[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"myotonic-dystrophy-type-1-dm1\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:myotonic-dystrophy-type-1-dm1":31},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,7,0,[8,56,93,130,175,198,219],{"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":99,"eligibilityCriteria":100,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":101,"targetDuration":4,"studyType":66,"phases":103,"briefSummary":105,"conditions":106,"keywords":110,"overallStatus":43,"whyStopped":4,"lastUpdateSubmitDate":120,"lastUpdatePostDateStruct":121,"startDateStruct":123,"completionDateStruct":125,"leadSponsor":127,"locationsCount":129},"100570532","personalized-training-for-people-with-rare-neuromuscular-disorders-100570532","NCT06708468","Personalized Training for People With Rare Neuromuscular Disorders","Personalized Exercise Training for People With Rare Neuromuscular Disorders: a Multi-center, Evaluator-blinded, Two Arm, Randomized Controlled Study to Assess the Effects on Physical Function From Personalized Strength and Balance Exercise in a Rehabilitation Setting.","PETRA-NMD","Inclusion Criteria:\n\n* A confirmed diagnosis of either FSHD, DM1 or CMT\n* 18-70 years of age at the time of signing the informed consent.\n* Any gender\n* Ability to stand, rise from a chair and walk at least 10 meters with or without any need of assistive devices\n* Indication for rehabilitation as confirmed by the treating neurologist or physiotherapist\n* Ability to understand and follow instructions in Norwegian\n* Capable of giving signed informed consent\n\nExclusion Criteria:\n\n* Pregnancy or planning to become pregnant\n* Any other neurological or non-neurological disorders affecting physical capacity, such as disabling arthritis, severe heart-failure\u002Fcardiomyopathy, on-going cancer treatment\n* Alcohol or drug abuse as per their medical chart\n* History of non-compliance to medical advice\u002Ffollow-up",{"count":102,"type":21},120,[104],"NA","The goal of this study is to investigate the effects of personalized exercise treatment on dynamic balance and physical function in comparison with regular follow-up in adults with rare-neuromuscular disorders: Charcot-Marie-Tooth (CMT), Facioscapulohumeral Muscular Dystrophy (FSHD), and Myotonic Dystrophy Type 1 (DM1).\n\nThe key objectives are:\n\n1. To investigate if the intervention group experiences improvements in dynamic balance that are superior to the control group\n2. To investigate if the intervention group experiences long-term improvements in dynamic balance that are superior to the control group during the follow-up\n3. To investigate if improvements in dynamic balance are associated with improvements in physical activity, body composition, estimated motor units, metabolomics, muscle echnogenecity and volume, and other indicators of health and quality of life.\n\nThis is a national study and will involve 120 individuals with rare-neuromuscular disorders from Norway's four health regions.",[107,108,109,71],"Neuromuscular Diseases (NMD)","Charcot Marie Tooth Disease (CMT)","Facioscapulohumeral Muscular Dystrophy",[111,112,113,114,115,116,117,118,119],"personalized training","rehabilitation","rare-neuromuscular disorders","motor unit number estimation","neuromuscular ultrasound","dual-energy x-ray absorptiometry","activity tracking","metabolomics","dynamic balance","2026-06-17",{"date":122,"type":47},"2026-06-18",{"date":124,"type":47},"2024-12-13",{"date":126,"type":21},"2028-12",{"name":128,"class":54},"Oslo University Hospital",5,{"id":131,"slug":132,"hasResults":11,"nctId":133,"briefTitle":134,"officialTitle":135,"acronym":136,"eligibilityCriteria":137,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":138,"enrollmentInfo":139,"targetDuration":4,"studyType":66,"phases":141,"briefSummary":144,"conditions":145,"keywords":146,"overallStatus":43,"whyStopped":4,"lastUpdateSubmitDate":165,"lastUpdatePostDateStruct":166,"startDateStruct":168,"completionDateStruct":170,"leadSponsor":172,"locationsCount":174},"100530360","phase-1-a-phase-12-study-of-vx-670-in-adult-participants-with-myotonic-dystrophy-1-dm1-100530360","NCT06185764","A Phase 1\u002F2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)","A Phase 1\u002F2, Randomized, Double-blind, Placebo-controlled Single- and Multiple-dose Escalation Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VX-670 in Adult Subjects With Myotonic Dystrophy Type 1","Galileo","Key Inclusion Criteria:\n\n\\- Documented clinical diagnosis of DM1 with age of onset greater than (\\>) 1 year of age and documented positive genetic test for