[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100432080":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":19,"centralContacts":24,"locations":30,"responsibleParty":111,"collaborators":11,"id":113,"slug":114,"hasResults":115,"nctId":116,"briefTitle":117,"officialTitle":118,"acronym":11,"eligibilityCriteria":119,"healthyVolunteers":115,"sex":120,"minAge":121,"maxAge":122,"enrollmentInfo":123,"targetDuration":11,"studyType":126,"phases":127,"briefSummary":130,"conditions":131,"keywords":11,"overallStatus":33,"whyStopped":11,"lastUpdateSubmitDate":133,"lastUpdatePostDateStruct":134,"startDateStruct":137,"completionDateStruct":139,"leadSponsor":141,"locationsCount":142},{"fullName":5,"class":6},"Wave Life Sciences USA, Inc.","INDUSTRY",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"WVE-N531","EXPERIMENTAL",null,[13],"Drug: WVE-N531",[15],{"type":16,"name":9,"description":17,"armGroupLabels":18,"otherNames":11},"DRUG","WVE-N531 is an antisense oligonucleotide (ASO)",[9],[20],{"name":21,"affiliation":22,"role":23},"Medical Director, MD","Wave Life Sciences","STUDY_DIRECTOR",[25],{"name":26,"role":27,"phone":28,"phoneExt":11,"email":29},"Clinical Operations","CONTACT","855-215-4687","clinicaltrials@wavelifesci.com",[31,52,68,83,93],{"facility":32,"status":33,"city":34,"state":35,"zip":36,"country":37,"countryCode":38,"cosmosGeoPoint":39,"geoPoint":44,"contacts":45},"Arkansas Children's Hospital","RECRUITING","Little Rock","Arkansas","72202-3500","United States","US",{"type":40,"coordinates":41},"Point",[42,43],-92.28959,34.74648,{"lat":43,"lon":42},[46,50],{"name":47,"role":27,"phone":48,"phoneExt":11,"email":49},"Aravindhan Veerapandiyan, Dr","501-364-1850","aveerapandiyan@uams.edu",{"name":47,"role":51,"phone":11,"phoneExt":11,"email":11},"PRINCIPAL_INVESTIGATOR",{"facility":53,"status":33,"city":54,"state":55,"zip":56,"country":37,"countryCode":38,"cosmosGeoPoint":57,"geoPoint":61,"contacts":62},"Rare Disease Research LLC","Atlanta","Georgia","30329",{"type":40,"coordinates":58},[59,60],-84.38798,33.749,{"lat":60,"lon":59},[63,67],{"name":64,"role":27,"phone":65,"phoneExt":11,"email":66},"Scott Batchelor, Dr","404-782-8239","sbatchelor@rarediseaseresearch.com",{"name":64,"role":51,"phone":11,"phoneExt":11,"email":11},{"facility":69,"status":33,"city":70,"state":11,"zip":11,"country":71,"countryCode":72,"cosmosGeoPoint":73,"geoPoint":77,"contacts":78},"Istiklal Hospital\u002F Clinical Research Unit","Amman","Jordan","JO",{"type":40,"coordinates":74},[75,76],35.94503,31.95522,{"lat":76,"lon":75},[79,82],{"name":80,"role":27,"phone":11,"phoneExt":11,"email":81},"Muath Alqurashi, Dr","drmuathalqurashi@yahoo.com",{"name":80,"role":51,"phone":11,"phoneExt":11,"email":11},{"facility":84,"status":33,"city":70,"state":11,"zip":11,"country":71,"countryCode":72,"cosmosGeoPoint":85,"geoPoint":87,"contacts":88},"The Specialty Hospital (TSH)\u002F Advanced Clinical Center",{"type":40,"coordinates":86},[75,76],{"lat":76,"lon":75},[89,92],{"name":90,"role":27,"phone":11,"phoneExt":11,"email":91},"Mai Bader, Dr","dr.mai.bader@hotmail.com",{"name":90,"role":51,"phone":11,"phoneExt":11,"email":11},{"facility":94,"status":33,"city":95,"state":96,"zip":97,"country":98,"countryCode":99,"cosmosGeoPoint":100,"geoPoint":104,"contacts":105},"Oxford Children's Hospital, Oxford University Hospitals NHS Foundation Trust","Headington","Oxford","OX3 9DU","United Kingdom","UK",{"type":40,"coordinates":101},[102,103],-1.21974,51.75737,{"lat":103,"lon":102},[106,110],{"name":107,"role":27,"phone":108,"phoneExt":11,"email":109},"Laurent Servais, MD, PhD","01865 227503","Laurent.Servais@ouh.nhs.uk",{"name":107,"role":51,"phone":11,"phoneExt":11,"email":11},{"type":112,"investigatorFullName":11,"investigatorTitle":11,"investigatorAffiliation":11,"oldNameTitle":11,"oldOrganization":11},"SPONSOR","100432080","phase-1-open-label-study-of-wve-n531-in-patients-with-duchenne-muscular-dystrophy-forward-53-100432080",false,"NCT04906460","Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)","An Open-label Phase 1b\u002F2 Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy","Inclusion Criteria:\n\nPart A and Part B:\n\n1. Part A patients may be screened for Part B upon completion of a washout period of ≥18 weeks from last dose in Part A. New patients may also be screened for Part B\n2. Diagnosis of DMD based on clinical phenotype.