[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100456529":3},{"organization":4,"armGroups":7,"interventions":10,"overallOfficials":12,"centralContacts":16,"locations":26,"responsibleParty":126,"collaborators":10,"id":128,"slug":129,"hasResults":130,"nctId":131,"briefTitle":132,"officialTitle":133,"acronym":134,"eligibilityCriteria":135,"healthyVolunteers":130,"sex":136,"minAge":10,"maxAge":137,"enrollmentInfo":138,"targetDuration":10,"studyType":141,"phases":10,"briefSummary":142,"conditions":143,"keywords":146,"overallStatus":29,"whyStopped":10,"lastUpdateSubmitDate":149,"lastUpdatePostDateStruct":150,"startDateStruct":153,"completionDateStruct":155,"leadSponsor":157,"locationsCount":158},{"fullName":5,"class":6},"Virginia Commonwealth University","OTHER",[8],{"label":9,"type":10,"description":11,"interventionNames":10},"Congenital Myotonic Dystrophy (CDM)",null,"CDM group includes those aged neonate to 3 years, 11 months at enrollment. Individuals must have a diagnosis of CDM, which is defined as children having symptoms of myotonic dystrophy in the newborn period (\\\u003C30 days), such as hypotonia, feeding or respiratory difficulty, requiring hospitalization to a ward or to the neonatal intensive care unit for more than 72 hours; and a genetic test confirming an expanded trinucleotide (CTG) repeat in the DMPK gene in the child or mother. An expanded CTG repeat size in the child is considered greater than 200 repeats or E1-E4 classification (E1= 200-500, E2=500-1,000, E3=1,000-1,500, E4\\>1,500).",[13],{"name":14,"affiliation":5,"role":15},"Nicholas E. Johnson, MD","PRINCIPAL_INVESTIGATOR",[17,22],{"name":18,"role":19,"phone":20,"phoneExt":10,"email":21},"Ruby Langeslay","CONTACT","804-828-8481","ruby.langeslay@vcuhealth.org",{"name":23,"role":19,"phone":24,"phoneExt":10,"email":25},"Jennifer Raymond","804-828-6318","jennifer.raymond@vcuhealth.org",[27,51,70,89,106],{"facility":28,"status":29,"city":30,"state":31,"zip":32,"country":33,"countryCode":34,"cosmosGeoPoint":35,"geoPoint":40,"contacts":41},"University of California, Los Angeles","RECRUITING","Los Angeles","California","90095","United States","US",{"type":36,"coordinates":37},"Point",[38,39],-118.24368,34.05223,{"lat":39,"lon":38},[42,45,49],{"name":43,"role":19,"phone":10,"phoneExt":10,"email":44},"Merve Kaleli","MKaleli@mednet.ucla.edu",{"name":46,"role":19,"phone":47,"phoneExt":10,"email":48},"Dennis Fernando","310-825-3264","DeFernando@mednet.ucla.edu",{"name":50,"role":15,"phone":10,"phoneExt":10,"email":10},"Perry Shieh, MD, PhD",{"facility":52,"status":29,"city":53,"state":54,"zip":55,"country":33,"countryCode":34,"cosmosGeoPoint":56,"geoPoint":60,"contacts":61},"University of Kansas Medical Center","Fairway","Kansas","66205",{"type":36,"coordinates":57},[58,59],-94.6319,39.02223,{"lat":59,"lon":58},[62,65,68],{"name":63,"role":19,"phone":10,"phoneExt":10,"email":64},"Cassidy Nelson","cnelson15@kumc.edu",{"name":66,"role":19,"phone":10,"phoneExt":10,"email":67},"Michaela Walker","mwalker20@kumc.edu",{"name":69,"role":15,"phone":10,"phoneExt":10,"email":10},"Jeffrey Statland, MD",{"facility":71,"status":29,"city":72,"state":73,"zip":74,"country":33,"countryCode":34,"cosmosGeoPoint":75,"geoPoint":79,"contacts":80},"University of Rochester Medical Center","Rochester","New