[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100549646":3},{"organization":4,"armGroups":7,"interventions":24,"overallOfficials":30,"centralContacts":36,"locations":42,"responsibleParty":161,"collaborators":30,"id":163,"slug":164,"hasResults":165,"nctId":166,"briefTitle":167,"officialTitle":168,"acronym":30,"eligibilityCriteria":169,"healthyVolunteers":165,"sex":170,"minAge":171,"maxAge":30,"enrollmentInfo":172,"targetDuration":30,"studyType":175,"phases":176,"briefSummary":178,"conditions":179,"keywords":30,"overallStatus":59,"whyStopped":30,"lastUpdateSubmitDate":182,"lastUpdatePostDateStruct":183,"startDateStruct":186,"completionDateStruct":188,"leadSponsor":190,"locationsCount":191},{"fullName":5,"class":6},"argenx","INDUSTRY",[8,14,20],{"label":9,"type":10,"description":11,"interventionNames":12},"Double-blinded treatment period - ARGX-119 IV","EXPERIMENTAL","Participants receive ARGX-119 during the double-blinded treatment period",[13],"Biological: ARGX-119",{"label":15,"type":16,"description":17,"interventionNames":18},"Double-blinded treatment period - Placebo IV","PLACEBO_COMPARATOR","Participants receive placebo during the double-blinded treatment period",[19],"Other: Placebo",{"label":21,"type":10,"description":22,"interventionNames":23},"Active-treatment period - ARGX-119 IV","Participants receive ARGX-119 during the active-treatment period",[13],[25,31],{"type":26,"name":27,"description":28,"armGroupLabels":29,"otherNames":30},"BIOLOGICAL","ARGX-119","Intravenous infusion of ARGX-119",[21,9],null,{"type":32,"name":33,"description":34,"armGroupLabels":35,"otherNames":30},"OTHER","Placebo","Intravenous infusion of placebo",[15],[37],{"name":38,"role":39,"phone":40,"phoneExt":30,"email":41},"Sabine Coppieters, MD","CONTACT","857-350-4834","clinicaltrials@argenx.com",[43,57,71,85,97,111,127,138,152],{"facility":44,"status":45,"city":46,"state":47,"zip":48,"country":49,"countryCode":50,"cosmosGeoPoint":51,"geoPoint":56,"contacts":30},"UC Davis Medical Center","ACTIVE_NOT_RECRUITING","Sacramento","California","95817","United States","US",{"type":52,"coordinates":53},"Point",[54,55],-121.4944,38.58157,{"lat":55,"lon":54},{"facility":58,"status":59,"city":60,"state":61,"zip":62,"country":49,"countryCode":50,"cosmosGeoPoint":63,"geoPoint":67,"contacts":68},"Ann and Robert H Lurie Childrens Hospital of Chicago","RECRUITING","Chicago","Illinois","60611",{"type":52,"coordinates":64},[65,66],-87.65005,41.85003,{"lat":66,"lon":65},[69],{"name":70,"role":39,"phone":40,"phoneExt":30,"email":41},"Nancy Kuntz, MD",{"facility":72,"status":59,"city":73,"state":30,"zip":74,"country":75,"countryCode":76,"cosmosGeoPoint":77,"geoPoint":81,"contacts":82},"Ottawa Hospital Research Institute - Civic Campus","Ottawa","K1Y 4E9","Canada","CA",{"type":52,"coordinates":78},[79,80],-75.69812,45.41117,{"lat":80,"lon":79},[83],{"name":84,"role":39,"phone":40,"phoneExt":30,"email":41},"Hanns Lochmuller, MD",{"facility":86,"status":87,"city":88,"state":30,"zip":89,"country":90,"countryCode":91,"cosmosGeoPoint":92,"geoPoint":96,"contacts":30},"CHU - Hospital de la Timone","COMPLETED","Marseille","13385","France","FR",{"type":52,"coordinates":93},[94,95],5.38107,43.29695,{"lat":95,"lon":94},{"facility":98,"status":59,"city":99,"state":30,"zip":100,"country":90,"countryCode":91,"cosmosGeoPoint":101,"geoPoint":105,"contacts":106},"Group Hospitalier Pitie-Salpetriere","Paris","75013",{"type":52,"coordinates":102},[103,104],2.3488,48.85341,{"lat":104,"lon":103},[107],{"name":108,"role":39,"phone":109,"phoneExt":30,"email":110},"Rocio-Nur Villar-Quiles, MD","+33142163791","rocionur.villarquiles@aphp.fr",{"facility":112,"status":59,"city":113,"state":30,"zip":114,"country":115,"countryCode":116,"cosmosGeoPoint":117,"geoPoint":121,"contacts":122},"Fondazione IRCCS Istituto Neurologico Carlo