[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"facio-scapulo-humeral-dystrophy\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:facio-scapulo-humeral-dystrophy":37},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,69,93],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":14,"conditions":26,"keywords":44,"overallStatus":56,"whyStopped":4,"lastUpdateSubmitDate":57,"lastUpdatePostDateStruct":58,"startDateStruct":61,"completionDateStruct":63,"leadSponsor":65,"locationsCount":68},"100595880","phase-3-a-study-to-evaluate-del-brax-also-referred-to-as-aoc-1020-in-participants-with-fshd-100595880",false,"NCT07038200","A Study to Evaluate Del-brax (Also Referred to as AOC 1020) in Participants With FSHD","A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)","FORTITUDE-3","Inclusion Criteria:\n\n* Clinical and genetic diagnosis of FSHD1 or FSHD2\n* Ability to walk independently at pre-specified walking speed (orthoses and ankle braces allowed) for at least 10 meters at screening\n* Adequate muscle strength based on QMT composite score\n\nExclusion Criteria:\n\n* Breastfeeding, pregnancy, or intent to become pregnant during the study\n* Unwilling or unable to comply with contraceptive requirements\n* Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study\n* Blood Pressure \\> 140\u002F90 mmHg at Screening\n* Treatment with another investigational drug or biological agent within 1 month of Screening or 5 half-lives of the drug, whichever is longer\n* Treatment with an oligonucleotide within 9 months of Screening","ALL","16 Years","70 Years",{"count":21,"type":22},200,"ESTIMATED","INTERVENTIONAL",[25],"PHASE3",[27,28,29,30,31,32,33,34,35,36,37,38,39,40,41,42,43],"Facioscapulohumeral Muscular Dystrophy","FSHD","FSHD - Facioscapulohumeral Muscular Dystrophy","FSHD1","FSHD2","Fascioscapulohumeral Muscular Dystrophy","Fascioscapulohumeral Muscular Dystrophy Type 1","Fascioscapulohumeral Muscular Dystrophy Type 2","Facioscapulohumeral Muscular Dystrophy 1","Facioscapulohumeral Dystrophy","Facio-Scapulo-Humeral Dystrophy","Facioscapulohumeral Muscular Dystrophy 2","Facioscapulohumeral Muscular Dystrophy Type 1 (FSHD1)","FSH Muscular Dystrophy","Landouzy Dejerine Dystrophy","Landouzy-Dejerine Muscular Dystrophy","Landouzy-Dejerine Syndrome",[45,46,47,48,49,50,51,52,15,53,54,55],"Avidity","Avidity Biosciences","del-brax","del brax","delbrax","AOC1020","AOC 1020","delpacibart braxlosiran","FORTITUDE Phase 3","FORTITUDE","FORTITUDE 3","RECRUITING","2026-05-27",{"date":59,"type":60},"2026-05-29","ACTUAL",{"date":62,"type":60},"2025-06-10",{"date":64,"type":22},"2028-07",{"name":66,"class":67},"Avidity Biosciences, Inc.","INDUSTRY",46,{"id":70,"slug":71,"hasResults":11,"nctId":72,"briefTitle":73,"officialTitle":74,"acronym":4,"eligibilityCriteria":75,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":76,"targetDuration":4,"studyType":23,"phases":78,"briefSummary":81,"conditions":82,"keywords":4,"overallStatus":56,"whyStopped":4,"lastUpdateSubmitDate":83,"lastUpdatePostDateStruct":84,"startDateStruct":86,"completionDateStruct":88,"leadSponsor":90,"locationsCount":92},"100526226","phase-1-study-of-aro-dux4-in-adult-and-adolescent-patients-with-facioscapulohumeral-muscular-dystrophy-type-1-100526226","NCT06131983","Study of ARO-DUX4 in Adult and Adolescent Patients With Facioscapulohumeral Muscular Dystrophy Type 1","A Phase1\u002F2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DUX4 in Adult Patients and Adolescent Patients With Facioscapulohumeral Muscular Dystrophy Type 1","Inclusion Criteria:\n\n* Genetically confirmed FSHD1 based on Screening evaluation or source verifiable medical record\n* Clinical severity score between 3 and 8 (scale, 0 to 10)\n* Must have eligible lower extremity muscle for biopsy as determined from MRI by a central reader\n* A 12-lead electrocardiogram (ECG) at Screening with no abnormalities that may compromise participant's safety in the study\n* Participants of childbearing potential and their partners must use highly effective contraception during the study and for at least 12 weeks following the end of study or last dose of study medication, whichever is later. Males must not donate sperm during the study from Day 1 until at least 12 weeks following the end of study or last dose of study medication, whichever is later.\n\nExclusion Criteria:\n\n* Human Immunodeficiency Virus (HIV) infection as shown by presence of anti-HIV antibody (seropositive) at Screening\n* Seropositive for hepatitis B (HBV) or hepatitis C (HCV) at Screening\n* Uncontrolled hypertension\n* Severe cardiovascular disease\n* History of thrombolic events\n* Platelet count less that the lower limit of normal at Screening\n* History or presence of: a hypercoagulable state, nephrotic range proteinuria, antiphospholipid antibody syndrome, myeloproliferative disease, inability to ambulate, use of hormone-based contraceptives.