York","14642",{"type":36,"coordinates":76},[77,78],-77.61556,43.15478,{"lat":78,"lon":77},[81,84,87],{"name":82,"role":19,"phone":10,"phoneExt":10,"email":83},"Jim Hilbert","james_hilbert@urmc.rochester.edu",{"name":85,"role":19,"phone":10,"phoneExt":10,"email":86},"Jeanne Dekdebrun","jeanne_dekdebrun@urmc.rochester.edu",{"name":88,"role":15,"phone":10,"phoneExt":10,"email":10},"Bohoon Lee, MD",{"facility":5,"status":29,"city":90,"state":91,"zip":92,"country":33,"countryCode":34,"cosmosGeoPoint":93,"geoPoint":97,"contacts":98},"Richmond","Virginia","23298",{"type":36,"coordinates":94},[95,96],-77.46026,37.55376,{"lat":96,"lon":95},[99,102,105],{"name":100,"role":19,"phone":10,"phoneExt":10,"email":101},"Shantel Kyles","shantel.brown@vcuhealth.org",{"name":103,"role":19,"phone":10,"phoneExt":10,"email":104},"Jodie Howell","jodie.howell@vcuhealth.org",{"name":14,"role":15,"phone":10,"phoneExt":10,"email":10},{"facility":107,"status":29,"city":108,"state":10,"zip":109,"country":110,"countryCode":111,"cosmosGeoPoint":112,"geoPoint":116,"contacts":117},"Centro Clinico NeMO","Milan","20162","Italy","IT",{"type":36,"coordinates":113},[114,115],9.18951,45.46427,{"lat":115,"lon":114},[118,121,124],{"name":119,"role":19,"phone":10,"phoneExt":10,"email":120},"Michela Nani","michela.nani@centrocliniconemo.it",{"name":122,"role":19,"phone":10,"phoneExt":10,"email":123},"Luca Mauro","luca.mauro@centrocliniconemo.it",{"name":125,"role":15,"phone":10,"phoneExt":10,"email":10},"Valeria Sansone, MD",{"type":127,"investigatorFullName":10,"investigatorTitle":10,"investigatorAffiliation":10,"oldNameTitle":10,"oldOrganization":10},"SPONSOR","100456529","dmcrn-02-001-assessing-pediatric-endpoints-in-dm1-100456529",false,"NCT05224778","DMCRN-02-001: Assessing Pediatric Endpoints in DM1","Assessing Pediatric Endpoints in DM1 (ASPIRE-DM1)","ASPIRE-DM1","Inclusion Criteria:\n\n* Age neonate to 3 years 11 months at enrollment.\n* A diagnosis of CDM, which is defined as children having symptoms of myotonic dystrophy in the newborn period (\\\u003C30 days), such as hypotonia, feeding or respiratory difficulty, requiring hospitalization to a ward or to the neonatal intensive care unit for more than 72 hours; and a genetic test confirming an expanded trinucleotide (CTG) repeat in the DMPK gene in the child or mother. An expanded CTG repeat size in the child is considered greater than 200 repeats or E1-E4 classification (E1= 200-500, E2=500-1,000, E3=1,000-1,500, E4\\>1,500).\n* Guardian is willing and able to sign consent and follow study procedures\n\nExclusion Criteria:\n\n* Any other non-DM1 illness that would interfere with the ability or results of the study in the opinion of the site investigator\n* Significant trauma within one month\n* Internal metal or devices (exclusion for DEXA component)\n* History of bleeding disorder or platelet count \\\u003C50,000\n* History of reaction to local anesthetic","ALL","59 Months",{"count":139,"type":140},50,"ESTIMATED","OBSERVATIONAL","The overall goal of the study is to establish valid clinical endpoint assessments for children with congenital myotonic dystrophy type 1 and develop biomarkers for the condition.",[144,145],"Congenital Myotonic Dystrophy","CDM",[147,148,144,145],"Clinical Research","Myotonic dystrophy","2026-06-08",{"date":151,"type":152},"2026-06-10","ACTUAL",{"date":154,"type":152},"2022-08-24",{"date":156,"type":140},"2026-12",{"name":5,"class":6},5]