Besta","Milan","20133","Italy","IT",{"type":52,"coordinates":118},[119,120],9.18951,45.46427,{"lat":120,"lon":119},[123],{"name":124,"role":39,"phone":125,"phoneExt":30,"email":126},"Lorenzo Maggi, MD","+393491237639","lorenzo.maggi@istituto-besta.it",{"facility":128,"status":45,"city":129,"state":30,"zip":130,"country":131,"countryCode":132,"cosmosGeoPoint":133,"geoPoint":137,"contacts":30},"Universitat de Valencia - Hospital Universitari i Politecnic La Fe de Valencia (Hospital La Fe Bulevar Sur)","Valencia","46026","Spain","ES",{"type":52,"coordinates":134},[135,136],-0.37966,39.47391,{"lat":136,"lon":135},{"facility":139,"status":59,"city":140,"state":30,"zip":141,"country":142,"countryCode":143,"cosmosGeoPoint":144,"geoPoint":148,"contacts":149},"Clinical Trials Centre - South Eastern Health and Social Care Trust - The Ulster Hospital","Belfast","BT16 1RH","United Kingdom","UK",{"type":52,"coordinates":145},[146,147],-5.92541,54.59682,{"lat":147,"lon":146},[150],{"name":151,"role":39,"phone":40,"phoneExt":30,"email":41},"John McConville, MD",{"facility":153,"status":45,"city":154,"state":30,"zip":155,"country":142,"countryCode":143,"cosmosGeoPoint":156,"geoPoint":160,"contacts":30},"John Radcliffe Hospital - Oxford University Hospitals NHS Foundation Trust","Oxford","OX3 9DU",{"type":52,"coordinates":157},[158,159],-1.25596,51.75222,{"lat":159,"lon":158},{"type":162,"investigatorFullName":30,"investigatorTitle":30,"investigatorAffiliation":30,"oldNameTitle":30,"oldOrganization":30},"SPONSOR","100549646","phase-1-a-phase-1b-study-to-investigate-safety-and-tolerability-of-argx-119-in-adult-participants-with-dok7-congenital-myasthenic-syndromes-cms-100549646",false,"NCT06436742","A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)","A Phase 1b, Double-Blinded, Randomized, Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, Immunogenicity, and Efficacy of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes","Inclusion Criteria:\n\n* At least 18 years of age.\n* Has genetically confirmed congenital myasthenic syndromes due to mutation of downstream of kinase 7 (DOK7-CMS).\n* Participants taking oral beta agonists (eg, albuterol, salbutamol, ephedrine) must have been receiving the medication for more than 3 months and agree to remain on a same stable dosing regimen of the same medication until the end of the study.\n\nExclusion Criteria:\n\n* Diagnosis of CMS due to mutation of any gene other than DOK7.\n* Known medical condition that would interfere with an accurate assessment of CMS, confound the results of the study, or put the patient at undue risk, as assessed by the investigator.\n* History of malignancy, cancer, unless considered cured by adequate treatment with no evidence of recurrence for more than 5 years. 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 findings of prostate cancer.\n* Pregnant or lactating state or intention to become pregnant during the study.","ALL","18 Years",{"count":173,"type":174},16,"ESTIMATED","INTERVENTIONAL",[177],"PHASE1","The purpose of this study is to assess the safety and tolerability of ARGX-119 in adult participants with DOK7- Congenital Myasthenic Syndromes. The study will also assess how ARGX-119 is processed by the body (pharmacokinetics), how the immune system reacts to it (immunogenicity), and how it may improve the way patients feel and function.\n\nAfter the screening period, eligible participants will be randomized in a 4:1 ratio to receive intravenous infusions of ARGX-119 or placebo during the double-blinded treatment period. Participants will then enter the follow-up period. After the follow-up period, participants may enrol in the active-treatment period, where they will receive open-label ARGX-119.\n\nThe full duration of the study is approximately 38 months.",[180,181],"Congenital Myasthenic Syndrome","CMS","2026-04-22",{"date":184,"type":185},"2026-04-23","ACTUAL",{"date":187,"type":185},"2024-09-24",{"date":189,"type":174},"2028-01-24",{"name":5,"class":6},9]