DM1 in the subject with cytosine thymine guanine (CTG) repeat of at least 100\n\nKey Exclusion Criteria:\n\n\\- History of any illness or any clinical condition as pre-specified in the protocol\n\nOther protocol defined Inclusion\u002FExclusion criteria may apply.","64 Years",{"count":140,"type":21},52,[142,143],"PHASE1","PHASE2","The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in participants with DM1.",[71],[26,147,28,148,71,27,149,40,75,76,72,150,151,152,153,154,155,32,156,157,158,159,160,161,162,163,164],"DM2","Myotonic Dystrophy 2","DM","Vertex","Entrada","VX-670","VX670","PMO","ASO","Muscular Disorders, Atrophic","Muscular Diseases","Musculoskeletal Diseases","Neuromuscular Diseases","Nervous System Diseases","Genetic Diseases, Inborn","Heredodegenerative Disorders, Nervous System","Neurodegenerative Diseases","Muscular Dystrophies","2026-06-05",{"date":167,"type":47},"2026-06-08",{"date":169,"type":47},"2024-02-20",{"date":171,"type":21},"2027-02-02",{"name":173,"class":91},"Vertex Pharmaceuticals Incorporated",26,{"id":176,"slug":177,"hasResults":11,"nctId":178,"briefTitle":179,"officialTitle":180,"acronym":181,"eligibilityCriteria":182,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":183,"enrollmentInfo":184,"targetDuration":4,"studyType":66,"phases":186,"briefSummary":187,"conditions":188,"keywords":4,"overallStatus":43,"whyStopped":4,"lastUpdateSubmitDate":189,"lastUpdatePostDateStruct":190,"startDateStruct":192,"completionDateStruct":194,"leadSponsor":196,"locationsCount":197},"100476280","phase-1-safety-tolerability-pharmacodynamic-efficacy-and-pharmacokinetic-study-of-dyne-101-in-participants-with-myotonic-dystrophy-type-1-100476280","NCT05481879","Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1","A Randomized, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-101 Administered to Participants With Myotonic Dystrophy Type 1","ACHIEVE","Inclusion Criteria:\n\n* Diagnosis of DM1 with trinucleotide repeat size \\>100.\n* Age of onset of DM1 muscle symptoms ≥12 years.\n* Clinically apparent myotonia equivalent to hand opening time of at least 2 seconds in the opinion of the Investigator.\n* Hand grip strength and ankle dorsiflexion strength.\n* Able to complete 10-MWRT, stair ascend\u002Fdescend (MAD cohorts only), and 5×STS at screening without the use of assistive devices such as canes, walkers, or orthoses.\n\nExclusion Criteria:\n\n* History of major surgical procedure within 12 weeks prior to the start of investigative product administration or an expectation of a major surgical procedure (eg, implantation of cardiac defibrillator) during the study.\n* History of anaphylaxis.\n* Medical condition other than DM1 that would significantly impact ambulation or participation in functional assessments.\n* Treatment with medications that can improve myotonia within a period of 5 half-lives of the medication prior to performing screening assessments.\n* Electrocardiogram (ECG) with the corrected QT interval by Fridericia's Formula (QTcF) ≥450 milliseconds (ms) in men and QTcF ≥460 ms in women, PR ≥240 ms, left bundle-branch block, or a conduction defect, which is clinically significant in the opinion of the Investigator.\n* Percent predicted forced vital capacity (FVC) \\\u003C50%.\n* History of tibialis anterior biopsy within 3 months of Day 1 or planning to undergo tibialis anterior biopsies during study period for reasons unrelated to the study.\n* Participant has a history of suicide attempt, suicidal behavior, or has any suicidal ideation within 6 months prior to Screening that meets criteria at a level of 4 or 5 of the Columbia Suicide Severity Rating Scale (C-SSRS) or who, in the opinion of the Investigator, is at significant risk to commit suicide.\n* Use of glucagon-like peptide 1 (GLP-1) agonist medications including semaglutide, dulaglutide, liraglutide, exenatide, or tirzepatide within a period of 5 half-lives of the medication prior to performing screening assessments.\n* Significant weight loss during study participation may impact weight-based dosing, performance on muscle function assessments, and pharmacodynamic (PD) biomarkers.\n\nNote: Other inclusion and exclusion criteria may apply.","65 Years",{"count":185,"type":21},116,[142,143],"The primary purpose of the study is to evaluate the safety and tolerability of multiple intravenous (IV) doses of DYNE-101 administered to participants with Myotonic Dystrophy Type 1 (DM1).