En undersøgelse for at vurdere den langsigtede sikkerhed og effektivitet af en subkutan formulering af Efgartigimod hos voksne med aktiv idiopatisk inflammatorisk myopati (ALKIVIA+)
En fase 3, single-arm, multicenter, åben udvidelse af undersøgelse ARGX-113-2007 for at undersøge den langsigtede sikkerhed, tolerabilitet og effektivitet af Efgartigimod PH20 SC hos deltagere i alderen 18 år og ældre 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.
Studieoversigt
Status
Status
Betingelser
Betingelser
Intervention / Behandling
Intervention / Behandling
Undersøgelsestype
Undersøgelsestype
Tilmelding (Anslået)
Tilmelding
Fase
Fase
- Fase 3
Kontakter og lokationer
Studiesteder
-
-
-
La Plata, Argentina, B1902COS
- Framingham Centro Medico
-
San Miguel, Argentina, B1663GKT
- Centro Dermatologico Schejtman
-
San Miguel de Tucumán, Argentina, 4000
- Centro Medico Privado de Reumatologia
-
-
-
-
-
Ghent, Belgien, 9000
- AZ Sint-Lucas - Campus Sint-Lucas - Neurology
-
Leuven, Belgien, 3000
- Universitair Ziekenhuis Leuven Gasthuisberg Campus
-
-
-
-
-
Plovdiv, Bulgarien, 4002
- Multiprofile hospital for active treatment Kaspela EOOD
-
Plovdiv, Bulgarien, 4002
- Medical Centre Artmed
-
-
-
-
-
Nicosia, Cypern, 2371
- Cyprus Institute of Neurology and Genetics
-
-
-
-
-
Copenhagen, Danmark, 2100
- Copenhagen University Hospital-Rigshospitalet University Hospital
-
-
-
-
-
Liverpool, Det Forenede Kongerige, L9 7AL
- Aintree University Hospital - Liverpool University Hospitals NHS Foundation Trust
-
London, Det Forenede Kongerige, NW3 2QG
- The Royal Free Hospital
-
Salford, Det Forenede Kongerige, M6 8HD
- Northern Care Allicance NHS Foundation Trust - Salford Royal - Rheumatology
-
-
-
-
Arizona
-
Phoenix, Arizona, Forenede Stater, 85028
- Neuromuscular Clinical and Research Center - Neurology
-
-
California
-
Beverly Hills, California, Forenede Stater, 90039
- Attune Health Research, Inc
-
Orange, California, Forenede Stater, 92868
- UCI Health - ALS and Neuromuscular Center - Neurology
-
San Francisco, California, Forenede Stater, 94143
- UCSF Health - ALS and Neurodegenerative Disease Center - Dermatology
-
-
Connecticut
-
New Haven, Connecticut, Forenede Stater, 06520
- Yale Cancer Center-Yale University School Of Medicine
-
-
Florida
-
Gainesville, Florida, Forenede Stater, 32610
- UF Health - Rheumatology Medical Specialties Medical Plaza
-
Jacksonville, Florida, Forenede Stater, 32224
- Mayo Clinic - Florida - Neurology
-
Tampa, Florida, Forenede Stater, 33616
- University of South Florida (USF) - Morsani Center (USF Health Carol and Frank Morsani Center for Advanced Healthcare)
-
-
Georgia
-
Atlanta, Georgia, Forenede Stater, 30322
- Emory Healthcare - Emory Clinic - Rheumatology
-
-
Illinois
-
Chicago, Illinois, Forenede Stater, 60611
- Northwestern Medicine - Northwestern Memorial Hospital
-
-
Maryland
-
Baltimore, Maryland, Forenede Stater, 21224-2734
- John Hopkins Medicine - Johns Hopkins Myositis Center
-
-
New York
-
Great Neck, New York, Forenede Stater, 11021
- Northwell Health Physician Partners Rheumatology
-
-
North Carolina
-
Chapel Hill, North Carolina, Forenede Stater, 27514
- University of North Carolina (UNC) School of Medicine
-
-
Ohio
-
Columbus, Ohio, Forenede Stater, 43201
- The Ohio State University
-
-
Oregon
-
Portland, Oregon, Forenede Stater, 97239
- Oregon Health and Science University