\n* Any contraindication to muscle biopsy or MRI\n\nNote: additional inclusion\u002Fexclusion criteria may apply per protocol",{"count":77,"type":22},60,[79,80],"PHASE1","PHASE2","The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ARO-DUX4 in participants with facioscapulohumeral muscular dystrophy Type 1 (FSHD1). In Part 1 of the study, participants will receive one dose of ARO-DUX4 or placebo. In Part 2 of the study, participants will receive 4 doses of ARO-DUX4 or placebo. Participants who complete Part 1 will have the option to re-screen and re-randomize into Part 2. All participants will undergo pre- and post-dose MRI-guided muscle biopsies (a total of 2 biopsies). Participants who complete Part 1 and enroll in Part 2 will be required to undergo an additional screening biopsy. Participants completing Part 1 or Part 2 may have the option to continue to receive drug in an open-label extension study or may be eligible to participate in later-stage clinical studies.",[37],"2026-02-04",{"date":85,"type":60},"2026-02-06",{"date":87,"type":60},"2024-02-22",{"date":89,"type":22},"2026-12",{"name":91,"class":67},"Arrowhead Pharmaceuticals",17,{"id":94,"slug":95,"hasResults":11,"nctId":96,"briefTitle":97,"officialTitle":97,"acronym":98,"eligibilityCriteria":99,"healthyVolunteers":11,"sex":17,"minAge":100,"maxAge":4,"enrollmentInfo":101,"targetDuration":4,"studyType":23,"phases":103,"briefSummary":105,"conditions":106,"keywords":4,"overallStatus":56,"whyStopped":4,"lastUpdateSubmitDate":118,"lastUpdatePostDateStruct":119,"startDateStruct":121,"completionDateStruct":123,"leadSponsor":125,"locationsCount":128},"100603463","gait-analysis-parameter-and-upper-limb-evaluation-in-adult-patients-with-neurological-or-metabolic-pathology-100603463","NCT07136844","Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology","Acti-Adult","Inclusion Criteria:\n\n* Ambulant patients (i.e. able to walk 10 meters without assistance)\n* Confirmed diagnosis by the investigator based on current gold standard in his\u002Fher disease (genetic testing, clinical criteria, etc.)\n\n  * Myotonic dystrophy type 1 (DM1) and Charcot-Marie-Tooth (CMT) patients should present sensitive of motor signs on physical examination.\n  * Myasthenic patients should be seropositive, and Myasthenia Gravis Foundation of America (MGFA) class II to IV.\n  * Patient with morbid obesity (Body Mass Index\\> or = 35 at inclusion visit).\n* Signed informed consent form by patient him\u002Fherself and patient willing and able to comply with all study procedures.\n\nExclusion Criteria:\n\n* Non-ambulant patients\n* Patients with extreme cognitive disorders that limit their understanding of the exercises to be performed\n* Patients who have undergone a surgical procedure or who have experienced recent trauma (within fewer than 6 months) affecting the upper or lower limbs\n* A concomitant chronic or acute neurological, endocrine, infectious, allergic, or inflammatory pathology within the 3-week period immediately prior to inclusion\n* Patients who are participating in an interventional clinical trial\n* Pregnant or breastfeeding women","18 Years",{"count":102,"type":22},300,[104],"NA","The ActiLiège-Adult study is a prospective, longitudinal, observational study designed to collect natural history data on adult patients with neurological or metabolic diseases affecting movement. Conducted at the Centre de Référence Liégeois des Maladies Neuromusculaires in Liège, Belgium, the study will enroll 300 ambulant patients, including individuals with neuromuscular disorders and obesity. Using the Syde® wearable device, the study aims to continuously monitor motor function in real-life settings over a period of up to two years. The primary objective is to evaluate the utility of digital mobility outcomes, such as the 95th centile of stride velocity (SV95C), as reliable and objective endpoints for future clinical trials.",[107,108,109,110,111,112,37,113,114,115,116,117],"Neuromuscular Diseases","Obesity (Disorder)","Myotonic Dystrophy 1","Myasthenic Syndrome","Charcot Marie Tooth Disease (CMT)","Glycogen Storage Disease Type II Pompe Disease","Myasthenia Gravis","Huntington Disease","Progressive Supranuclear Palsy (PSP)","Hereditary Spastic Paraplegia","Ataxia, Spinocerebellar","2025-08-14",{"date":120,"type":60},"2025-08-22",{"date":122,"type":60},"2024-03-29",{"date":124,"type":22},"2030-12",{"name":126,"class":127},"Centre Hospitalier Universitaire de Liege","OTHER",1]