[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100650264":3},{"organization":4,"armGroups":7,"interventions":24,"overallOfficials":30,"centralContacts":36,"locations":30,"responsibleParty":42,"collaborators":30,"id":44,"slug":45,"hasResults":46,"nctId":47,"briefTitle":48,"officialTitle":49,"acronym":50,"eligibilityCriteria":51,"healthyVolunteers":46,"sex":52,"minAge":53,"maxAge":30,"enrollmentInfo":54,"targetDuration":30,"studyType":57,"phases":58,"briefSummary":60,"conditions":61,"keywords":30,"overallStatus":64,"whyStopped":30,"lastUpdateSubmitDate":65,"lastUpdatePostDateStruct":66,"startDateStruct":69,"completionDateStruct":71,"leadSponsor":73,"locationsCount":30},{"fullName":5,"class":6},"argenx","INDUSTRY",[8,14,20],{"label":9,"type":10,"description":11,"interventionNames":12},"Double-blinded treatment period (DBTP) - Adimanebart IV","EXPERIMENTAL","Participants randomized to receive Adimanebart IV",[13],"Biological: Adimanebart IV",{"label":15,"type":16,"description":17,"interventionNames":18},"Double-blinded treatment period (DBTP) - Placebo IV","PLACEBO_COMPARATOR","Participants randomized to receive Placebo IV",[19],"Other: Placebo IV",{"label":21,"type":10,"description":22,"interventionNames":23},"Open-label extension (OLE) - Adimanebart IV","Participants receive Adimanebart IV",[13],[25,31],{"type":26,"name":27,"description":28,"armGroupLabels":29,"otherNames":30},"BIOLOGICAL","Adimanebart IV","Intravenous infusion of Adimanebart",[9,21],null,{"type":32,"name":33,"description":34,"armGroupLabels":35,"otherNames":30},"OTHER","Placebo IV","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",{"type":43,"investigatorFullName":30,"investigatorTitle":30,"investigatorAffiliation":30,"oldNameTitle":30,"oldOrganization":30},"SPONSOR","100650264","phase-3-a-study-to-assess-efficacy-and-safety-of-adimanebart-in-adult-and-pediatric-participants-with-dok7-musk--agrn--or-lrp4--congenital-myasthenic-syndromes-cms-100650264",false,"NCT07746089","A Study to Assess Efficacy and Safety of Adimanebart in Adult and Pediatric Participants With DOK7-,MUSK-, AGRN-, or LRP4- Congenital Myasthenic Syndromes (CMS)","Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Intravenous Adimanebart in Adult and Pediatric Participants With DOK7-,MUSK-, AGRN-, or LRP4-CMS","CoMetS","Inclusion Criteria:\n\nDBTP:\n\n* At least 12 years of age.\n* Has a diagnosis of DOK7-, MUSK-, AGRN-, or LRP4-CMS with documented mutations.\n* Participants taking oral beta agonists (eg, albuterol, salbutamol, ephedrine) or other CMS medication must have been receiving the medication for at least 6 months and agree to remain on a same stable dosing regimen of the same medication unless directed to change their CMS medication(s) by their treating physician.\n\nOLE:\n\n* Completed part of the active-treatment period of ARGX-119-2302.\n\nExclusion Criteria:\n\nDBTP:\n\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\nOLE:\n\n* Investigational study drug discontinuation in ARGX-119-2302.","ALL","12 Years",{"count":55,"type":56},105,"ESTIMATED","INTERVENTIONAL",[59],"PHASE3","The purpose of this study is to assess efficacy and safety of adimanebart in participants at least 12 years of age with DOK7-, MUSK-, AGRN-, or LRP4- Congenital Myasthenic Syndromes (CMS). The study aims to determine whether adimanebart is safe and can help people with CMS feel better and perform daily activities more easily.\n\nThe study includes a double-blinded treatment period (DBTP) and an Open- label extension period (OLE). In the DBTP, all participants will be randomized in a 1:1 ratio to adimanebart or placebo. Participants who complete the DBTP will continue to the OLE.\n\nAdditionally, participants who complete part of the active-treatment period of ARGX-119-2302 study are eligible to enroll in the OLE of this study. In the OLE, all participants will receive open-label adimanebart. After final IMP dose, the participants will enter a follow-up period and their health will be monitored.\n\nThe total duration of the study is up to approximately 152 weeks (2 years and 11 months).\n\nMore information can be found here: clinicaltrials.argenx.com\u002FComets",[62,63],"Congenital Myasthenic Syndrome","CMS","NOT_YET_RECRUITING","2026-07-30",{"date":67,"type":68},"2026-08-04","ACTUAL",{"date":70,"type":56},"2026-09",{"date":72,"type":56},"2030-10",{"name":5,"class":6}]