\n3. Documented mutation in the DMD gene associated with DMD that is amenable to exon 53 intervention\n4. Score of ≥1 on item 1 or 2 of the shoulder component of the Performance of the Upper Limb (PUL) (Part B ).\n5. Ambulatory or non-ambulatory male\n6. Stable pulmonary and cardiac function, as measured by the following: (Part B):\n\n1\\. Reproducible percent predicted forced vital capacity (FVC) ≥50%; 2. Left ventricular ejection fraction (LVEF) \\>55% in patients \\\u003C10 years of age and \\>45% in patients ≥10 years of age, as measured (and documented) by echocardiogram (ECHO) and\u002For cardiac magnetic resonance imaging (MRI), within 6 months prior to enrollment into the study.\n\n7.Adequate muscle at Screening to perform open muscle biopsies, preferably deltoid.\n\n8\\. Currently on a stable corticosteroid therapy regimen, defined as initiation of systemic corticosteroid therapy that occurred ≥6 months prior to Screening and no changes in dose ≤3 months prior to Screening visit (Part B ).\n\nPart C\n\n1. New patients to be screened for Part C.\n2. Diagnosis of DMD based on clinical phenotype.\n3. Documented mutation in the DMD gene associated with DMD that is amenable to exon 53 intervention\n4. Score of ≥1 on item 1 or 2 of the shoulder component of the Performance of the Upper Limb (PUL) .\n5. Ambulatory male\n6. Stable pulmonary and cardiac function, as measured by the following:\n\n1\\. Reproducible percent predicted forced vital capacity (FVC) ≥50%; 2. Left ventricular ejection fraction (LVEF) \\>55% in patients as measured (and documented) by echocardiogram (ECHO) and\u002For cardiac magnetic resonance imaging (MRI), within 6 months prior to enrollment into the study.\n\n7\\. Adequate muscle at Screening to perform open muscle biopsies, preferably deltoid.\n\n8\\. Currently on a stable corticosteroid therapy regimen, defined as initiation of systemic corticosteroid therapy that occurred ≥6 months prior to Screening and no changes in dose ≤3 months prior to Screening visit .\n\nExclusion Criteria:\n\n1. Clinically significant medical finding on the physical examination other than DMD that, in the judgment of the Investigator, will make the patient unsuitable for participation in, and\u002For completion of the study procedures.\n2. Part B and Part C: Major surgery within 3 months prior to Day 1 or planned major surgery for any time during the study.\n3. Part B: Diagnosis of active alcohol, cannabinoid, or other substance use disorder (except nicotine) within 6 months prior to the Screening visit\n4. Part C: Any recreational substance use (including prescribed cannabinoids), with the exception of nicotine, irrespective of legality, within 2 months prior to Screening and\u002For unwilling to refrain from such use for the duration of the study.","MALE","4 Years","18 Years",{"count":124,"type":125},26,"ESTIMATED","INTERVENTIONAL",[128,129],"PHASE1","PHASE2","This is a Phase 1b\u002F2 open-label study to evaluate the safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and clinical effects of intravenous (IV) WVE-N531 in patients with Duchenne muscular dystrophy (DMD). To participate in the study, patients must have a documented mutation of the DMD gene that is amenable to exon 53 skipping intervention. This study has 3 parts, Part A, Part B, including Part B Extension Arm, and Part C. Part A is completed. Part B is completed. Following completion of Part B, all patients elected to continue to receive study drug in the optional Part B open-label Extension Arm. Part C has been added to the study and will enroll new patients.",[132],"Duchenne Muscular Dystrophy","2025-12-08",{"date":135,"type":136},"2025-12-15","ACTUAL",{"date":138,"type":136},"2021-09-28",{"date":140,"type":125},"2027-04-24",{"name":5,"class":6},5]