[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"congenital-myotonic-dystrophy\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:congenital-myotonic-dystrophy":25},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,6,0,[8,43,82,104,131,162],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":4,"briefSummary":23,"conditions":24,"keywords":27,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":31,"lastUpdatePostDateStruct":32,"startDateStruct":35,"completionDateStruct":37,"leadSponsor":39,"locationsCount":42},"100456529","dmcrn-02-001-assessing-pediatric-endpoints-in-dm1-100456529",false,"NCT05224778","DMCRN-02-001: Assessing Pediatric Endpoints in DM1","Assessing Pediatric Endpoints in DM1 (ASPIRE-DM1)","ASPIRE-DM1","Inclusion Criteria:\n\n* Age neonate to 3 years 11 months at enrollment.\n* 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 more than 72 hours; and a genetic test confirming an expanded trinucleotide (CTG) repeat in the DMPK gene in the child or mother. 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* Guardian is willing and able to sign consent and follow study procedures\n\nExclusion Criteria:\n\n* Any other non-DM1 illness that would interfere with the ability or results of the study in the opinion of the site investigator\n* Significant trauma within one month\n* Internal metal or devices (exclusion for DEXA component)\n* History of bleeding disorder or platelet count \\\u003C50,000\n* History of reaction to local anesthetic","ALL","59 Months",{"count":20,"type":21},50,"ESTIMATED","OBSERVATIONAL","The overall goal of the study is to establish valid clinical endpoint assessments for children with congenital myotonic dystrophy type 1 and develop biomarkers for the condition.",[25,26],"Congenital Myotonic Dystrophy","CDM",[28,29,25,26],"Clinical Research","Myotonic dystrophy","RECRUITING","2026-06-08",{"date":33,"type":34},"2026-06-10","ACTUAL",{"date":36,"type":34},"2022-08-24",{"date":38,"type":21},"2026-12",{"name":40,"class":41},"Virginia Commonwealth University","OTHER",5,{"id":44,"slug":45,"hasResults":11,"nctId":46,"briefTitle":47,"officialTitle":48,"acronym":4,"eligibilityCriteria":49,"healthyVolunteers":50,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":51,"targetDuration":4,"studyType":22,"phases":4,"briefSummary":53,"conditions":54,"keywords":63,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":72,"lastUpdatePostDateStruct":73,"startDateStruct":75,"completionDateStruct":77,"leadSponsor":79,"locationsCount":81},"100063959","myotonic-dystrophy-and-facioscapulohumeral-muscular-dystrophy-registry-100063959","NCT00082108","Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Registry","National Registry of Myotonic Dystrophy and Facioscapulohumeral Muscular Dystrophy Patients and Family Members","Inclusion Criteria:\n\n* Diagnosed with DM, FSHD, or related diseases or are an unaffected family member of someone diagnosed with one of these diseases",true,{"count":52,"type":21},3000,"Myotonic dystrophy (DM) and facioscapulohumeral muscular dystrophy (FSHD) are inherited disorders characterized by progressive muscle weakness and loss of muscle tissue. The purpose of this registry is to connect people with DM or FSHD with researchers studying these diseases. The registry will offer individuals with DM and FSHD an opportunity to participate in research that focuses of their diseases. The registry will also help scientists to accomplish research on DM and FSHD and to distribute their findings to patients and care providers.",[55,56,57,58,59,25,60,61,62],"Myotonic Dystrophy","Facioscapulohumeral Muscular Dystrophy","Muscular Dystrophy","Myotonic Dystrophy Type 1","Myotonic Dystrophy Type 2","PROMM (Proximal Myotonic Myopathy)","Steinert's Disease","Myotonic Muscular Dystrophy",[64,65,66,57,67,55,68,69,70,71],"Registry","FSHD","DM","Facioscapulohumeral Myotonic Dystrophy","DM1","DM2","FSH","MMD","2025-10-10",{"date":74,"type":34},"2025-10-15",{"date":76,"type":4},"2000-09",{"date":78,"type":21},"2028-06",{"name":80,"class":41},"University of Rochester",1,{"id":83,"slug":84,"hasResults":11,"nctId":85,"briefTitle":86,"officialTitle":4,"acronym":4,"eligibilityCriteria":4,"healthyVolunteers":11,"sex":4,"minAge":4,"maxAge":4,"enrollmentInfo":4,"targetDuration":4,"studyType":87,"phases":4,"briefSummary":88,"conditions":89,"keywords":90,"overallStatus":97,"whyStopped":4,"lastUpdateSubmitDate":98,"lastUpdatePostDateStruct":99,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":101,"locationsCount":4},"100602151","tideglusib-expanded-access-use-in-congenital-myotonic-dystrophy-100602151","NCT07119775","Tideglusib: