[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100361086":3},{"organization":4,"armGroups":7,"interventions":10,"overallOfficials":12,"centralContacts":19,"locations":29,"responsibleParty":327,"collaborators":329,"id":346,"slug":10,"hasResults":347,"nctId":348,"briefTitle":349,"officialTitle":350,"acronym":10,"eligibilityCriteria":351,"healthyVolunteers":347,"sex":352,"minAge":353,"maxAge":354,"enrollmentInfo":355,"targetDuration":10,"studyType":358,"phases":10,"briefSummary":359,"conditions":360,"keywords":363,"overallStatus":32,"whyStopped":10,"lastUpdateSubmitDate":368,"lastUpdatePostDateStruct":369,"startDateStruct":372,"completionDateStruct":374,"leadSponsor":376,"locationsCount":377},{"fullName":5,"class":6},"Virginia Commonwealth University","OTHER",[8],{"label":9,"type":10,"description":11,"interventionNames":10},"Study Visits",null,"Patients will receive standard of care as determined by their treating physician. Study visits occur at baseline\u002F0 months, 12 months, and 24 months",[13,16],{"name":14,"affiliation":5,"role":15},"Nicholas Johnson, MD","PRINCIPAL_INVESTIGATOR",{"name":17,"affiliation":18,"role":15},"Charles Thornton, MD","University of Rochester",[20,25],{"name":21,"role":22,"phone":23,"phoneExt":10,"email":24},"Jennifer Raymond","CONTACT","804-828-6318","jennifer.raymond@vcuhealth.org",{"name":26,"role":22,"phone":27,"phoneExt":10,"email":28},"Ruby Langeslay","804-828-8481","ruby.langeslay@vcuhealth.org",[30,52,68,89,108,128,147,167,187,204,221,231,251,261,280,299,314],{"facility":31,"status":32,"city":33,"state":34,"zip":35,"country":36,"cosmosGeoPoint":37,"geoPoint":42,"contacts":43},"University of California, San Diego","RECRUITING","La Jolla","California","92703","United States",{"type":38,"coordinates":39},"Point",[40,41],-117.2742,32.84727,{"lat":41,"lon":40},[44,47,50],{"name":45,"role":22,"phone":10,"phoneExt":10,"email":46},"Elizabeth Moreno","e4moreno@health.ucsd.edu",{"name":48,"role":22,"phone":10,"phoneExt":10,"email":49},"Gabriella Penner","gpenner@health.ucsd.edu",{"name":51,"role":15,"phone":10,"phoneExt":10,"email":10},"Chamindra Laverty, MD",{"facility":53,"status":32,"city":54,"state":34,"zip":55,"country":36,"cosmosGeoPoint":56,"geoPoint":60,"contacts":61},"University of California, Los Angeles","Los Angeles","90095",{"type":38,"coordinates":57},[58,59],-118.24368,34.05223,{"lat":59,"lon":58},[62,66],{"name":63,"role":22,"phone":64,"phoneExt":10,"email":65},"Dennis Fernando","310-825-3264","DeFernando@mednet.ucla.edu",{"name":67,"role":15,"phone":10,"phoneExt":10,"email":10},"Perry Shieh, MD, PhD",{"facility":69,"status":32,"city":70,"state":71,"zip":72,"country":36,"cosmosGeoPoint":73,"geoPoint":77,"contacts":78},"University of Colorado - 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Greene",{"facility":5,"status":32,"city":205,"state":206,"zip":207,"country":36,"cosmosGeoPoint":208,"geoPoint":212,"contacts":213},"Richmond","Virginia","23298",{"type":38,"coordinates":209},[210,211],-77.46026,37.55376,{"lat":211,"lon":210},[214,217,220],{"name":215,"role":22,"phone":10,"phoneExt":10,"email":216},"Carino Jennings","Carino.Garza@vcuhealth.org",{"name":218,"role":22,"phone":10,"phoneExt":10,"email":219},"Jodie Howell","jodie.howell@vcuhealth.org",{"name":14,"role":15,"phone":10,"phoneExt":10,"email":10},{"facility":222,"status":223,"city":224,"state":10,"zip":10,"country":225,"cosmosGeoPoint":226,"geoPoint":230,"contacts":10},"Université de Sherbrooke","ACTIVE_NOT_RECRUITING","Québec","Canada",{"type":38,"coordinates":227},[228,229],-71.21454,46.81228,{"lat":229,"lon":228},{"facility":232,"status":32,"city":233,"state":10,"zip":10,"country":234,"cosmosGeoPoint":235,"geoPoint":239,"contacts":240},"Friedrich