[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"myotonic-dystrophy-1\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:myotonic-dystrophy-1":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,12,0,[8,56,79,111,143,167,192,215,241,262,294,320],{"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":17,"maxAge":18,"enrollmentInfo":63,"targetDuration":4,"studyType":23,"phases":4,"briefSummary":65,"conditions":66,"keywords":67,"overallStatus":43,"whyStopped":4,"lastUpdateSubmitDate":70,"lastUpdatePostDateStruct":71,"startDateStruct":73,"completionDateStruct":75,"leadSponsor":77,"locationsCount":78},"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":64,"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],[27,68,69,41],"END DM-1","Muscular Dystophy","2026-06-08",{"date":72,"type":47},"2026-06-10",{"date":74,"type":47},"2019-01-01",{"date":76,"type":21},"2026-12-01",{"name":53,"class":54},17,{"id":80,"slug":81,"hasResults":11,"nctId":82,"briefTitle":83,"officialTitle":84,"acronym":85,"eligibilityCriteria":86,"healthyVolunteers":11,"sex":16,"minAge":87,"maxAge":88,"enrollmentInfo":89,"targetDuration":4,"studyType":91,"phases":92,"briefSummary":94,"conditions":95,"keywords":96,"overallStatus":43,"whyStopped":4,"lastUpdateSubmitDate":100,"lastUpdatePostDateStruct":101,"startDateStruct":103,"completionDateStruct":105,"leadSponsor":107,"locationsCount":110},"100567381","phase-2-a-clinical-study-of-pgn-edodm1-in-people-with-myotonic-dystrophy-type-1-100567381","NCT06667453","A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1","A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study of PGN-EDODM1 in Adult Participants With Myotonic Dystrophy Type 1 (FREEDOM2-DM1)","FREEDOM2-DM1","Inclusion Criteria:\n\n* Confirmed diagnosis of DM1, as defined as having a repeat sequence in the DMPK gene with at least 100 CTG repeats\n* Presence of myotonia\n* Have sufficient muscle mass in bilateral tibialis anterior (TA) muscles that a needle biopsy can safely be performed\n* Body Mass Index (BMI) of \\\u003C 35.0 kg\u002Fm\\^2\n\nExclusion Criteria:\n\n* Congenital DM1\n* Known history or presence of any clinically significant conditions that may interfere with study safety assessments\n* Abnormal laboratory tests at screening considered clinically significant by the Investigator\n* Medications specific for the treatment of myotonia within 2 weeks prior to screening\n* Percent predicted forced vital capacity (FVC) \\\u003C40%\n* Use of an investigational drug, device, or product within 30 days of 5 half-lives of the study drug (whichever is longer) prior to Screening\n\nNote: Other inclusion and exclusion criteria may apply.","16 Years","65 Years",{"count":90,"type":21},24,"INTERVENTIONAL",[93],"PHASE2","The purpose of this study is to learn about the effects of an investigational medicine, PGN-EDODM1, to see how safe and tolerable multiple administrations of PGN-EDODM1 are for people with myotonic dystrophy type 1 (DM1) compared to placebo.",[28],[26,28,27,97,98,32,99],"PepGen","PGN-EDODM1","Steinert's Disease","2026-04-23",{"date":102,"type":47},"2026-04-28",{"date":104,"type":47},"2024-12-10",{"date":106,"type":21},"2027-03",{"name":108,"class":109},"PepGen Inc","INDUSTRY",8,{"id":112,"slug":113,"hasResults":11,"nctId":114,"briefTitle":115,"officialTitle":116,"acronym":4,"eligibilityCriteria":117,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":118,"targetDuration":4,"studyType":91,"phases":120,"briefSummary":121,"conditions":122,"keywords":123,"overallStatus":43,"whyStopped":4,"lastUpdateSubmitDate":134,"lastUpdatePostDateStruct":135,"startDateStruct":137,"completionDateStruct":139,"leadSponsor":141,"locationsCount":142},"100609901","phase-2-an-open-label-extension-study-of-pgn-edodm1-in-people-with-myotonic-dystrophy-type-1-freedom-ole-100609901","NCT07220603","An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE)","An Open-Label Extension