En studie for å vurdere den langsiktige sikkerheten og effekten av en subkutan formulering av Efgartigimod hos voksne med aktiv idiopatisk inflammatorisk myopati (ALKIVIA+)
En fase 3, enkeltarm, multisenter, åpen utvidelse av studien ARGX-113-2007 for å undersøke langsiktig sikkerhet, tolerabilitet og effekt av Efgartigimod PH20 SC hos deltakere i alderen 18 år og eldre med aktiv idiopatisk inflammatorisk myopati
The main purpose of this study is to measure the long-term safety and tolerability of efgartigimod PH20 SC in adult participants with Idiopathic Inflammatory Myopathy (IIM) who previously participated in ARGX-113-2007.
The study consists of a treatment period where participants will receive efgartigimod PH20 SC for up to 51 months. The treatment period will be followed by a treatment-free safety follow-up period of 56 days.
Studieoversikt
Status
Forhold
Intervensjon / Behandling
Studietype
Registrering (Antatt)
Fase
- Fase 3
Kontakter og plasseringer
Studiesteder
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La Plata, Argentina, B1902COS
- Framingham Centro Medico
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San Miguel, Argentina, B1663GKT
- Centro Dermatologico Schejtman
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San Miguel de Tucumán, Argentina, 4000
- Centro Medico Privado de Reumatologia
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Ghent, Belgia, 9000
- AZ Sint-Lucas - Campus Sint-Lucas - Neurology
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Leuven, Belgia, 3000
- Universitair Ziekenhuis Leuven Gasthuisberg Campus
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Plovdiv, Bulgaria, 4002
- Multiprofile hospital for active treatment Kaspela EOOD
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Plovdiv, Bulgaria, 4002
- Medical Centre Artmed
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Copenhagen, Danmark, 2100
- Copenhagen University Hospital-Rigshospitalet University Hospital
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Arizona
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Phoenix, Arizona, Forente stater, 85028
- Neuromuscular Clinical and Research Center - Neurology
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California
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Beverly Hills, California, Forente stater, 90039
- Attune Health Research, Inc
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Orange, California, Forente stater, 92868
- UCI Health - ALS and Neuromuscular Center - Neurology
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San Francisco, California, Forente stater, 94143
- UCSF Health - ALS and Neurodegenerative Disease Center - Dermatology
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Connecticut
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New Haven, Connecticut, Forente stater, 06520
- Yale Cancer Center-Yale University School Of Medicine
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Florida
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Gainesville, Florida, Forente stater, 32610
- UF Health - Rheumatology Medical Specialties Medical Plaza
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Jacksonville, Florida, Forente stater, 32224
- Mayo Clinic - Florida - Neurology
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Tampa, Florida, Forente stater, 33616
- University of South Florida (USF) - Morsani Center (USF Health Carol and Frank Morsani Center for Advanced Healthcare)
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Georgia
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Atlanta, Georgia, Forente stater, 30322
- Emory Healthcare - Emory Clinic - Rheumatology
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Illinois
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Chicago, Illinois, Forente stater, 60611
- Northwestern Medicine - Northwestern Memorial Hospital
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Maryland
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Baltimore, Maryland, Forente stater, 21224-2734
- John Hopkins Medicine - Johns Hopkins Myositis Center
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New York
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Great Neck, New York, Forente stater, 11021
- Northwell Health Physician Partners Rheumatology
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North Carolina
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Chapel Hill, North Carolina, Forente stater, 27514
- University of North Carolina (UNC) School of Medicine
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Ohio
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Columbus, Ohio, Forente stater, 43201
- The Ohio State University
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Oregon
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Portland, Oregon, Forente stater, 97239
- Oregon Health and Science University
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Pennsylvania
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Pittsburgh, Pennsylvania, Forente stater, 15261
- University of Pittsburgh Medical Center
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Texas