Baur Institute, Ludwig-Maximilians-Universität München","München","Germany",{"type":38,"coordinates":236},[237,238],13.31243,51.60698,{"lat":238,"lon":237},[241,245,249],{"name":242,"role":22,"phone":243,"phoneExt":10,"email":244},"Benedikt Schoser","+49 (0)89 4400 57400","benedikt.schoser@med.lmu.ede",{"name":246,"role":22,"phone":247,"phoneExt":10,"email":248},"Corinna Wirner-Piotrowski","+49 89 4400 57400","Corinna.Wirner@med.uni-muenchen.de",{"name":250,"role":15,"phone":10,"phoneExt":10,"email":10},"Benedikt Schoser, FEAN",{"facility":252,"status":253,"city":254,"state":10,"zip":10,"country":255,"cosmosGeoPoint":256,"geoPoint":260,"contacts":10},"Centro Clinico NeMO","COMPLETED","Milan","Italy",{"type":38,"coordinates":257},[258,259],9.18951,45.46427,{"lat":259,"lon":258},{"facility":262,"status":32,"city":263,"state":10,"zip":10,"country":264,"cosmosGeoPoint":265,"geoPoint":269,"contacts":270},"Radboud University Medical Center","Nijmegen","Netherlands",{"type":38,"coordinates":266},[267,268],5.85278,51.8425,{"lat":268,"lon":267},[271,275,278],{"name":272,"role":22,"phone":273,"phoneExt":10,"email":274},"Baziel vanEngelen","(024) 366 8374","Baziel.vanEngelen@radboudumc.nl",{"name":276,"role":22,"phone":10,"phoneExt":10,"email":277},"Monique Plieger","Monique.Plieger@radboudumc.nl",{"name":279,"role":15,"phone":10,"phoneExt":10,"email":10},"Baziel van Engelen, MD",{"facility":281,"status":32,"city":282,"state":10,"zip":10,"country":283,"cosmosGeoPoint":284,"geoPoint":288,"contacts":289},"University of Auckland","Auckland","New Zealand",{"type":38,"coordinates":285},[286,287],174.76349,-36.84853,{"lat":287,"lon":286},[290,293,297],{"name":291,"role":22,"phone":10,"phoneExt":10,"email":292},"Neerja Singh","neerja.singh@auckland.ac.nz",{"name":294,"role":22,"phone":295,"phoneExt":10,"email":296},"Miriam Rodrigues","021896662","mrodrigues@adhb.govt.nz",{"name":298,"role":15,"phone":10,"phoneExt":10,"email":10},"Richard Roxburgh",{"facility":300,"status":32,"city":301,"state":10,"zip":10,"country":302,"cosmosGeoPoint":303,"geoPoint":307,"contacts":308},"St. George's, University of London","London","United Kingdom",{"type":38,"coordinates":304},[305,306],-0.12574,51.50853,{"lat":306,"lon":305},[309,313],{"name":310,"role":22,"phone":311,"phoneExt":10,"email":312},"Emma Matthews","020 8725 4162","ematthew@sgul.ac.uk",{"name":310,"role":15,"phone":10,"phoneExt":10,"email":10},{"facility":315,"status":32,"city":301,"state":10,"zip":10,"country":302,"cosmosGeoPoint":316,"geoPoint":318,"contacts":319},"University College London",{"type":38,"coordinates":317},[305,306],{"lat":306,"lon":305},[320,323,326],{"name":321,"role":22,"phone":10,"phoneExt":10,"email":322},"Chris Turner","chris.turner7@nhs.net",{"name":324,"role":22,"phone":10,"phoneExt":10,"email":325},"Iqraa Afzal","iqraa.afzal@nhs.net",{"name":321,"role":15,"phone":10,"phoneExt":10,"email":10},{"type":328,"investigatorFullName":10,"investigatorTitle":10,"investigatorAffiliation":10,"oldNameTitle":10,"oldOrganization":10},"SPONSOR",[330,331,333,334,335,336,338,340,342,343,345],{"name":18,"class":6},{"name":332,"class":6},"Stanford University",{"name":168,"class":6},{"name":90,"class":6},{"name":109,"class":6},{"name":337,"class":6},"Ludwig-Maximilians - University of Munich",{"name":339,"class":6},"Fondazione Serena Onlus - Centro Clinico NeMO Milano",{"name":341,"class":6},"The Methodist Hospital Research Institute",{"name":262,"class":6},{"name":344,"class":6},"University