Study Evaluating Safety and Pharmacokinetics in Participants With Myotonic Dystrophy Type 1 (FREEDOM-OLE)","Inclusion Criteria:\n\n* Participant has completed a prior study with PGN-EDODM1\n\nExclusion Criteria:\n\n* Abnormal laboratory tests at screening considered clinically significant by the Investigator\n* Use of an investigational drug (other than PGN-EDODM1), device, or product, within 30 days or 5 half-lives of the study drug (whichever is longer) prior to study entry",{"count":119,"type":21},48,[93],"The purpose of this study is to learn about the long-term safety and tolerability of PGN-EDODM1 in participants with myotonic dystrophy type 1 (DM1) who have completed a prior study with PGN-EDODM1.",[28],[26,28,27,97,98,32,124,125,126,127,128,129,130,131,132,133],"Steinhert's Disease","Myotonic Dystrophies","Genetic Diseases, Inborn","Neuromuscular Diseases","Nervous System Diseases","Musculoskeletal Diseases","Myotonic Disorders","Muscular Disorders, Atrophic","Heredodegenerative Disorders, Nervous System","Muscular Diseases","2026-03-26",{"date":136,"type":47},"2026-03-30",{"date":138,"type":47},"2025-12-23",{"date":140,"type":21},"2029-01",{"name":108,"class":109},3,{"id":144,"slug":145,"hasResults":11,"nctId":146,"briefTitle":147,"officialTitle":148,"acronym":149,"eligibilityCriteria":150,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":151,"enrollmentInfo":152,"targetDuration":4,"studyType":91,"phases":154,"briefSummary":156,"conditions":157,"keywords":4,"overallStatus":43,"whyStopped":4,"lastUpdateSubmitDate":158,"lastUpdatePostDateStruct":159,"startDateStruct":161,"completionDateStruct":163,"leadSponsor":165,"locationsCount":5},"100539166","phase-1-study-of-atx-01-in-participants-with-dm1-100539166","NCT06300307","Study of ATX-01 in Participants With DM1","A Phase 1\u002F2a Double-Blind, Placebo-controlled, Single- and Multiple Ascending Dose Study to Assess the Safety, Tolerability, PK, PD and Efficacy of IV Administration of ATX-01 In Male and Female Participants Aged 18 to 64 With Classic DM1","ArthemiR","Key Inclusion Criteria:\n\n* Participants with a documented clinical diagnosis of DM1 (CTG expansion of \\>150 repeats in DMPK gene measured in peripheral blood mononuclear cells)\n* Ambulatory, defined as able to complete a 10-meter walk\u002Frun test at screening without the use of assistive devices such as canes, walkers, or orthoses, except for ankle-foot orthoses\n* Presence for \\>3 seconds of grip myotonia as confirmed by a central reader\n\nKey Exclusion Criteria:\n\n* Participants with congenital DM1\n* Medical Research Council Muscle Scale score of less than 4 on ankle dorsiflexion or significant tibialis anterior atrophy that prevents a muscle biopsy\n* Use of mexiletine or other agent for myotonia within 21 days or 5 half-lives, whichever is longer, prior to screening","64 Years",{"count":153,"type":21},56,[155,93],"PHASE1","The goal of this clinical trial is to test ATX-01 in participants with myotonic dystrophy type 1 (DM1). The main question it aims to answer is if ATX-01 is safe and well tolerated. The trial will compare the safety and tolerability of ATX-01 and a matching placebo.\n\nThere will be a single-ascending dose part of the trial and a multiple-ascending dose part. In the single-ascending dose, participants will receive one dose of ATX-01 or placebo. In the multiple-ascending dose part, participants will receive three doses of ATX-01 or placebo.\n\nATX-01 is a novel anti-miR (synthetic single stranded oligonucleotide) that inhibits a microRNA called miR-23b.",[28],"2026-02-06",{"date":160,"type":47},"2026-02-10",{"date":162,"type":47},"2024-10-15",{"date":164,"type":21},"2027-07",{"name":166,"class":109},"ARTHEx Biotech S.L.",{"id":168,"slug":169,"hasResults":11,"nctId":170,"briefTitle":171,"officialTitle":172,"acronym":173,"eligibilityCriteria":174,"healthyVolunteers":175,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":176,"targetDuration":178,"studyType":23,"phases":4,"briefSummary":179,"conditions":180,"keywords":4,"overallStatus":43,"whyStopped":4,"lastUpdateSubmitDate":183,"lastUpdatePostDateStruct":184,"startDateStruct":186,"completionDateStruct":188,"leadSponsor":190,"locationsCount":110},"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":177,"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.",[28,26,29,181,182],"Myotonic Dystrophy, Congenital","Steinert Disease","2026-01-29",{"date":185,"type":47},"2026-02-04",{"date":187,"type":47},"2025-06-02",{"date":189,"type":21},"2026-12-31",{"name":191,"class":54},"Fundació Institut Germans Trias i Pujol",{"id":193,"slug":194,"hasResults":11,"nctId":195,"briefTitle":196,"officialTitle":197,"acronym":198,"eligibilityCriteria":199,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":88,"enrollmentInfo":200,"targetDuration":4,"studyType":91,"phases":202,"briefSummary":203,"conditions":204,"keywords":4,"overallStatus":205,"whyStopped":4,"lastUpdateSubmitDate":206,"lastUpdatePostDateStruct":207,"startDateStruct":209,"completionDateStruct":211,"leadSponsor":213,"locationsCount":4},"100598782","phase-1-calcium-channel-blocker-in-myotonic-dystrophy-type-1-100598782","NCT07075965","Calcium Channel Blocker in Myotonic Dystrophy Type 1","Calcium Channel Blocker in Myotonic Dystrophy Type 1 (CAP DM1)","CAP DM1","Inclusion Criteria:\n\n* Male or Female between the ages 18 and 65, inclusive.\n* A genetically confirmed diagnosis of DM1, having a repeat expansion in the DMPK gene with at least 100 CTG repeats.\n* Grip strength less than 50% predicted based on age, height, and sex.\n* Video hand opening time is 4 sec or greater for at least one hand.\n* Ambulatory and able to walk 10 meters.\n* Willingness to discontinue anti-myotonia drugs at least 2 weeks prior to screening.\n* Highly effective method of contraception in women with childbearing potential.\n\nExclusion Criteria:\n\n* Congenital DM1 as defined by symptom onset in the first 4 weeks of life.\n* Abnormal liver function tests (LFTs): alanine aminotransferase (ALT), or aspartate aminotransferase (AST) \\>3 x upper limit of normal. Total bilirubin \\> 1.5 mg\u002FdL, or INR \\> 1.3, or evidence of current active or chronic infection with hepatitis C, hepatitis B or other hepatobiliary conditions other than DM1 (or attributed to DM1) that cause abnormal liver laboratory parameters (e.g., hemochromatosis, Wilson's disease, autoimmune hepatitis)\n* Current or recent infection requiring antibiotic treatment within 2 weeks prior to screening.\n* Abnormal vital signs, including systolic blood pressure \\\u003C 90 mmHg and diastolic blood pressure \\\u003C 60 mmHg.\n* Treatment with concomitant medications with potential interactions with amlodipine, these may include but are not limited to sildenafil, cyclosporin, atorvastatin at high dosages (80 mg daily), simvastatin (at dosages higher than 20 mg daily) or strong inhibitors of CYP3A4.\n* A history of syncope.\n* A history of symptomatic hypotension.\n* Initiation or change in doses of concomitant medications, including herbal supplements, if in the opinion of the Investigator, may impact the results of the study.\n* Women of childbearing potential must have a negative pregnancy test, cannot be planning a pregnancy, and cannot be breastfeeding at any time during the study.\n* Known history of substance and\u002For alcohol abuse within one year prior to screening.\n* The presence of comorbidities that, in the opinion of the Investigator, may influence study results, including, but not limited to, uncontrolled diabetes, generalized or mononeuropathy of the upper extremities, or cervical radiculopathy resulting in weakness and atrophy.\n* Concurrent treatment with calcium channel blocker.\n* Known ischemic or non-ischemic moderate or severe heart failure as defined by symptoms suggestive of heart failure or reduced left ventricular ejection fraction (LVEF) on echocardiogram \\\u003C 55%.