[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"cms\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:cms":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,1,0,[8],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":30,"lastUpdatePostDateStruct":31,"startDateStruct":34,"completionDateStruct":36,"leadSponsor":38,"locationsCount":4},"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":20,"type":21},105,"ESTIMATED","INTERVENTIONAL",[24],"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",[27,28],"Congenital Myasthenic Syndrome","CMS","NOT_YET_RECRUITING","2026-07-30",{"date":32,"type":33},"2026-08-04","ACTUAL",{"date":35,"type":21},"2026-09",{"date":37,"type":21},"2030-10",{"name":39,"class":40},"argenx","INDUSTRY"]