-
-
Pennsylvania
-
Pittsburgh, Pennsylvania, Forenede Stater, 15261
- University of Pittsburgh Medical Center
-
-
Texas
-
Austin, Texas, Forenede Stater, 78759
- Austin Neuromuscular Center (National Neuromuscular Research Institute, PLLC)
-
Houston, Texas, Forenede Stater, 77030
- Nerve And Muscle Center Of Texas
-
-
-
-
-
Paris, Frankrig, 75013
- Reference Center Neuro-Muscular Diseases - CHU Paris Group Hospitalier La Pitie Salpetriere-Charles Foix
-
-
-
-
-
Tbilisi, Georgien, 0160
- Aversi Clinic
-
Tbilisi, Georgien, 0180
- The First Medical Center
-
Tbilisi, Georgien, 0160
- LLC MediClub Georgia
-
Tbilisi, Georgien, 0162
- New Hospitals
-
-
-
-
-
Athens, Grækenland, 115 27
- National and Kapodistrian University of Athens - School of Health Sciences - Faculty of Medicine
-
Athens, Grækenland, 115 28
- National and Kapodistrian University of Athens - Eginition Hospital
-
Athens, Grækenland, 12462
- ATTIKON University General Hospital - Neurology
-
-
-
-
-
Amsterdam, Holland, 1105 AZ
- Amsterdam UMC Research BV
-
-
-
-
-
Dublin, Irland, D04 T6F4
- University College Dublin (UCD) - St. Vincent's University Hospital (SVUH)
-
-
-
-
-
Bari, Italien, 70124
- Azienda Ospedaliero Universitaria Consorziale Policlinico
-
Milan, Italien, 20132
- Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - Ospedale San Raffaele Hospital
-
Pisa, Italien, 56126
- Azienda Ospedaliero Universitaria Pisana - Ospedale Santa Chiara - Rheumatology
-
Roma, Italien, 00189
- Ospedale Sant'Andrea Hospital
-
Roma, Italien, 168
- Fondazione Policlinico Universitario Agostino Gemelli - Neurology
-
Rozzano, Italien, 20089
- IRCCS Istituto Clinico Humanitas
-
Udine, Italien, 33100
- Azienda sanitaria universitaria Friuli Centrale - ASUFC
-
-
-
-
-
Bunkyō City, Japan, 113-8431
- Juntendo University Hospital
-
Bunkyō City, Japan, 113-8655
- The University of Tokyo Hospital
-
Nagoya, Japan, 457-8510
- Japan Community Healthcare Organization Chukyo Hospital
-
Tokyo, Japan, 113-8519
- Institute of Science Tokyo Hospital
-
Wakayama, Japan, 641-8510
- Wakayama Medical University Hospital
-
Yamaguchi, Japan, 755-8505
- Yamaguchi University Hospital
-
-
-
-
-
Beijing, Kina, 100730
- Peking Union Medical College Hospital
-
Beijing, Kina, 100191
- Peking University Third Hospital
-
Chengdu, Kina, 610072
- Sichuan Provincial Academy of Medical Sciences and Sichuan Provincial People's Hospital
-
Shanghai, Kina, 200040
- Huashan Hospital Affiliated to Fudan University
-
Shanghai, Kina, 200001
- Shanghai Jiao Tong University School of Medicine - Renji Hospital - Shanghai Institute of Digestive Disease (SIDD)
-
Shenyang, Kina, 110001
- China Medical University (CMU) - First Affiliated Hospital
-
Zhengzhou, Kina, 450052
- First Affiliated Hospital (Henan Medical University - First Affiliated Hospital)-Zhengzhou University
-
-
Guangdong
-
Guangzhou, Guangdong, Kina, 510515
- Nanfang Hospital, Southern Medical University
-
-
Jingsu
-
Nanjing, Jingsu, Kina, 210000
- Nanjing University Medical School - Taikang Xianlin Drum Tower Hospital (Nanjing Drum Tower Hospital)
-
-
-
-
-
Vilnius, Litauen, 08410
- Santaros University Clinic
-
-
-
-
-
Mexico City, Mexico, 06700
- Hospital Angeles Clinica Londres
-
-
-
-
-
Arequipa, Peru, 4000
- Clinica San Juan de Dios - sede Arequipa - Rheumatology
-
Lima, Peru, 33
- Investigaciones Clinicas S.A.C.