\n* Moderate or severe aortic stenosis or other obstruction of the left ventricle outflow tract.\n* Known sensitivity or allergy to amlodipine.",{"count":201,"type":21},20,[155],"This is a Phase 1 clinical trial designed to evaluate the safety and tolerability of amlodipine, a calcium channel blocker, in adults with Myotonic Dystrophy Type 1 (DM1). Amlodipine is being studied to see if it can improve muscle strength, reduce stiffness (myotonia), and improve function by modifying calcium flow in muscle cells. All participants will receive amlodipine starting at 2.5 mg daily for 2 weeks, then 5 mg for 4 weeks. After that, participants will be randomly assigned to continue on 5 mg or increase to 10 mg for an additional 4 weeks. The main goals are to assess changes in blood pressure and any adverse events to determine whether the drug is safe in this population. The study will also explore how amlodipine affects muscle strength, mobility, fatigue, and daily function using clinical tests and questionnaires. Findings will inform a future phase 2 trial.",[28],"NOT_YET_RECRUITING","2025-12-03",{"date":208,"type":47},"2025-12-09",{"date":210,"type":21},"2026-12-07",{"date":212,"type":21},"2030-10-01",{"name":214,"class":54},"University of Rochester",{"id":216,"slug":217,"hasResults":11,"nctId":218,"briefTitle":219,"officialTitle":220,"acronym":221,"eligibilityCriteria":222,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":223,"targetDuration":4,"studyType":91,"phases":225,"briefSummary":227,"conditions":228,"keywords":230,"overallStatus":43,"whyStopped":4,"lastUpdateSubmitDate":232,"lastUpdatePostDateStruct":233,"startDateStruct":235,"completionDateStruct":237,"leadSponsor":239,"locationsCount":55},"100480194","phase-3-evaluation-of-the-efficacy-and-safety-of-metformin-in-the-myotonic-dystrophy-type-1-steinerts-disease-100480194","NCT05532813","Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease)","Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease). A Phase III, Prospective, Multicentre, Randomized, Double-blind Controlled Study","METFORMYO","Inclusion Criteria:\n\n* DM1 disease confirmed by genetic analysis\n* Men and women between 18 and 70 years of age.\n* Preserved walking abilities (stick assistance possible)\n* MIRS score 3 or 4\n* Women of childbearing potential under efficient contraception during treatment\n* Patient able to consent\n* All patients who have completed and signed the specific information and informed consent form\n* Affiliation to a social security system\n\nExclusion Criteria:\n\n* Pregnant or breast-feeding women\n* Men with an intention to conceive a child during the time of the study\n* Contraindications to Metformin (hypersensitivity to metformin or to one of the excipients)\n* Respiratory:\n\n  * Patient requiring tracheotomy or\n  * Patient requiring non-invasive-ventilation: - more than 12 hours per day; - insufficiently ventilated\n* Creatinine clearance inferior to 50 ml\u002Fmin\n* Cardiac:\n\n  * Left ventricular ejection fraction below 35%\n  * Conduction system disease on the electrocardiogram with PR interval \\>200 ms or QRS duration \\>110 ms without a pacemaker or an implantable defibrillator or cardiac electrophysiological study performed over the past 5 years\n  * Third-degree or Second degree type II atrioventricular block without a pacemaker or an implantable defibrillator\n  * Sustained ventricular tachycardia\n* Acute disease that may lead to tissue hypoxia",{"count":224,"type":21},142,[226],"PHASE3","The study team hypothesize that non-diabetic patients with Myotonic dystrophy type I (DM1) will improve their symptoms, especially their motor deficit which is the main feature of the disease, because of the splicing defect correction by metformin.