[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100361086":3},{"organization":4,"armGroups":7,"interventions":10,"overallOfficials":12,"centralContacts":19,"locations":29,"responsibleParty":334,"collaborators":336,"id":353,"slug":354,"hasResults":355,"nctId":356,"briefTitle":357,"officialTitle":358,"acronym":10,"eligibilityCriteria":359,"healthyVolunteers":355,"sex":360,"minAge":361,"maxAge":362,"enrollmentInfo":363,"targetDuration":10,"studyType":366,"phases":10,"briefSummary":367,"conditions":368,"keywords":371,"overallStatus":32,"whyStopped":10,"lastUpdateSubmitDate":376,"lastUpdatePostDateStruct":377,"startDateStruct":380,"completionDateStruct":382,"leadSponsor":384,"locationsCount":385},{"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",[30,53,69,90,109,129,148,168,188,205,222,233,254,265,285,305,321],{"facility":31,"status":32,"city":33,"state":34,"zip":35,"country":36,"countryCode":37,"cosmosGeoPoint":38,"geoPoint":43,"contacts":44},"University of California, San Diego","RECRUITING","La Jolla","California","92703","United States","US",{"type":39,"coordinates":40},"Point",[41,42],-117.2742,32.84727,{"lat":42,"lon":41},[45,48,51],{"name":46,"role":22,"phone":10,"phoneExt":10,"email":47},"Elizabeth Moreno","e4moreno@health.ucsd.edu",{"name":49,"role":22,"phone":10,"phoneExt":10,"email":50},"Gabriella Penner","gpenner@health.ucsd.edu",{"name":52,"role":15,"phone":10,"phoneExt":10,"email":10},"Chamindra Laverty, MD",{"facility":54,"status":32,"city":55,"state":34,"zip":56,"country":36,"countryCode":37,"cosmosGeoPoint":57,"geoPoint":61,"contacts":62},"University of California, Los Angeles","Los Angeles","90095",{"type":39,"coordinates":58},[59,60],-118.24368,34.05223,{"lat":60,"lon":59},[63,67],{"name":64,"role":22,"phone":65,"phoneExt":10,"email":66},"Dennis Fernando","310-825-3264","DeFernando@mednet.ucla.edu",{"name":68,"role":15,"phone":10,"phoneExt":10,"email":10},"Perry Shieh, MD, PhD",{"facility":70,"status":32,"city":71,"state":72,"zip":73,"country":36,"countryCode":37,"cosmosGeoPoint":74,"geoPoint":78,"contacts":79},"University of Colorado - Denver","Denver","Colorado","80204",{"type":39,"coordinates":75},[76,77],-104.9847,39.73915,{"lat":77,"lon":76},[80,84,88],{"name":81,"role":22,"phone":82,"phoneExt":10,"email":83},"Alyssa Avilez","303-724-4644","alyssa.avilez@cuanschutz.edu",{"name":85,"role":22,"phone":86,"phoneExt":10,"email":87},"Brianna Blume","303-724-6386","brianna.blume@cuanschutz.edu",{"name":89,"role":15,"phone":10,"phoneExt":10,"email":10},"Matthew Wicklund, MD",{"facility":91,"status":32,"city":92,"state":93,"zip":94,"country":36,"countryCode":37,"cosmosGeoPoint":95,"geoPoint":99,"contacts":100},"University of Florida","Gainesville","Florida","32611",{"type":39,"coordinates":96},[97,98],-82.32483,29.65163,{"lat":98,"lon":97},[101,104,107],{"name":102,"role":22,"phone":10,"phoneExt":10,"email":103},"Whitney Miller","whitney.miller@neurology.ufl.edu",{"name":105,"role":22,"phone":10,"phoneExt":10,"email":106},"Donovan Lott","djlottpt@phhp.ufl.edu",{"name":108,"role":15,"phone":10,"phoneExt":10,"email":10},"Sankarsubramoney Subramony, MD",{"facility":110,"status":32,"city":111,"state":112,"zip":113,"country":36,"countryCode":37,"cosmosGeoPoint":114,"geoPoint":118,"contacts":119},"University of Iowa","Iowa City","Iowa","52242",{"type":39,"coordinates":115},[116,117],-91.53017,41.66113,{"lat":117,"lon":116},[120,124,127],{"name":121,"role":22,"phone":122,"phoneExt":10,"email":123},"Maegan Tyrrell","319-356-8323","maegan-tyrrell@uiowa.edu",{"name":125,"role":22,"phone":10,"phoneExt":10,"email":126},"Loraine Brenner","loraine-brenner@uiowa.edu",{"name":128,"role":15,"phone":10,"phoneExt":10,"email":10},"Andrea