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Austin, Texas, Forente stater, 78759
- Austin Neuromuscular Center (National Neuromuscular Research Institute, PLLC)
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Houston, Texas, Forente stater, 77030
- Nerve And Muscle Center Of Texas
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Paris, Frankrike, 75013
- Reference Center Neuro-Muscular Diseases - CHU Paris Group Hospitalier La Pitie Salpetriere-Charles Foix
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Tbilisi, Georgia, 0160
- Aversi Clinic
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Tbilisi, Georgia, 0180
- The First Medical Center
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Tbilisi, Georgia, 0160
- LLC MediClub Georgia
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Tbilisi, Georgia, 0162
- New Hospitals
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Athens, Hellas, 115 27
- National and Kapodistrian University of Athens - School of Health Sciences - Faculty of Medicine
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Athens, Hellas, 115 28
- National and Kapodistrian University of Athens - Eginition Hospital
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Athens, Hellas, 12462
- ATTIKON University General Hospital - Neurology
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Dublin, Irland, D04 T6F4
- University College Dublin (UCD) - St. Vincent's University Hospital (SVUH)
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Bari, Italia, 70124
- Azienda Ospedaliero Universitaria Consorziale Policlinico
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Milan, Italia, 20132
- Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - Ospedale San Raffaele Hospital
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Pisa, Italia, 56126
- Azienda Ospedaliero Universitaria Pisana - Ospedale Santa Chiara - Rheumatology
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Roma, Italia, 00189
- Ospedale Sant'Andrea Hospital
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Roma, Italia, 168
- Fondazione Policlinico Universitario Agostino Gemelli - Neurology
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Rozzano, Italia, 20089
- IRCCS Istituto Clinico Humanitas
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Udine, Italia, 33100
- Azienda sanitaria universitaria Friuli Centrale - ASUFC
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Bunkyō City, Japan, 113-8431
- Juntendo University Hospital
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Bunkyō City, Japan, 113-8655
- The University of Tokyo Hospital
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Nagoya, Japan, 457-8510
- Japan Community Healthcare Organization Chukyo Hospital
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Tokyo, Japan, 113-8519
- Institute of Science Tokyo Hospital
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Wakayama, Japan, 641-8510
- Wakayama Medical University Hospital
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Yamaguchi, Japan, 755-8505
- Yamaguchi University Hospital
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Beijing, Kina, 100730
- Peking Union Medical College Hospital
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Beijing, Kina, 100191
- Peking University Third Hospital
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Chengdu, Kina, 610072
- Sichuan Provincial Academy of Medical Sciences and Sichuan Provincial People's Hospital
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Shanghai, Kina, 200040
- Huashan Hospital Affiliated to Fudan University
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Shanghai, Kina, 200001
- Shanghai Jiao Tong University School of Medicine - Renji Hospital - Shanghai Institute of Digestive Disease (SIDD)
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Shenyang, Kina, 110001
- China Medical University (CMU) - First Affiliated Hospital
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Zhengzhou, Kina, 450052
- First Affiliated Hospital (Henan Medical University - First Affiliated Hospital)-Zhengzhou University
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Guangdong
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Guangzhou, Guangdong, Kina, 510515
- Nanfang Hospital, Southern Medical University
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Jingsu
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Nanjing, Jingsu, Kina, 210000
- Nanjing University Medical School - Taikang Xianlin Drum Tower Hospital (Nanjing Drum Tower Hospital)
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Nicosia, Kypros, 2371
- Cyprus Institute of Neurology and Genetics
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Vilnius, Litauen, 08410
- Santaros University Clinic
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Mexico City, Mexico, 06700
- Hospital Angeles Clinica Londres
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Amsterdam, Nederland, 1105 AZ
- Amsterdam UMC Research BV
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Arequipa, Peru, 4000
- Clinica San Juan de Dios - sede Arequipa - Rheumatology
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Lima, Peru, 33
- Investigaciones Clinicas S.A.C.