Expanded Access Use in Congenital Myotonic Dystrophy","EXPANDED_ACCESS","This treatment plan is limited to a single patient with Congenital Myotonic Dystrophy, who is ineligible or otherwise unable to participate in ongoing clinical trials.",[25],[91,25,55,92,93,94,95,96,61],"Tideglusib","Dystrophia Myotonica","Myotonia Atrophica","Myotonia Dystrophica","Myotonic Dystrophy, Congenital Steinert Disease","Steinert Myotonic Dystrophy","AVAILABLE","2025-08-06",{"date":100,"type":34},"2025-08-13",{"name":102,"class":103},"AMO Pharma Limited","INDUSTRY",{"id":105,"slug":106,"hasResults":11,"nctId":107,"briefTitle":108,"officialTitle":108,"acronym":109,"eligibilityCriteria":110,"healthyVolunteers":11,"sex":17,"minAge":111,"maxAge":112,"enrollmentInfo":113,"targetDuration":4,"studyType":22,"phases":4,"briefSummary":115,"conditions":116,"keywords":118,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":123,"lastUpdatePostDateStruct":124,"startDateStruct":126,"completionDateStruct":128,"leadSponsor":130,"locationsCount":81},"100573561","trial-readiness-and-endpoint-assessment-in-pediatric-myotonic-dystrophy-extension-100573561","NCT06747884","Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension","TREAT-EXT","Inclusion Criteria (Congenital Myotonic Dystrophy Group):\n\n* Age 5-17 years, 11 months at enrollment. Lower age limit not applicable for participants who have completed ASPIRE-DM1 protocol. Upper age limit not applicable for participants who previously participated in TREAT-01-001 (TREAT-CDM) study\n* A diagnosis of CDM, 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 more than 72 hours; and a genetic test confirming an expanded trinucleotide (CTG) repeat in the DMPK gene in the child or mother. 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* Written, voluntary informed consent must be obtained before any study related procedures are conducted.\n\nInclusion Criteria (Childhood Myotonic Dystrophy Group):\n\n* Age 3-17 years, 11 months at enrollment. Upper age limit not applicable for participants who previously participated in TREAT-01-001 (TREAT-CDM) study.\n* A diagnosis of ChDM, defined as: children having cognitive deficits, muscle weakness, myotonia that developed after age 1 and prior to age 10 and a genetic test confirming an expanded trinucleotide (CTG) repeat in the DMPK gene in the child or mother. 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* Written, voluntary informed consent must be obtained before any study related procedures are conducted.\n\nExclusion Criteria:\n\n* Any other non-DM1 illness that would interfere with the ability to undergo safe testing or would affect the interpretation of the results, in the opinion of the site investigator\n* Significant trauma within the past month\n* Internal metal or devices (exclusion for DEXA component)\n* Use of anticoagulants, such as warfarin or a direct oral anticoagulant (e.g., dabigatran) due to the increased risk of bleeding with biopsy\n* Platelet count \\\u003C50,000\n* History of a bleeding disorder\n* Participation in a clinical trial involving an investigational product\n* History of adverse reaction to lidocaine (if participating in muscle biopsy)","3 Years","17 Years",{"count":114,"type":21},200,"This is a natural history study to improve the types of assessments and biological samples that will be used in clinical drug trials in both congenital myotonic dystrophy and childhood myotonic dystrophy.",[25,117,55],"Childhood Myotonic Dystrophy",[68,117,25,55,119,120,121,109,122],"Myotonia","Dystrophy Myotonic","DMCRN","TREAT CDM","2025-07-28",{"date":125,"type":34},"2025-07-30",{"date":127,"type":34},"2025-06-06",{"date":129,"type":21},"2030-06",{"name":40,"class":41},{"id":132,"slug":133,"hasResults":11,"nctId":134,"briefTitle":135,"officialTitle":136,"acronym":137,"eligibilityCriteria":138,"healthyVolunteers":11,"sex":17,"minAge":139,"maxAge":140,"enrollmentInfo":141,"targetDuration":4,"studyType":143,"phases":144,"briefSummary":147,"conditions":148,"keywords":149,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":153,"lastUpdatePostDateStruct":154,"startDateStruct":156,"completionDateStruct":158,"leadSponsor":160,"locationsCount":161},"100439579","phase-2-safety-and-efficacy-of-tideglusib-in-congenital-or-childhood-onset-myotonic-dystrophy-100439579","NCT05004129","Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy","An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Tideglusib for the Treatment of Congenital or Childhood Onset DM1 (REACH CDM X)","REACH CDM X","Inclusion Criteria:\n\nSubjects who do not enter this study directly from completing the AMO-02-MD-2-003 study (i.e. subjects who did not complete AMO-02-MD-2-003, subjects who completed AMO-02-MD-2-003 but did not directly rollover or subjects who are re-entering AMO-02-MD-2-004), will not be considered eligible for the study without meeting all of the criteria below:\n\n1. Subjects under study must be individuals with a diagnosis of Congenital or Childhood Onset DM1.