[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100575624":3},{"organization":4,"armGroups":7,"interventions":10,"overallOfficials":12,"centralContacts":37,"locations":46,"responsibleParty":150,"collaborators":153,"id":156,"slug":157,"hasResults":158,"nctId":159,"briefTitle":160,"officialTitle":160,"acronym":10,"eligibilityCriteria":161,"healthyVolunteers":158,"sex":162,"minAge":163,"maxAge":164,"enrollmentInfo":165,"targetDuration":168,"studyType":169,"phases":10,"briefSummary":170,"conditions":171,"keywords":173,"overallStatus":178,"whyStopped":10,"lastUpdateSubmitDate":179,"lastUpdatePostDateStruct":180,"startDateStruct":183,"completionDateStruct":185,"leadSponsor":187,"locationsCount":188},{"fullName":5,"class":6},"Stanford University","OTHER",[8],{"label":9,"type":10,"description":11,"interventionNames":10},"Individuals with Nemaline Myopathy",null,"All participants in the study will have a diagnosis of Nemaline Myopathy, with either a pathogenic or likely pathogenic mutation in ACTA1 (AD) or NEB (AR). Participants can be either ambulatory or non-ambulatory and must be between the ages of 0-18.",[13,16,18,20,23,25,28,31,34],{"name":14,"affiliation":5,"role":15},"Carolina Tesi-Rocha, MD","PRINCIPAL_INVESTIGATOR",{"name":17,"affiliation":5,"role":15},"Tina Duong, PT, PhD",{"name":19,"affiliation":5,"role":15},"John W Day, MD, PhD",{"name":21,"affiliation":22,"role":15},"Leslie Hayes, MD","Boston Children's Hospital",{"name":24,"affiliation":22,"role":15},"Alan Beggs, PhD",{"name":26,"affiliation":27,"role":15},"Jim Dowling, MD, PhD","The Hospital for Sick Children",{"name":29,"affiliation":30,"role":15},"Richard Finkel, MD","St Jude Children's Hospital",{"name":32,"affiliation":33,"role":15},"Carsten Bonnemann, MD, PhD","National Institutes of Health (NIH)",{"name":35,"affiliation":36,"role":15},"Kaitlin Batley, MD","UT Southwestern Medical Centre",[38,42],{"name":14,"role":39,"phone":40,"phoneExt":10,"email":41},"CONTACT","650-723-0993","ctesiroc@stanford.edu",{"name":43,"role":39,"phone":44,"phoneExt":10,"email":45},"Sarah Ismail, BSc","650-460-4596","sismail@stanford.edu",[47,66,82,98,114,130],{"facility":48,"status":10,"city":49,"state":50,"zip":51,"country":52,"countryCode":53,"cosmosGeoPoint":54,"geoPoint":59,"contacts":60},"Stanford University\u002FLucile Packard Children's Hospital","Palo Alto","California","94304","United States","US",{"type":55,"coordinates":56},"Point",[57,58],-122.14302,37.44188,{"lat":58,"lon":57},[61,62,63,65],{"name":43,"role":39,"phone":44,"phoneExt":10,"email":45},{"name":14,"role":39,"phone":10,"phoneExt":10,"email":10},{"name":64,"role":39,"phone":10,"phoneExt":10,"email":10},"Tina Duong, PhD, PT",{"name":19,"role":39,"phone":10,"phoneExt":10,"email":10},{"facility":67,"status":10,"city":68,"state":69,"zip":70,"country":52,"countryCode":53,"cosmosGeoPoint":71,"geoPoint":75,"contacts":76},"National Institute of Health","Bethesda","Maryland","20892",{"type":55,"coordinates":72},[73,74],-77.10026,38.98067,{"lat":74,"lon":73},[77,80],{"name":78,"role":39,"phone":10,"phoneExt":10,"email":79},"Christopher Mendoza","christopher.mendoza2@nih.gov",{"name":81,"role":39,"phone":10,"phoneExt":10,"email":10},"Carsten Bonnemann, MD",{"facility":22,"status":10,"city":83,"state":84,"zip":85,"country":52,"countryCode":53,"cosmosGeoPoint":86,"geoPoint":90,"contacts":91},"Boston","Massachusetts","02115",{"type":55,"coordinates":87},[88,89],-71.05977,42.35843,{"lat":89,"lon":88},[92,96,97],{"name":93,"role":39,"phone":94,"phoneExt":10,"email":95},"Aleah