Swenson, MD",{"facility":130,"status":32,"city":131,"state":132,"zip":133,"country":36,"countryCode":37,"cosmosGeoPoint":134,"geoPoint":138,"contacts":139},"Kansas University Medical Center","Kansas City","Kansas","66160",{"type":39,"coordinates":135},[136,137],-94.62746,39.11417,{"lat":137,"lon":136},[140,143,146],{"name":141,"role":22,"phone":10,"phoneExt":10,"email":142},"Rebecca Clay","rclay@kumc.edu",{"name":144,"role":22,"phone":10,"phoneExt":10,"email":145},"Michaela Walker","mwalker20@kumc.edu",{"name":147,"role":15,"phone":10,"phoneExt":10,"email":10},"Jeffrey Statland, MD",{"facility":18,"status":32,"city":149,"state":150,"zip":151,"country":36,"countryCode":37,"cosmosGeoPoint":152,"geoPoint":156,"contacts":157},"Rochester","New York","14642",{"type":39,"coordinates":153},[154,155],-77.61556,43.15478,{"lat":155,"lon":154},[158,162,166],{"name":159,"role":22,"phone":160,"phoneExt":10,"email":161},"Jim Hilbert","585-273-5590","james_hilbert@urmc.rochester.edu",{"name":163,"role":22,"phone":164,"phoneExt":10,"email":165},"Jeanne Dekdebrun","585-276-4611","Jeanne_dekdebrun@urmc.rochester.edu",{"name":167,"role":15,"phone":10,"phoneExt":10,"email":10},"Johanna Hamel, MD",{"facility":169,"status":32,"city":170,"state":171,"zip":172,"country":36,"countryCode":37,"cosmosGeoPoint":173,"geoPoint":177,"contacts":178},"Ohio State University","Columbus","Ohio","43210",{"type":39,"coordinates":174},[175,176],-82.99879,39.96118,{"lat":176,"lon":175},[179,183,186],{"name":180,"role":22,"phone":181,"phoneExt":10,"email":182},"Kaneshia Hives","614-685-5661","Kaneshia.Hives@osumc.edu",{"name":184,"role":22,"phone":10,"phoneExt":10,"email":185},"Lischele Watkins","lischele.watkins@osumc.edu",{"name":187,"role":15,"phone":10,"phoneExt":10,"email":10},"Bakri Elsheikh, MD",{"facility":189,"status":32,"city":190,"state":191,"zip":192,"country":36,"countryCode":37,"cosmosGeoPoint":193,"geoPoint":197,"contacts":198},"Houston Methodist Neurological Institute","Houston","Texas","77030",{"type":39,"coordinates":194},[195,196],-95.36327,29.76328,{"lat":196,"lon":195},[199,203],{"name":200,"role":22,"phone":201,"phoneExt":10,"email":202},"Kadeesia Brown","346-238-2295","kbrown5@houstonmethodist.org",{"name":204,"role":15,"phone":10,"phoneExt":10,"email":10},"Erika P. Greene",{"facility":5,"status":32,"city":206,"state":207,"zip":208,"country":36,"countryCode":37,"cosmosGeoPoint":209,"geoPoint":213,"contacts":214},"Richmond","Virginia","23298",{"type":39,"coordinates":210},[211,212],-77.46026,37.55376,{"lat":212,"lon":211},[215,218,221],{"name":216,"role":22,"phone":10,"phoneExt":10,"email":217},"Carino Jennings","Carino.Garza@vcuhealth.org",{"name":219,"role":22,"phone":10,"phoneExt":10,"email":220},"Jodie Howell","jodie.howell@vcuhealth.org",{"name":14,"role":15,"phone":10,"phoneExt":10,"email":10},{"facility":223,"status":224,"city":225,"state":10,"zip":10,"country":226,"countryCode":227,"cosmosGeoPoint":228,"geoPoint":232,"contacts":10},"Université de Sherbrooke","ACTIVE_NOT_RECRUITING","Québec","Canada","CA",{"type":39,"coordinates":229},[230,231],-71.21454,46.81228,{"lat":231,"lon":230},{"facility":234,"status":32,"city":235,"state":10,"zip":10,"country":236,"countryCode":237,"cosmosGeoPoint":238,"geoPoint":242,"contacts":243},"Friedrich Baur Institute, Ludwig-Maximilians-Universität München","München","Germany","DE",{"type":39,"coordinates":239},[240,241],13.31243,51.60698,{"lat":241,"lon":240},[244,248,252],{"name":245,"role":22,"phone":246,"phoneExt":10,"email":247},"Benedikt Schoser","+49 (0)89 4400 57400","benedikt.schoser@med.lmu.ede",{"name":249,"role":22,"phone":250,"phoneExt":10,"email":251},"Corinna Wirner-Piotrowski","+49 89 4400 