[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"dm1\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:dm1":26},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,5,0,[8,56,93,116,137],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":14,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":22,"studyType":23,"phases":4,"briefSummary":24,"conditions":25,"keywords":33,"overallStatus":43,"whyStopped":4,"lastUpdateSubmitDate":44,"lastUpdatePostDateStruct":45,"startDateStruct":48,"completionDateStruct":50,"leadSponsor":52,"locationsCount":55},"100053754","establishing-biomarkers-and-clinical-endpoints-in-myotonic-dystrophy-type-1-end-dm1-extension-100053754",false,"NCT07700225","Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) Extension","END-EXT","Inclusion Criteria:\n\n* Age 18 to 70 years (inclusive)\n* Written, voluntary informed consent must be obtained prior to any study procedures. In cases where a Legally Authorized Representative (LAR) provides consent, verbal assent will be obtained from the subject, as determined by the investigator and documented directly on the consent form. Capacity to consent will be determined by the neurologist at the Baseline visit and will be signed off on the Inclusion\u002FExclusion checklist.\n* Clinical diagnosis of DM1 based on research criteria or positive genetic test. The research criteria for clinical diagnosis of DM1 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. OR 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 3 days or more; and a genetic test suspicious of an expanded trinucleotide (CTG) repeat in the DMPK gene in the child or first degree relative. 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\nExclusion Criteria:\n\n* Symptomatic renal or liver disease, uncontrolled diabetes or thyroid disorder, or active malignancy other than in situ skin cancer.\n* Current alcohol or substance use disorder.\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* Use of mexiletine or other anti-myotonia agents within 72 hours of any study visit.","ALL","18 Years","70 Years",{"count":20,"type":21},1000,"ESTIMATED","4 Years","OBSERVATIONAL","Myotonic Dystrophy type 1 (DM1) is an autosomal dominant multisystemic disorder that causes progressive disability and shortened life expectancy. It is characterized by progressive weakness and myotonia, which preferentially affects the craniofacial, hand, and distal leg muscles. Many patients also experience difficulties with cognition, cardiac arrhythmias, respiratory failure, or cataracts. Currently there is no treatment to slow progression or reverse the symptoms.",[26,27,28,29,30,31,32],"DM1","Myotonic Dystrophy","Myotonic Dystrophy 1","Myotonic Dystrophy Type 1","Myotonic Dystrophy Type-1","Myotonic Dystrophy, Type 1 (DM1)","Myotonic Muscular Dystrophy",[26,14,34,29,35,36,37,38,39,40,41,42],"END-DM1 Extension","Steinert's Disease","Muscular Dystrophy","Neuromuscular Disease","DMPK","Natural History","Myotonia","DMCRN","Myotonic Dystrophy Clinical Research Network","RECRUITING","2026-07-08",{"date":46,"type":47},"2026-07-13","ACTUAL",{"date":49,"type":21},"2026-07",{"date":51,"type":21},"2032-12",{"name":53,"class":54},"Virginia Commonwealth University","OTHER",1,{"id":57,"slug":58,"hasResults":11,"nctId":59,"briefTitle":60,"officialTitle":61,"acronym":4,"eligibilityCriteria":62,"healthyVolunteers":11,"sex":16,"minAge":63,"maxAge":4,"enrollmentInfo":64,"targetDuration":4,"studyType":66,"phases":67,"briefSummary":69,"conditions":70,"keywords":74,"overallStatus":43,"whyStopped":4,"lastUpdateSubmitDate":83,"lastUpdatePostDateStruct":84,"startDateStruct":86,"completionDateStruct":88,"leadSponsor":90,"locationsCount":92},"100630381","phase-3-efficacy-safety-and-tolerability-of-zeleciment-basivarsen-dyne-101-in-participants-with-myotonic-dystrophy-type-1-100630381","NCT07486934","Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1","A Phase 3, Randomized, Double-Blind, 48-Week Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of DYNE-101 Administered to Participants With Myotonic Dystrophy Type 1","Inclusion Criteria:\n\n* Diagnosis of DM1 confirmed by molecular genetics with trinucleotide repeat size greater than (\\>) 100. Historical results from clinical testing are acceptable.\n* Able to walk 10 meters and complete 5 times sit to stand independently (inserts or supports that don't go above the ankle are allowed).\n* Body mass index (BMI) less than (\\\u003C) 35 kilograms per meter square (kg\u002Fm\\^2).\n\nExclusion Criteria:\n\n* A known diagnosis of congenital DM1.\n* History of major surgical procedure (based on Investigator judgment) within 12 weeks prior to the start of screening, with the exception of implanted pacemaker or defibrillator.\n* Use of glucagon-like peptide 1 (GLP-1) agonist\u002Fincretin medications including semaglutide, dulaglutide, liraglutide, exenatide, or tirzepatide within a period of 5 half-lives of the medication prior to performing screening assessments.