\n\nThe primary objective of the study is to evaluate the efficacy of metformin vs placebo, on the improvement of muscle function in patients with DM1 compared to its placebo.\n\nAs the secondary objectives, the study aims:\n\n* To evaluate the safety of metformin on patient with DM1.\n* To evaluate the efficacy of metformin vs placebo on:\n\n  1. The hand-grip strength;\n  2. The thumb-index pinch strength;\n  3. The locomotor function;\n  4. The respiratory function;\n  5. The cardiac function;\n  6. The quality of life;\n  7. The daily and social activity.",[35,28,229],"Metformin",[35,28,229,231],"Muscle function","2025-11-19",{"date":234,"type":47},"2025-11-24",{"date":236,"type":47},"2024-11-29",{"date":238,"type":21},"2026-12",{"name":240,"class":54},"Assistance Publique - Hôpitaux de Paris",{"id":242,"slug":243,"hasResults":11,"nctId":244,"briefTitle":245,"officialTitle":246,"acronym":4,"eligibilityCriteria":247,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":88,"enrollmentInfo":248,"targetDuration":4,"studyType":91,"phases":250,"briefSummary":251,"conditions":252,"keywords":4,"overallStatus":43,"whyStopped":4,"lastUpdateSubmitDate":253,"lastUpdatePostDateStruct":254,"startDateStruct":256,"completionDateStruct":258,"leadSponsor":259,"locationsCount":261},"100526745","phase-1-study-of-aro-dm1-in-subjects-with-type-1-myotonic-dystrophy-100526745","NCT06138743","Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy","A Phase 1\u002F2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy Who Are ≥18 to ≤ 65 Years","Inclusion Criteria:\n\n* Genetically confirmed diagnosis of DM1\n* Clinician-assessed signs of DM1 including clinically apparent myotonia\n* Onset of DM1 symptoms occurred after the age of 12 years\n* Walk for at least 10 meters independently at Screening\n* Subjects of childbearing potential must agree to use highly effective contraception in addition to a condom during the study and for at least 90 days following the end of study or last dose of study drug, whichever is later. Subjects must not donate sperm or eggs during the study and for at least 90 days following the end of study or last dose of study drug whichever is later.\n\nExclusion Criteria:\n\n* Inadequately controlled diabetes\n* Confirmed diagnosis of congenital DM1\n* Uncontrolled hypertension\n* History of tibialis anterior (TA) biopsy within 3 months of Day 1 or planning to undergo TA biopsies during the study period\n* Clinically significant cardiac, liver or renal disease\n* HIV infection (seropositive) at Screening\n* Seropositive for hepatitis B (HBV) or hepatitis C (HCV) at screening\n* Untreated or poorly controlled epilepsy\n* Treatment with anti-myotonia medication within a period of 5 half-lives of the medication prior to Screening.\n* Abnormal coagulation parameters at Screening including platelet count, international normalized ratio (INR), prothrombin time, and activated partial thromboplastin time (APTT)\n\nNote: Additional inclusion\u002Fexclusion criteria may apply per protocol",{"count":249,"type":21},78,[155,93],"This is a phase 1\u002F2a double-blinded, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single and multiple ascending doses of ARO-DM1 compared to placebo in male and female subjects with type 1 myotonic dystrophy (DM1). Participants who have provided written informed consent and met all protocol eligibility requirements will be randomized to receive single (Part 1) or multiple (Part 2) doses of ARO-DM1 or placebo.",[28],"2025-11-06",{"date":255,"type":47},"2025-11-10",{"date":257,"type":47},"2024-03-04",{"date":238,"type":21},{"name":260,"class":109},"Arrowhead Pharmaceuticals",11,{"id":263,"slug":264,"hasResults":11,"nctId":265,"briefTitle":266,"officialTitle":266,"acronym":267,"eligibilityCriteria":268,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":269,"targetDuration":4,"studyType":91,"phases":271,"briefSummary":273,"conditions":274,"keywords":4,"overallStatus":43,"whyStopped":4,"lastUpdateSubmitDate":285,"lastUpdatePostDateStruct":286,"startDateStruct":288,"completionDateStruct":290,"leadSponsor":292,"locationsCount":55},"100603463","gait-analysis-parameter-and-upper-limb-evaluation-in-adult-patients-with-neurological-or-metabolic-pathology-100603463","NCT07136844","Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology","Acti-Adult","Inclusion Criteria:\n\n* Ambulant patients (i.e. able to walk 10 meters without assistance)\n* Confirmed diagnosis by the investigator based on current gold standard in his\u002Fher disease (genetic testing, clinical criteria, etc.)