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Lima, Peru, 27
- Instituto Peruano Del Hueso Y La Articulacion Sac-Privado-Lima,Centro De Investigacion Iphar
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Lodz, Polen, 90-549
- USK-WAM Centralny Szpital Weteranow - Szpital Kniniczny ul. Zeromskiego 113 - Rheumatology
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Szczecin, Polen, 71-252
- Centrum Wsparcia Badan Klinicznych Pomorskiego Uniwersytetu Medycznego w Szczecinie (CWBK PUM) - Rheumatology
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Porto, Portugal, 4099-001
- Centro Hospitalar do Porto - Hospital Santo Antonio
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Belgrade, Serbia, 11000
- Clinical Center of Serbia
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Belgrade, Serbia, 11000
- Institute of Rheumatology
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Barcelona, Spania, 08041
- Hospital de la Santa Creu i Sant Pau
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Madrid, Spania, 28041
- Hospital Universitario 12 de Octubre
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Valencia, Spania, 46026
- Hospital Universitari i Politecnic La Fe de Valencia - Neurology
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Liverpool, Storbritannia, L9 7AL
- Aintree University Hospital - Liverpool University Hospitals NHS Foundation Trust
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London, Storbritannia, NW3 2QG
- The Royal Free Hospital
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Salford, Storbritannia, M6 8HD
- Northern Care Allicance NHS Foundation Trust - Salford Royal - Rheumatology
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Solna, Sverige, 17176
- Karolinska Universitestssjukhus Solna
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Seoul, Sør -Korea, 03080
- Seoul National University Hospital
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Taichung, Taiwan, 402
- Chung Shan Medical University Hospital
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Taichung, Taiwan
- Taichung Veterans General Hospital
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Bangkok, Thailand, 10400
- Phramongkutklao Hospital
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Bangkok, Thailand, 10330
- King Chulalongkorn Memorial Hospital - Neurology
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Bangkok, Thailand, 10700
- Siriraj Hospital, Mahidol University of Internal medicine
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Bangkok, Thailand, 10400
- Ramathibodi hospital
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Khon Kaen, Thailand, 40002
- Faculty Of Medicine, Khon Kaen University
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Prague, Tsjekkia, 12850
- Revmatologicky Ustav
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Berlin, Tyskland, 10117
- Charite Universitaetsmedizin Berlin - Campus Charite Mitte (CCM) - Rheumatology
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Hamburg, Tyskland, 20246
- Universitaetsklinikum Hamburg-Eppendorf
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Herne, Tyskland, 44649
- Rheumazentrum Ruhrgebiet
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Mannheim, Tyskland, 68167
- Universitatsmedizin Mannheim GmbH
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Tübingen, Tyskland, 72076
- Universitaetsklinikum Tuebingen (UKT)
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Ulm, Tyskland, 89081
- Klinikum der Universitaet Ulm
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Innsbruck, Østerrike, 6020
- Tirol Kliniken - A.o. Landeskrankenhaus Innsbruck - Neurology
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Deltakelseskriterier
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
Tar imot friske frivillige
Kjønn som er kvalifisert for studier
Beskrivelse
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 study drug
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 the study drug or 1 of its excipients
- Development of any malignancy, either new or recurrent, other than basal cell carcinoma of the skin, regardless of relatedness
Studieplan
Hvordan er studiet utformet?