-
Lima, Peru, 27
- Instituto Peruano Del Hueso Y La Articulacion Sac-Privado-Lima,Centro De Investigacion Iphar
-
-
-
-
-
Lodz, Polen, 90-549
- USK-WAM Centralny Szpital Weteranow - Szpital Kniniczny ul. Zeromskiego 113 - Rheumatology
-
Szczecin, Polen, 71-252
- Centrum Wsparcia Badan Klinicznych Pomorskiego Uniwersytetu Medycznego w Szczecinie (CWBK PUM) - Rheumatology
-
-
-
-
-
Porto, Portugal, 4099-001
- Centro Hospitalar do Porto - Hospital Santo Antonio
-
-
-
-
-
Belgrade, Serbien, 11000
- Clinical Center of Serbia
-
Belgrade, Serbien, 11000
- Institute of Rheumatology
-
-
-
-
-
Barcelona, Spanien, 08041
- Hospital de la Santa Creu i Sant Pau
-
Madrid, Spanien, 28041
- Hospital Universitario 12 de Octubre
-
Valencia, Spanien, 46026
- Hospital Universitari i Politecnic La Fe de Valencia - Neurology
-
-
-
-
-
Solna, Sverige, 17176
- Karolinska Universitestssjukhus Solna
-
-
-
-
-
Seoul, Sydkorea, 03080
- Seoul National University Hospital
-
-
-
-
-
Taichung, Taiwan, 402
- Chung Shan Medical University Hospital
-
Taichung, Taiwan
- Taichung Veterans General Hospital
-
-
-
-
-
Bangkok, Thailand, 10400
- Phramongkutklao Hospital
-
Bangkok, Thailand, 10330
- King Chulalongkorn Memorial Hospital - Neurology
-
Bangkok, Thailand, 10700
- Siriraj Hospital, Mahidol University of Internal medicine
-
Bangkok, Thailand, 10400
- Ramathibodi hospital
-
Khon Kaen, Thailand, 40002
- Faculty Of Medicine, Khon Kaen University
-
-
-
-
-
Prague, Tjekkiet, 12850
- Revmatologicky Ustav
-
-
-
-
-
Berlin, Tyskland, 10117
- Charite Universitaetsmedizin Berlin - Campus Charite Mitte (CCM) - Rheumatology
-
Hamburg, Tyskland, 20246
- Universitaetsklinikum Hamburg-Eppendorf
-
Herne, Tyskland, 44649
- Rheumazentrum Ruhrgebiet
-
Mannheim, Tyskland, 68167
- Universitatsmedizin Mannheim GmbH
-
Tübingen, Tyskland, 72076
- Universitaetsklinikum Tuebingen (UKT)
-
Ulm, Tyskland, 89081
- Klinikum der Universitaet Ulm
-
-
-
-
-
Innsbruck, Østrig, 6020
- Tirol Kliniken - A.o. Landeskrankenhaus Innsbruck - Neurology
-
-
Deltagelseskriterier
Berettigelseskriterier
Berettigelseskriterier
Aldre berettiget til at studere
Tager imod sunde frivillige
Køn, der er berettiget til at studere
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 undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Behandling
- Tildeling: N/A
- Interventionel model: Enkelt gruppeopgave
- Maskning: Ingen (Åben etiket)
Antal våben
Våben og indgreb
Deltagergruppe / ArmDeltagergruppe / Arm |
Intervention / BehandlingIntervention / Behandling |
|---|---|
|
Eksperimentel: Efgartigimod PH20 SC
Participants receiving efgartigimod PH20 subcutaneously |
Subcutaneous injection of Efgartigimod PH20 given by vials or prefilled syringe (PFS) |
Hvad måler undersøgelsen?
Primære resultatmål
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
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
Sekundære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Prednison dosisreduktion (gennemsnitlig månedlig dosis) over tid
Tidsramme: Op til 51 måneder
|
Op til 51 måneder
|
|
|
Procentvis ændring fra baseline i totale IgG-niveauer i serum over tid
Tidsramme: Op til 53 måneder
|
Op til 53 måneder
|
|
|
Behandlingsvarighed hver 2. uge
Tidsramme: Op til 51 måneder
|
Op til 51 måneder
|
|
|
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
|
|
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
|
|
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
|
|
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
|
|
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
|
Samarbejdspartnere og efterforskere
Sponsor
Sponsor
Datoer for undersøgelser
Studer store datoer
Studiestart (Faktiske)
Studiestart
Primær færdiggørelse (Anslået)
Primær færdiggørelse
Studieafslutning (Anslået)
Studieafslutning
Datoer for studieregistrering
Først indsendt
Først indsendt
Først indsendt, der opfyldte QC-kriterier
Først indsendt, der opfyldte QC-kriterier
Først opslået (Faktiske)
Først opslået
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
Sidste opdatering sendt
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
Sidst verificeret
Sidst verificeret
Mere information
Begreber relateret til denne undersøgelse
Yderligere relevante MeSH-vilkår
Andre undersøgelses-id-numre
Andre undersøgelses-id-numre
- ARGX-113-2011
- 2022-502851-79-00 (Ctis)
Plan for individuelle deltagerdata (IPD)
Planlægger du at dele individuelle deltagerdata (IPD)?
Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter
Studerer et amerikansk FDA-reguleret lægemiddelprodukt
Studerer et amerikansk FDA-reguleret enhedsprodukt
Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .