced Healthcare)	Tampa	Florida
United States	Georgetown University Hospital	Washington	District of Columbia
United States	Carolina Arthritis Associates	Wilmington	North Carolina

Sponsors (1)

Lead Sponsor	Collaborator
argenx

Countries where clinical trial is conducted

United States,  Argentina,  Australia,  Austria,  Belgium,  Bulgaria,  Canada,  Cyprus,  Denmark,  France,  Georgia,  Germany,  Greece,  Hungary,  Ireland,  Italy,  Korea, Republic of,  Lithuania,  Mexico,  Netherlands,  Peru,  Poland,  Portugal,  Serbia,  Spain,  Sweden,  Switzerland,  Taiwan,  Thailand,  Turkey,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Total improvement score (TIS); measured on a [0,100] scale. Higher scores represent improvement; zero indicates no improvement or worsening (from baseline).		phase 2: 24 weeks; phase 3: 52 weeks
Secondary	Time to reach TIS = 20 (first "minimal clinical improvement")		phase 2: up to 24 weeks; phase 3: up to 52 weeks
Secondary	Percentage of participants with TIS = 20		phase 2: 24 weeks; phase 3: 52 weeks
Secondary	Time to reach TIS = 40 (first "moderate clinical improvement")		phase 2: up to 24 weeks; phase 3: up to 52 weeks
Secondary	Percentage of participants with TIS = 40		phase 2: 24 weeks; phase 3: 52 weeks
Secondary	Change in manual muscle testing-8 (MMT8) score		phase 2: 24 weeks; phase 3: 52 weeks
Secondary	Change in Patient Global Assessment of Disease Activity (PGA)		phase 2: 24 weeks; phase 3: 52 weeks
Secondary	Change in Physician Global Assessment of Disease Activity (MDGA)		phase 2: 24 weeks; phase 3: 52 weeks
Secondary	Proportion of participants achieving target dose of = 5 mg (prednisone equivalent)		phase 2: 24 weeks; phase 3: 52 weeks

External Links

•   Study website