57400","Corinna.Wirner@med.uni-muenchen.de",{"name":253,"role":15,"phone":10,"phoneExt":10,"email":10},"Benedikt Schoser, FEAN",{"facility":255,"status":256,"city":257,"state":10,"zip":10,"country":258,"countryCode":259,"cosmosGeoPoint":260,"geoPoint":264,"contacts":10},"Centro Clinico NeMO","COMPLETED","Milan","Italy","IT",{"type":39,"coordinates":261},[262,263],9.18951,45.46427,{"lat":263,"lon":262},{"facility":266,"status":32,"city":267,"state":10,"zip":10,"country":268,"countryCode":269,"cosmosGeoPoint":270,"geoPoint":274,"contacts":275},"Radboud University Medical Center","Nijmegen","Netherlands","NL",{"type":39,"coordinates":271},[272,273],5.85278,51.8425,{"lat":273,"lon":272},[276,280,283],{"name":277,"role":22,"phone":278,"phoneExt":10,"email":279},"Baziel vanEngelen","(024) 366 8374","Baziel.vanEngelen@radboudumc.nl",{"name":281,"role":22,"phone":10,"phoneExt":10,"email":282},"Monique Plieger","Monique.Plieger@radboudumc.nl",{"name":284,"role":15,"phone":10,"phoneExt":10,"email":10},"Baziel van Engelen, MD",{"facility":286,"status":32,"city":287,"state":10,"zip":10,"country":288,"countryCode":289,"cosmosGeoPoint":290,"geoPoint":294,"contacts":295},"University of Auckland","Auckland","New Zealand","NZ",{"type":39,"coordinates":291},[292,293],174.76349,-36.84853,{"lat":293,"lon":292},[296,299,303],{"name":297,"role":22,"phone":10,"phoneExt":10,"email":298},"Neerja Singh","neerja.singh@auckland.ac.nz",{"name":300,"role":22,"phone":301,"phoneExt":10,"email":302},"Miriam Rodrigues","021896662","mrodrigues@adhb.govt.nz",{"name":304,"role":15,"phone":10,"phoneExt":10,"email":10},"Richard Roxburgh",{"facility":306,"status":32,"city":307,"state":10,"zip":10,"country":308,"countryCode":309,"cosmosGeoPoint":310,"geoPoint":314,"contacts":315},"St. George's, University of London","London","United Kingdom","UK",{"type":39,"coordinates":311},[312,313],-0.12574,51.50853,{"lat":313,"lon":312},[316,320],{"name":317,"role":22,"phone":318,"phoneExt":10,"email":319},"Emma Matthews","020 8725 4162","ematthew@sgul.ac.uk",{"name":317,"role":15,"phone":10,"phoneExt":10,"email":10},{"facility":322,"status":32,"city":307,"state":10,"zip":10,"country":308,"countryCode":309,"cosmosGeoPoint":323,"geoPoint":325,"contacts":326},"University College London",{"type":39,"coordinates":324},[312,313],{"lat":313,"lon":312},[327,330,333],{"name":328,"role":22,"phone":10,"phoneExt":10,"email":329},"Chris Turner","chris.turner7@nhs.net",{"name":331,"role":22,"phone":10,"phoneExt":10,"email":332},"Iqraa Afzal","iqraa.afzal@nhs.net",{"name":328,"role":15,"phone":10,"phoneExt":10,"email":10},{"type":335,"investigatorFullName":10,"investigatorTitle":10,"investigatorAffiliation":10,"oldNameTitle":10,"oldOrganization":10},"SPONSOR",[337,338,340,341,342,343,345,347,349,350,352],{"name":18,"class":6},{"name":339,"class":6},"Stanford University",{"name":169,"class":6},{"name":91,"class":6},{"name":110,"class":6},{"name":344,"class":6},"Ludwig-Maximilians - University of Munich",{"name":346,"class":6},"Fondazione Serena Onlus - Centro Clinico NeMO Milano",{"name":348,"class":6},"The Methodist Hospital Research Institute",{"name":266,"class":6},{"name":351,"class":6},"University College London Hospitals",{"name":54,"class":6},"100361086","estab-biomarkers-and-clinical-endpoints-in-myotonic-dystrophy-type-1-end-dm1-100361086",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":364,"type":365},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",[369,370],"Myotonic Dystrophy 1","DM1",[372,373,374,375],"Myotonic Dystrophy","END DM-1","Muscular Dystophy","DMCRN","2026-06-08",{"date":378,"type":379},"2026-06-10","ACTUAL",{"date":381,"type":379},"2019-01-01",{"date":383,"type":365},"2026-12-01",{"name":5,"class":6},17]