\n\nThe study consists of 4 periods: A Screening Period (up to 8 weeks), a Placebo-Controlled Period (24 weeks), a Treatment Period (24 weeks) and a Long-Term Extension (LTE) Period (168 weeks) in both multiple-ascending dose (MAD) and dose expansion cohorts.",[71],"2026-05-08",{"date":191,"type":47},"2026-05-12",{"date":193,"type":47},"2022-09-05",{"date":195,"type":21},"2029-07",{"name":78,"class":91},20,{"id":199,"slug":200,"hasResults":11,"nctId":201,"briefTitle":202,"officialTitle":203,"acronym":204,"eligibilityCriteria":205,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":206,"enrollmentInfo":207,"targetDuration":4,"studyType":23,"phases":4,"briefSummary":208,"conditions":209,"keywords":4,"overallStatus":43,"whyStopped":4,"lastUpdateSubmitDate":210,"lastUpdatePostDateStruct":211,"startDateStruct":213,"completionDateStruct":215,"leadSponsor":217,"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":212,"type":47},"2026-04-01",{"date":214,"type":47},"2022-05-10",{"date":216,"type":21},"2030-01-01",{"name":218,"class":54},"University of Rochester",{"id":220,"slug":221,"hasResults":11,"nctId":222,"briefTitle":223,"officialTitle":223,"acronym":4,"eligibilityCriteria":224,"healthyVolunteers":11,"sex":16,"minAge":225,"maxAge":17,"enrollmentInfo":226,"targetDuration":4,"studyType":66,"phases":228,"briefSummary":229,"conditions":230,"keywords":232,"overallStatus":43,"whyStopped":4,"lastUpdateSubmitDate":236,"lastUpdatePostDateStruct":237,"startDateStruct":239,"completionDateStruct":241,"leadSponsor":242,"locationsCount":55},"100578265","music-intervention-for-brain-heart-disease-in-myotonic-dystrophy-type-1-dm1-100578265","NCT06809049","Music Intervention for Brain-Heart Disease in Myotonic Dystrophy Type 1 (DM1)","Inclusion Criteria:\n\n* Participants between the ages of 6 to 18 with genetically confirmed congenital or infantile-onset myotonic dystrophy type 1 (DM1).\n* Participants willing to stay on stable medication from the day of screening to end of study.\n\nExclusion Criteria:\n\n* Insufficient English language skills to complete required assessments and questionnaires.\n* Participant is non-verbal.\n* Failure to provide signed informed consent by participant or designated decision maker (i.e. parent, legal guardian or power of attorney).\n* Failure to provide signed informed consent by parents\u002Fcaregivers for dyad participation.\n* Participant is not a resident of Canada, due to risk of attrition.\n* Patients for whom - in the opinion of the investigator - it would not be safe to participate in the study.","6 Years",{"count":227,"type":21},13,[104],"The goal of this interventional study is to demonstrate the feasibility and tolerability of music and movement intervention for children with congenital DM1, while providing indications of its effectiveness in improving brain and heart symptoms of DM1. Additionally, information from the collection of biological samples and wearable devices (accelerometer, EEG headband and ECG chest strap) will be used to identify brain-heart biomarkers and outcome measures for use in future research and trials.\n\nResearchers will compare the results of physical and cognitive assessments for each participant to assessments from baseline after 10 weeks of weekly music sessions. Qualitative measures (questionnaires and focus groups) will inform the feasibility of this intervention for this population. The main questions this study aims to answer are:\n\n* Are weekly music education sessions feasible for children with DM1?\n* Are weekly music education sessions tolerable for children with DM1?\n\nParticipants will:\n\n* Attend 45-minute-long music sessions once weekly for 10 weeks.\n* Attend two clinic visits for cognitive and physical assessments.\n* Provide blood, saliva, stool and urine samples.\n* Use wearable devices both at-home and during music sessions.\n* Parents\u002Fcaregivers of participants will complete questionnaires and participate in three focus groups.\n\nProgression from feasibility study to a full-scale clinical trial will be informed by four progression criteria:\n\n1. The feasibility of attendance, as assessed by attendance rate to 10 music sessions (≥ 60%)\n2. Feasibility of attendance, as rated by parents\u002Fcaregivers of participants (≥60% rate \"extremely\" or \"very\" practical to attend)\n3. Attrition rate of the study, as determined by percentage of participants who complete the study (≥ 60%)\n4. Overall satisfaction, as rated by parents\u002Fcaregivers of participants (≥60% rate \"very satisfied\" or \"satisfied\")",[231,31,29,71],"Myotonic Dystrophy, Congenital",[233,234,26,235],"Music intervention","Myotonic dystrophy","Rare disease","2026-02-17",{"date":238,"type":47},"2026-02-19",{"date":240,"type":47},"2025-03-25",{"date":49,"type":21},{"name":243,"class":54},"Hanns Lochmuller"]