\n2. Diagnosis must be genetically confirmed\n3. Subjects must be male or female aged ≥6 years to ≤45 years at Screening\n4. Subjects must have a Clinical Global Impression - Severity (CGI-S) score of 3 or greater at Screening (V-1)\n5. Written, voluntary informed consent must be obtained before any study related procedures are conducted. Where a parent or legally authorized representative (LAR) provides consent, there must also be assent from the subject (as required by local regulations)\n6. Subject's caregiver must be willing and able to support participation for duration of study\n7. Subject must be willing and able to comply with the required food intake restrictions as outlined per protocol\n\nSubjects entering directly from completing the antecedent AMO-02-MD-2-003 study will not be considered eligible for the study without meeting all of the criteria below:\n\n1. Subjects who have completed the antecedent AMO-02-MD-2-003 study through V11\n2. Written, voluntary informed consent must be obtained before any study related procedures are conducted. Where a parent or LAR provides consent, there must also be assent from the subject (as required by local regulations)\n3. Subject's caregiver must be willing and able to support participation for duration of study\n4. Subject must be willing and able to comply with the required food intake restrictions as outlined per protocol\n\nKey Exclusion Criteria:\n\n1. Body mass index (BMI) less than 13.5 kg\u002Fm² or greater than 40 kg\u002Fm²\n2. New or change in medications\u002Ftherapies within 4 weeks prior to Eligibility\u002FBaseline Visit\n3. Use within 4 weeks prior to Eligibility\u002FBaseline Visit of strong CYP3A4 inhibitors (eg.clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole, nefazodone, idinavir and ritonavir)\n4. Concurrent use of drugs metabolized by CYP3A4 with a narrow therapeutic window (e.g. warfarin and digitoxin)\n5. Current enrollment in a clinical trial of an investigational drug or enrollment in a clinical trial of an investigational drug in the last 6 months other than the AMO-02- MD-2-003 study\n6. Existing or historical medical conditions or complications (eg. neurological, cardiovascular, renal, hepatic, gastrointestinal, endocrine or respiratory disease) that may impact the interpretability of the study results\n7. Hypersensitivity to tideglusib or any components of its formulation including allergy to strawberry","6 Years","45 Years",{"count":142,"type":21},76,"INTERVENTIONAL",[145,146],"PHASE2","PHASE3","This is an open-label phase 2\u002F3 study for individuals with Congenital Myotonic Dystrophy (Congenital DM1) who participated in the preceding AMO-02-MD-2-003 study or individuals with either Congenital or Childhood Onset DM1 who are treatment naïve.",[25],[91,150,25,55,92,93,94,151,152,96,61],"AMO-02-MD-2-004","Myotonic Dystrophy, Congenital","Steinert Disease","2025-05-22",{"date":155,"type":34},"2025-05-28",{"date":157,"type":34},"2021-08-23",{"date":159,"type":21},"2026-12-31",{"name":102,"class":103},14,{"id":163,"slug":164,"hasResults":11,"nctId":165,"briefTitle":166,"officialTitle":166,"acronym":167,"eligibilityCriteria":168,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":169,"targetDuration":171,"studyType":22,"phases":4,"briefSummary":172,"conditions":173,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":180,"lastUpdatePostDateStruct":181,"startDateStruct":183,"completionDateStruct":185,"leadSponsor":187,"locationsCount":81},"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":170,"type":21},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.",[55,25,174,175,92,176,177,94,151,178,60,179,152,96,61,93],"Myotonic Dystrophy 1","Myotonic Dystrophy 2","Dystrophia Myotonica 1","Dystrophia Myotonica 2","Myotonic Myopathy, Proximal","Proximal Myotonic Myopathy","2024-11-19",{"date":182,"type":34},"2024-11-21",{"date":184,"type":4},"2013-02",{"date":186,"type":21},"2030-02",{"name":188,"class":41},"Myotonic Dystrophy Foundation"]