[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"myotonic-dystrophy-congenital\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:myotonic-dystrophy-congenital":26},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,48,75],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":4,"eligibilityCriteria":14,"healthyVolunteers":11,"sex":15,"minAge":16,"maxAge":17,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":24,"conditions":25,"keywords":30,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":36,"lastUpdatePostDateStruct":37,"startDateStruct":40,"completionDateStruct":42,"leadSponsor":44,"locationsCount":47},"100578265","music-intervention-for-brain-heart-disease-in-myotonic-dystrophy-type-1-dm1-100578265",false,"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.","ALL","6 Years","18 Years",{"count":19,"type":20},13,"ESTIMATED","INTERVENTIONAL",[23],"NA","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\")",[26,27,28,29],"Myotonic Dystrophy, Congenital","Myotonic Dystrophy, Type 1 (DM1)","Myotonic Dystrophy Type 1","Myotonic Dystrophy Type 1 (DM1)",[31,32,33,34],"Music intervention","Myotonic dystrophy","DM1","Rare disease","RECRUITING","2026-02-17",{"date":38,"type":39},"2026-02-19","ACTUAL",{"date":41,"type":39},"2025-03-25",{"date":43,"type":20},"2026-07",{"name":45,"class":46},"Hanns Lochmuller","OTHER",1,{"id":49,"slug":50,"hasResults":11,"nctId":51,"briefTitle":52,"officialTitle":53,"acronym":54,"eligibilityCriteria":55,"healthyVolunteers":56,"sex":15,"minAge":4,"maxAge":4,"enrollmentInfo":57,"targetDuration":59,"studyType":60,"phases":4,"briefSummary":61,"conditions":62,"keywords":4,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":65,"lastUpdatePostDateStruct":66,"startDateStruct":68,"completionDateStruct":70,"leadSponsor":72,"locationsCount":74},"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":58,"type":20},3000,"10 Years","OBSERVATIONAL","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.",[63,33,28,26,64],"Myotonic Dystrophy 1","Steinert Disease","2026-01-29",{"date":67,"type":39},"2026-02-04",{"date":69,"type":39},"2025-06-02",{"date":71,"type":20},"2026-12-31",{"name":73,"class":46},"Fundació Institut Germans Trias i Pujol",8,{"id":76,"slug":77,"hasResults":11,"nctId":78,"briefTitle":79,"officialTitle":79,"acronym":80,"eligibilityCriteria":81,"healthyVolunteers":11,"sex":15,"minAge":4,"maxAge":4,"enrollmentInfo":82,"targetDuration":84,"studyType":60,"phases":4,"briefSummary":85,"conditions":86,"keywords":4,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":100,"lastUpdatePostDateStruct":101,"startDateStruct":103,"completionDateStruct":105,"leadSponsor":107,"locationsCount":47},"100239721","myotonic-dystrophy-family-registry-100239721","NCT02398786","Myotonic Dystrophy Family Registry","MDFR","Inclusion Criteria:\n\n* Diagnosed with congenital, juvenile-onset or adult onset DM1 or DM2 (confirmed by clinical exam or genetic test)\n\nExclusion Criteria:\n\n* Not diagnosed with DM, unaffected family members",{"count":83,"type":20},3500,"5 Years","The Myotonic Dystrophy Family Registry (MDFR) is an online, patient-entered database that collects information on myotonic dystrophy (DM) to aid researchers in developing new, effective treatments and help identify participants for research studies and clinical trials.",[87,88,63,89,90,91,92,93,26,94,95,96,64,97,98,99],"Myotonic Dystrophy","Congenital Myotonic Dystrophy","Myotonic Dystrophy 2","Dystrophia Myotonica","Dystrophia Myotonica 1","Dystrophia Myotonica 2","Myotonia Dystrophica","Myotonic Myopathy, Proximal","PROMM (Proximal Myotonic Myopathy)","Proximal Myotonic Myopathy","Steinert Myotonic Dystrophy","Steinert's Disease","Myotonia Atrophica","2024-11-19",{"date":102,"type":39},"2024-11-21",{"date":104,"type":4},"2013-02",{"date":106,"type":20},"2030-02",{"name":108,"class":46},"Myotonic Dystrophy Foundation"]