\n\nNote: Other inclusion and exclusion criteria may apply.","16 Years",{"count":65,"type":21},150,"INTERVENTIONAL",[68],"PHASE3","The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment basivarsen (DYNE-101) for the treatment of myotonic dystrophy 1 (DM1).",[71,26,27,72,73],"Myotonic Dystrophy Type 1 (DM1)","Steinert Disease","Steinert",[26,27,28,40,71,75,76,72,73,32,77,78,79,80,81,82],"Dystrophy Myotonic","Myotonic Disorders","HARMONIA","Dyne Therapeutics","Dyne","DYNE-101","zeleciment basivarsen","z-basivarsen","2026-06-26",{"date":85,"type":47},"2026-06-30",{"date":87,"type":47},"2026-05-14",{"date":89,"type":21},"2029-01",{"name":78,"class":91},"INDUSTRY",14,{"id":94,"slug":95,"hasResults":11,"nctId":96,"briefTitle":97,"officialTitle":98,"acronym":4,"eligibilityCriteria":99,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":100,"targetDuration":4,"studyType":23,"phases":4,"briefSummary":102,"conditions":103,"keywords":104,"overallStatus":43,"whyStopped":4,"lastUpdateSubmitDate":107,"lastUpdatePostDateStruct":108,"startDateStruct":110,"completionDateStruct":112,"leadSponsor":114,"locationsCount":115},"100361086","estab-biomarkers-and-clinical-endpoints-in-myotonic-dystrophy-type-1-end-dm1-100361086","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.",{"count":101,"type":21},700,"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",[28,26],[27,105,106,41],"END DM-1","Muscular Dystophy","2026-06-08",{"date":109,"type":47},"2026-06-10",{"date":111,"type":47},"2019-01-01",{"date":113,"type":21},"2026-12-01",{"name":53,"class":54},17,{"id":117,"slug":118,"hasResults":11,"nctId":119,"briefTitle":120,"officialTitle":121,"acronym":122,"eligibilityCriteria":123,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":124,"enrollmentInfo":125,"targetDuration":4,"studyType":23,"phases":4,"briefSummary":126,"conditions":127,"keywords":4,"overallStatus":43,"whyStopped":4,"lastUpdateSubmitDate":128,"lastUpdatePostDateStruct":129,"startDateStruct":131,"completionDateStruct":133,"leadSponsor":135,"locationsCount":55},"100631796","remote-assessments-and-genetic-determinants-of-myotonic-dystrophy-100631796","NCT07505342","Remote Assessments and Genetic Determinants of Myotonic Dystrophy","REACH DM - Remote Assessments and Genetic Determinants of Myotonic Dystrophy","REACH-DM","Inclusion Criteria:\n\n* Age 18-88 years\n* Clinical diagnosis of DM1\n* English speaking\n* Able to provide informed consent\n* Available wifi","88 Years",{"count":20,"type":21},"The goal of this observational study, conducted in participants' homes and requiring no travel to a study site, is to better understand disease variability in people with myotonic dystrophy type 1 (DM1) and to identify effective ways to measure symptoms.\n\nMyotonic dystrophy is one of the most variable diseases. Some people develop symptoms when they are young, others when they are much older. In the same family, some people may have mild problems, while others are strongly affected. The goal of this study is to find out more about what is causing these differences. To accomplish this, investigators will study the effects of DM1 on skeletal and smooth muscles, the heart, and the nervous system. Then, investigators will evaluate genetic differences with a blood sample.\n\n* Participants will receive a toolkit in the mail which includes all necessary equipment to participate in the study, including an iPad with video conferencing software.\n* Then the study team will connect with participants via videoconferencing for medical interview about DM1 symptoms and functional assessments\n* Participants will have their blood drawn in a lab in their community or using a home draw device, and ship it to us for research genetic analysis\n* Participants can chose to have their research genetic test result returned to them",[71,26],"2026-03-25",{"date":130,"type":47},"2026-04-01",{"date":132,"type":47},"2022-05-10",{"date":134,"type":21},"2030-01-01",{"name":136,"class":54},"University of Rochester",{"id":138,"slug":139,"hasResults":11,"nctId":140,"briefTitle":141,"officialTitle":142,"acronym":143,"eligibilityCriteria":144,"healthyVolunteers":145,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":146,"targetDuration":148,"studyType":23,"phases":4,"briefSummary":149,"conditions":150,"keywords":4,"overallStatus":43,"whyStopped":4,"lastUpdateSubmitDate":152,"lastUpdatePostDateStruct":153,"startDateStruct":155,"completionDateStruct":157,"leadSponsor":159,"locationsCount":161},"100622578","the-spanish-national-registry-for-myotonic-dystrophy-type-1-100622578","NCT07385443","The Spanish National Registry for Myotonic Dystrophy Type 1","Creación de un Nodo Integral Para la Distrofia Miotónica Tipo 1 en España: Registro clínico, Mapas genómicos, epigenómicos y proteómicos (DM1-Hub)","DM1-Hub","Inclusion Criteria:\n\n* Confirmed diagnosis of Myotonic Dystrophy Type 1 (DM1) through genetic testing.\n\nExclusion Criteria:\n\n* There are no exclusion criteria for the registry",true,{"count":147,"type":21},3000,"10 Years","Myotonic Dystrophy Type 1 (DM1) is a rare genetic neuromuscular condition that can affect multiple organs and varies widely in how it presents. DM1 is the most common form of adult-onset muscular dystrophy, with an estimated prevalence of approximately 1-5 per 10,000 people. In Spain, the condition shows notable regional differences, making it especially important to understand its characteristics within the population.\n\nThe aim of this study is to support a research initiative designed to better characterise DM1. We are developing a comprehensive national registry, collecting patient-reported information, clinical data and omics data that will improve our understanding of the disease and help identify individuals who may be eligible for clinical trials.",[28,26,29,151,72],"Myotonic Dystrophy, Congenital","2026-01-29",{"date":154,"type":47},"2026-02-04",{"date":156,"type":47},"2025-06-02",{"date":158,"type":21},"2026-12-31",{"name":160,"class":54},"Fundació Institut Germans Trias i Pujol",8]