College London Hospitals",{"name":53,"class":6},"100361086",false,"NCT03981575","Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)","Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)","Inclusion criteria:\n\n* Age 18 to 70 (inclusive)\n* Competent to provide informed consent\n* Clinical diagnosis of DM1 based on research criteria1 or positive genetic test\n* Comment: The clinical research criteria require myotonia, muscle weakness in a characteristic distribution, and history of similar findings in a first degree relative. Genetic testing confirmed the diagnosis of DM1 in \\> 99% of individuals who satisfied these criteria.2\n\nExclusion criteria:\n\n* Symptomatic renal or liver disease, uncontrolled diabetes or thyroid disorder, or active malignancy other than skin cancer.\n* Current alcohol or substance abuse\n* Concurrent enrollment in clinical trial for DM1, or participation in trial within 6 months of entry.\n* Concurrent pregnancy or planned pregnancy during the course of the study.\n* Concurrent medical condition that would, in the opinion of the investigator or clinical evaluator, compromise performance on study measures.\n* Note: non-ambulatory participants are not excluded, but are limited to \\\u003C15% of enrollment.\n\nInclusion criteria for participants in the muscle biopsy sub-study:\n\n• Of the 95 patients undergoing the tibialis anterior muscle biopsy, at least half will have at least moderate weakness of ankle dorsiflexion, defined as MRC score ≤ 4+. This is in order to obtain a muscle tissue sample in a person more severely affected with myotonic dystrophy. Approximately 10 patients at each site will undergo the muscle biopsy.\n\nExclusion criteria for 95 participants in the muscle biopsy sub-study:\n\n* Known CTG repeat expansion size less than 100 repeats, unless there are clear cut signs of limb weakness and muscle wasting. This is in order to obtain a muscle tissue sample in a person more severely affected with myotonic dystrophy.\n* Use of anticoagulant such as warfarin or a direct oral anticoagulant (e.g. dabigatran) due to the increased risk of bleeding.\n* Use of aspirin or non-steroidal anti-inflammatory agents should be discontinued 3 days prior to the biopsy procedure, if possible.\n* Platelet count \\\u003C50,000 (if known) due to the increased risk of bleeding.\n* History of a bleeding disorder due to the increased risk of bleeding.\n* Advanced wasting of tibialis anterior (TA) muscle that precludes needle muscle biopsy in order to ensure that a sample taken would be of muscle and not just fat and fascia.\n* Previous muscle biopsy of either TA in order to provide muscle tissue samples of non-biopsied muscles.","ALL","18 Years","70 Years",{"count":356,"type":357},700,"ESTIMATED","OBSERVATIONAL","Building on previous work of the Myotonic Dystrophy Clinical Research Network (DMCRN), the present study seeks to overcome insufficient data on natural history; lack of reliable biomarkers; and incomplete characterization and limited biological understanding of the phenotypic heterogeneity of Myotonic Dystrophy 1 by examining strategies to improve the reliability by making further refinements in our sample collection and analysis procedures by developing strategies for managing patient heterogeneity going forward.\n\nFunding Source- FDA OOPD",[361,362],"Myotonic Dystrophy 1","DM1",[364,365,366,367],"Myotonic Dystrophy","END DM-1","Muscular Dystophy","DMCRN","2026-06-08",{"date":370,"type":371},"2026-06-10","ACTUAL",{"date":373,"type":371},"2019-01-01",{"date":375,"type":357},"2026-12-01",{"name":5,"class":6},17]