Designdetaljer
- Primært formål: Behandling
- Tildeling: N/A
- Intervensjonsmodell: Enkeltgruppeoppdrag
- Masking: Ingen (Open Label)
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
|---|---|
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Eksperimentell: Efgartigimod PH20 SC
Participants receiving efgartigimod PH20 subcutaneously |
Subcutaneous injection of Efgartigimod PH20 given by vials or prefilled syringe (PFS) |
Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
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Incidence of AEs, SAEs and AESIs over time
Tidsramme: Up to 53 months
|
AE : adverse event ; SAE : serious adverse event ; AESI : adverse event of special interest. |
Up to 53 months
|
Sekundære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
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Prednison-dosereduksjon (gjennomsnittlig månedlig dose) over tid
Tidsramme: Opptil 51 måneder
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Opptil 51 måneder
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Prosentvis endring fra utgangspunkt i totale IgG-nivåer i serum over tid
Tidsramme: Opptil 53 måneder
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Opptil 53 måneder
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Varighet av dosering hver 2. uke
Tidsramme: Opptil 51 måneder
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Opptil 51 måneder
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C-GTI comprising the AIS over time
Tidsramme: Up to 51 months
|
The Composite Glucocorticoid Toxicity Index (C-GTI) serves as a primary instrument to capture toxicities likely caused by glucocorticoid exposure and enables monitoring of long-term tolerability of glucocorticoids during prolonged use. The Aggregate Improvement Score (AIS) is an analytical scores generated from the weighted C-GTI. The AIS can be used to assess whether a new therapy is effective in reducing glucocorticoid toxicity over time; higher AIS scores are associated with less toxicity. |
Up to 51 months
|
|
C-GTI comprising the CWS over time
Tidsramme: Up to 51 months
|
The Composite Glucocorticoid Toxicity Index (C-GTI) serves as a primary instrument to capture toxicities likely caused by glucocorticoid exposure and enables monitoring of long-term tolerability of glucocorticoids during prolonged use. The Cumulative Worsening Score (CWS) is an analytical score generated from the weighted C-GTI. The CWS is designed to assess cumulative glucocorticoid toxicity; higher scores indicate increased steroid toxicity. |
Up to 51 months
|
|
C-GTI comprising the GTI-MD over time
Tidsramme: Up to 51 months
|
The Composite Glucocorticoid Toxicity Index (C-GTI) serves as a primary instrument to capture toxicities likely caused by glucocorticoid exposure and enables monitoring of long-term tolerability of glucocorticoids during prolonged use. The Glucocorticoid Toxicity Index-Metabolic Domains (GTI-MD) is an abbreviated GTI version limited to the 4 metabolic domains. A GTI-MD score of 0 indicates a low likelihood of toxicity in the assessed metabolic domains. |
Up to 51 months
|
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TIS over time
Tidsramme: Up to 51 months
|
The total improvement score (TIS) assesses minimal, moderate, and major clinical response, and it is assessed using the ACR/EULAR criteria. The total score varies between 0 and 100, with higher scores indicating greater improvement. |
Up to 51 months
|
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Proportion of TIS responders (minimal, moderate, major) over time
Tidsramme: Up to 51 months
|
The total improvement score (TIS) assesses minimal, moderate, and major clinical response, and it is assessed using the ACR/EULAR criteria. The total score varies between 0 and 100, with higher scores indicating greater improvement |
Up to 51 months
|
|
Individual CSMs of the TIS over time
Tidsramme: Up to 51 months
|
The total improvement score (TIS) assesses minimal, moderate, and major clinical response, and it is assessed using the ACR/EULAR criteria. The ACR/EULAR criteria calculate TIS using the 6 core set measure (CSMs): MDGA, PGA, MMT8, HAQ-DI, muscle enzymes, and extramuscular global assessment (assessed by the MDAAT). |
Up to 51 months
|
|
Percentage of participants with clinically inactive disease
Tidsramme: Up to 51 months