Pagan, BA","617-919-7498","aleah.pagan@childrens.harvard.edu",{"name":24,"role":39,"phone":10,"phoneExt":10,"email":10},{"name":21,"role":39,"phone":10,"phoneExt":10,"email":10},{"facility":99,"status":10,"city":100,"state":101,"zip":102,"country":52,"countryCode":53,"cosmosGeoPoint":103,"geoPoint":107,"contacts":108},"St Jude Children's Research Hospital","Memphis","Tennessee","38105",{"type":55,"coordinates":104},[105,106],-90.04898,35.14953,{"lat":106,"lon":105},[109,113],{"name":110,"role":39,"phone":111,"phoneExt":10,"email":112},"Jean Laboe, MSN, RN","901-595-1693","jean.laboe@stjude.org",{"name":29,"role":39,"phone":10,"phoneExt":10,"email":10},{"facility":115,"status":10,"city":116,"state":117,"zip":118,"country":52,"countryCode":53,"cosmosGeoPoint":119,"geoPoint":123,"contacts":124},"UT Southwestern Medical Centre\u002FChildren's Health Dallas","Dallas","Texas","75207",{"type":55,"coordinates":120},[121,122],-96.80667,32.78306,{"lat":122,"lon":121},[125,129],{"name":126,"role":39,"phone":127,"phoneExt":10,"email":128},"Holly Lawrence, CCRC","214-456-9561","holly.lawrence@utsouthwestern.edu",{"name":35,"role":39,"phone":10,"phoneExt":10,"email":10},{"facility":27,"status":10,"city":131,"state":132,"zip":133,"country":134,"countryCode":135,"cosmosGeoPoint":136,"geoPoint":140,"contacts":141},"Toronto","Ontario","M5G 1E8","Canada","CA",{"type":55,"coordinates":137},[138,139],-79.39864,43.70643,{"lat":139,"lon":138},[142,146,148],{"name":143,"role":39,"phone":144,"phoneExt":10,"email":145},"Ana Stosic, MSc","416-648-6831","ana.stosic@sickkids.ca",{"name":147,"role":39,"phone":10,"phoneExt":10,"email":10},"Jim Dowling, MD",{"name":149,"role":39,"phone":10,"phoneExt":10,"email":10},"Kimberley Amburgey, MSc",{"type":15,"investigatorFullName":151,"investigatorTitle":152,"investigatorAffiliation":5,"oldNameTitle":10,"oldOrganization":10},"Carolina Tesi-Rocha","Clinical Professor",[154],{"name":155,"class":6},"A Foundation Building Strength","100575624","nemaline-myopathy-clinical-research-network-nm-ctrn-100575624",false,"NCT06774703","Nemaline Myopathy Clinical Research Network (NM-CTRN)","Inclusion Criteria:\n\n* 0-18 years of age at recruitment\n* Confirmation of Nemaline Myopathy (pathogenic or likely pathogenic mutations in ACTA1 (AD) or NEB (AR)\n* Patient and\u002For parent or legal guardian must be willing and able to provide informed consent\n\nExclusion Criteria:\n\n* Clinically significant medical finding on the physical examination, other than NM, that the Investigator deems unsuitable for participation in and\u002For completion of the study procedures\n* Any confirmed chronic or acute condition or disease affecting any system(s), which could interfere with the results of the study and\u002For the compliance with the study procedures. This will be subject to the clinical judgement of the Principal Investigator (PI)\n* Participants of ongoing (interventional) clinical trials that assess the efficacy of potential treatments will be excluded\n* Safety concerns","ALL","0 Years","18 Years",{"count":166,"type":167},50,"ESTIMATED","3 Years","OBSERVATIONAL","The goal of this study is to establish a research network to help define the natural disease history and clinical outcome measures for Nemaline Myopathy (NM).",[172],"Nemaline Myopathy",[174,175,176,177],"Neuromuscular disease","Observational study","Congenital myopathy","Nemaline rod","NOT_YET_RECRUITING","2025-01-08",{"date":181,"type":182},"2025-01-14","ACTUAL",{"date":184,"type":167},"2025-01",{"date":186,"type":167},"2029-12",{"name":5,"class":6},6]