\n\n  * Myotonic dystrophy type 1 (DM1) and Charcot-Marie-Tooth (CMT) patients should present sensitive of motor signs on physical examination.\n  * Myasthenic patients should be seropositive, and Myasthenia Gravis Foundation of America (MGFA) class II to IV.\n  * Patient with morbid obesity (Body Mass Index\\> or = 35 at inclusion visit).\n* Signed informed consent form by patient him\u002Fherself and patient willing and able to comply with all study procedures.\n\nExclusion Criteria:\n\n* Non-ambulant patients\n* Patients with extreme cognitive disorders that limit their understanding of the exercises to be performed\n* Patients who have undergone a surgical procedure or who have experienced recent trauma (within fewer than 6 months) affecting the upper or lower limbs\n* A concomitant chronic or acute neurological, endocrine, infectious, allergic, or inflammatory pathology within the 3-week period immediately prior to inclusion\n* Patients who are participating in an interventional clinical trial\n* Pregnant or breastfeeding women",{"count":270,"type":21},300,[272],"NA","The ActiLiège-Adult study is a prospective, longitudinal, observational study designed to collect natural history data on adult patients with neurological or metabolic diseases affecting movement. Conducted at the Centre de Référence Liégeois des Maladies Neuromusculaires in Liège, Belgium, the study will enroll 300 ambulant patients, including individuals with neuromuscular disorders and obesity. Using the Syde® wearable device, the study aims to continuously monitor motor function in real-life settings over a period of up to two years. The primary objective is to evaluate the utility of digital mobility outcomes, such as the 95th centile of stride velocity (SV95C), as reliable and objective endpoints for future clinical trials.",[127,275,28,276,277,278,279,280,281,282,283,284],"Obesity (Disorder)","Myasthenic Syndrome","Charcot Marie Tooth Disease (CMT)","Glycogen Storage Disease Type II Pompe Disease","Facio-Scapulo-Humeral Dystrophy","Myasthenia Gravis","Huntington Disease","Progressive Supranuclear Palsy (PSP)","Hereditary Spastic Paraplegia","Ataxia, Spinocerebellar","2025-08-14",{"date":287,"type":47},"2025-08-22",{"date":289,"type":47},"2024-03-29",{"date":291,"type":21},"2030-12",{"name":293,"class":54},"Centre Hospitalier Universitaire de Liege",{"id":295,"slug":296,"hasResults":11,"nctId":297,"briefTitle":298,"officialTitle":299,"acronym":4,"eligibilityCriteria":300,"healthyVolunteers":175,"sex":16,"minAge":301,"maxAge":302,"enrollmentInfo":303,"targetDuration":4,"studyType":91,"phases":304,"briefSummary":305,"conditions":306,"keywords":4,"overallStatus":43,"whyStopped":4,"lastUpdateSubmitDate":312,"lastUpdatePostDateStruct":313,"startDateStruct":315,"completionDateStruct":317,"leadSponsor":319,"locationsCount":110},"100514716","assessments-in-patients-with-muscular-pathology-and-in-control-subjects--the-actilige-next-study-100514716","NCT05982119","Assessments in Patients With Muscular Pathology and in Control Subjects : The ActiLiège Next Study","Gait Analysis Parameter, Stair Climbing and Upper Limb Evaluation in Patients With Muscular Pathology and in Control Subjects: The ActiLiège Next Study","Inclusion criteria\n\n* For the patients:\n\n  * Genetically confirmed diagnosis of DMD, FSHD, DM1, CMT or FKRP mutations or confirmed CNM based on muscle biopsy.