|
Clinically inactive disease is defined as no evidence of disease activity, based on an Physician Global Assessment of Disease Activity (MDGA) and Extramuscular Global Assessment of the Myositis Disease Activity Assessment Tool (MDAAT) of 0 and normal creatine kinase (CK) values for at least 12 weeks (at week 28 and every 24 weeks thereafter). |
Up to 51 months
|
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Percentage of participants with remission during the study
Tidsramme: Up to 51 months
|
Remission is defined as a clinically inactive disease for at least 24 weeks |
Up to 51 months
|
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PGI-S over time
Tidsramme: Up to 51 months
|
The Patient Global Impression of Severity (PGI-S) asks participants to rate the severity of their disease symptoms over the past 7 days on a 4-point Likert scale, where scores range from "no symptoms" to "severe." |
Up to 51 months
|
|
CGI-S over time
Tidsramme: Up to 51 months
|
The Clinical Global Impression of Severity (CGI-S) asks the physician to rate the severity of the participant's disease on a 4-point Likert scale, where scores range from "no activity" to "severe". |
Up to 51 months
|
|
Physical Functioning subscale and Physical Component Summary scores of the SF-36v2 over time
Tidsramme: Up to 51 months
|
The 36-Item Short Form Survey version 2 (SF-36v2) is a 36-item scale constructed to survey health-related quality of life in 8 domains. |
Up to 51 months
|
|
EQ-5D-5L utilities over time
Tidsramme: Up to 51 months
|
The EQ-5D-5L questionnaire is a standardized test recognized by many health authorities as a generic measure of health status for clinical and economic appraisal. The descriptive system comprises 5 dimensions, each having 5 levels ranging from "No problem" to "Extreme problem". |
Up to 51 months
|
|
EQ-5D-5L VAS over time
Tidsramme: Up to 51 months
|
The EQ-5D-5L questionnaire is a standardized test recognized by many health authorities as a generic measure of health status for clinical and economic appraisal. A visual analog scale (VAS) is included in the EQ-5D-5L, where participants will mark their health status from 0 (the worst health you can imagine) to 100 (the best health you can imagine). |
Up to 51 months
|
|
Incidence of ADA against efgartigimod (serum levels) over time
Tidsramme: Up to 53 months
|
ADA : anti-drug antibodies. |
Up to 53 months
|
|
Proportion of participants achieving sustained low disease activity while on IMP, over time
Tidsramme: Up to 51 months
|
Sustained low disease activity is defined as a CGI-S score of "mild" or "no activity" and a PGI-S score of "mild" or "no symptoms" for at least 12 weeks. |
Up to 51 months
|
|
Proportion of participants with low disease activity over time, while on IMP
Tidsramme: Up to 51 months
|
Low disease activity is defined as a CGI-S score of "mild" or "no activity" and a PGI-S score of "mild" or "no symptoms" |
Up to 51 months
|
|
Proportion of participants with low disease activity who continue dosing every 2 weeks at week 52 and every 24 weeks thereafter
Tidsramme: Up to 51 months
|
Low disease activity is defined as a CGI-S score of "mild" or "no activity" and a PGI-S score of "mild" or "no symptoms" |
Up to 51 months
|
|
Proportion of participants who have marked worsening during the period of dosing every 2 weeks
Tidsramme: Up to 51 months
|
Marked worsening is defined as a CGI-S score of "moderate" or "severe" and a PGI-S score of "moderate" or "severe" |
Up to 51 months
|
Samarbeidspartnere og etterforskere
Sponsor
Studierekorddatoer
Studer hoveddatoer
Studiestart (Faktiske)
Primær fullføring (Antatt)
Studiet fullført (Antatt)
Datoer for studieregistrering
Først innsendt
Først innsendt som oppfylte QC-kriteriene
Først lagt ut (Faktiske)
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
Siste oppdatering sendt inn som oppfylte QC-kriteriene
Sist bekreftet
Mer informasjon
Begreper knyttet til denne studien
Ytterligere relevante MeSH-vilkår
Andre studie-ID-numre
- ARGX-113-2011
- 2022-502851-79-00 (Ctis)
Plan for individuelle deltakerdata (IPD)
Planlegger du å dele individuelle deltakerdata (IPD)?
Legemiddel- og utstyrsinformasjon, studiedokumenter
Studerer et amerikansk FDA-regulert medikamentprodukt
Studerer et amerikansk FDA-regulert enhetsprodukt
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