\n  * FSHD, DM1, CMT and CNM patients should be ambulant or in transition.\n  * DM1 and CMT patients should present sensori-motor signs on physical examination.\n  * Under the age of 20 years for patients with DMD, CNM or between the ages of 5 and 80 years for patients with FSHD, CMT and DM1.\n  * More than 2 years old for patients with FKRP mutations\n  * Non-ambulant DMD patients must be able to remain seated in an arm- or a wheelchair for at least one hour.\n  * Patients with DMD treated with corticosteroids for at least 6 months or initiated corticosteroid at V0 (except for patients under 4).\n  * Signed informed consent form by patient himself or, in case of minor patients, signed informed consent form by patient's parents or legal guardians.\n* For the control subjects:\n\n  * Ambulant boys and girls under 20 years old\n  * Signed informed consent form by patient him\u002Fherself or, in case of minor patients, signed informed consent form by patient's parents or legal guardians.\n\nExclusion Criteria:\n\n* For the patients:\n\n  * Patients with extreme cognitive disorders that limit their understanding of the exercises to be performed.\n  * Patients who have undergone a surgical procedure or who have experienced recent trauma (within fewer than 6 months) affecting the upper or lower limbs (for ambulant patients).\n  * A concomitant chronic or acute neurological, endocrine, infectious, allergic, or inflammatory pathology within the 3-week period immediately prior to inclusion.\n  * Patients who are participating in an interventional clinical trial.\n  * DMD patients in transition who are not on corticosteroids.\n* For the control subjects:\n\n  * Patients who have undergone a surgical procedure or who have experienced recent trauma (within fewer than 6 months) affecting the upper or lower limbs.\n  * Elite athletes (at the national level).\n  * A chronic or acute muscular, neurological, infectious, or inflammatory pathology within the 3-week period immediately prior to inclusion.\n  * An orthopedic, neuromuscular, or neurological pathology that affects the quality of the subject's walking gait.","1 Year","80 Years",{"count":270,"type":21},[272],"The objective of the ActiLiège Next study is to collect longitudinal data from patients and control subjects using a wearable magneto-inertial device. By collecting natural history data in various neuromuscular disorders (Duchenne Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy, Myotonic Dystrophy 1, Charcot-Marie-Tooth, Centronuclear Myopathy, Congenital Muscular Dystrophy), we aim to validate digital outcome measures to continuously assess motor function in real-life.",[307,308,28,309,310,311],"Duchenne Muscular Dystrophy","Fascioscapulohumeral Muscular Dystrophy","Charcot-Marie-Tooth","Centronuclear Myopathy","Congenital Muscular Dystrophy","2025-05-15",{"date":314,"type":47},"2025-05-20",{"date":316,"type":47},"2020-07-10",{"date":318,"type":21},"2026-03",{"name":293,"class":54},{"id":321,"slug":322,"hasResults":11,"nctId":323,"briefTitle":324,"officialTitle":324,"acronym":325,"eligibilityCriteria":326,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":327,"targetDuration":329,"studyType":23,"phases":4,"briefSummary":330,"conditions":331,"keywords":4,"overallStatus":43,"whyStopped":4,"lastUpdateSubmitDate":343,"lastUpdatePostDateStruct":344,"startDateStruct":346,"completionDateStruct":348,"leadSponsor":350,"locationsCount":55},"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":328,"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.",[27,332,28,333,334,335,336,337,181,338,339,340,182,341,35,342],"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","Myotonia Atrophica","2024-11-19",{"date":345,"type":47},"2024-11-21",{"date":347,"type":4},"2013-02",{"date":349,"type":21},"2030-02",{"name":351,"class":54},"Myotonic Dystrophy Foundation"]