[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"duchene-muscular-dystrophy\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:duchene-muscular-dystrophy":39},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,5,0,[8,69,113,145,157],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":26,"conditions":27,"keywords":42,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":58,"lastUpdatePostDateStruct":59,"startDateStruct":62,"completionDateStruct":64,"leadSponsor":66,"locationsCount":4},"100053565","phase-3-a-phase-3-study-to-evaluate-the-safety-and-efficacy-of-aoc-1044-also-referred-to-as-delpacibart-zotadirsen-in-participants-with-dmd-with-gene-mutations-amenable-to-exon-44-skipping-100053565",false,"NCT07587242","A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping","A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Global Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Intravenous AOC 1044 (Delpacibart Zotadirsen) for the Treatment of DMD With Gene Mutations Amenable to Exon 44 Skipping","SAFARI44","Key Inclusion Criteria:\n\n* Ambulatory males with clinical and genetic diagnosis of DMD\n* Acceptable genetic test confirming dystrophin gene mutation amenable to exon 44 skipping\n* 7 to 16 years of age at time of consent\n* TTR and NSAA assessment completed within the protocol specified parameters at Screening\n* On a stable regimen of corticosteroids (including Vamolorone) for at least 6 months prior to Day 1. Steroid regimen must be anticipated to remain stable.\n\nKey Exclusion Criteria:\n\n* Previous treatment cell or gene therapy.\n* Treatment with another oligonucleotide within 6 months of informed consent (not including COVID-19 RNA vaccines).\n* Lab values outside of the protocol specified range at Screening\n* If on any of the following treatments (growth hormone, testosterone or givinostat), participants must be on a stable regimen and must plan to maintain it for the duration of the study. Participants will be excluded if regimen stability prior to informed consent is as follows:\n* Less than 1 month, for growth hormone and\u002For testosterone\n* Less than 6 months for givinostat","MALE","7 Years","16 Years",{"count":21,"type":22},70,"ESTIMATED","INTERVENTIONAL",[25],"PHASE3","A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1044 for the treatment of Duchenne Muscular Dystrophy (DMD) with Gene Mutations Amenable to Exon 44 Skipping",[28,29,30,31,32,33,34,35,36,37,38,39,40,41],"Muscular Dystrophies","Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy)","Muscular Disorders, Atrophic","Muscular Disease","Musculoskeletal Diseases","Neuromuscular Diseases (NMD)","Nervous System Diseases","Genetic Diseases","X-Linked","Hereditary","Neonatal Disease","Duchene Muscular Dystrophy","Congenital","DMD",[43,44,45,46,47,48,49,50,15,51,52,53,54,55,56,41],"AOC","AOC 1044","AOC 1044-CS3","AOC 1044-CS1","AOC 1044-CS2","EXPLORE44","EXPLORE44-OLE","SAFARI","SAFARI 44","Avidity","Avidity Biosciences","Exon Skipping Therapy","Avidity Biosciences Inc., A Novartis Company","del-zota","NOT_YET_RECRUITING","2026-07-10",{"date":60,"type":61},"2026-07-13","ACTUAL",{"date":63,"type":22},"2026-06",{"date":65,"type":22},"2030-07",{"name":67,"class":68},"Avidity Biosciences, Inc.","INDUSTRY",{"id":70,"slug":71,"hasResults":11,"nctId":72,"briefTitle":73,"officialTitle":74,"acronym":75,"eligibilityCriteria":76,"healthyVolunteers":11,"sex":77,"minAge":78,"maxAge":4,"enrollmentInfo":79,"targetDuration":4,"studyType":81,"phases":4,"briefSummary":82,"conditions":83,"keywords":84,"overallStatus":102,"whyStopped":4,"lastUpdateSubmitDate":103,"lastUpdatePostDateStruct":104,"startDateStruct":106,"completionDateStruct":108,"leadSponsor":110,"locationsCount":112},"100602781","a-study-evaluating-the-real-world-experience-of-givinostat-in-patients-with-duchenne-muscular-dystrophy-100602781","NCT07127978","A Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy","A Prospective, Observational Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy","PROVIDUS","Inclusion Criteria:\n\n* Patients of any gender at least 6 years of age, diagnosed with DMD (according to genetic test) and have received a prescription for oral givinostat in accordance with the USPI.\n* Patient has provided informed consent (and assent when applicable) for participation in the study.\n* Patient's index date (first date of givinostat treatment) is no more than 6 months prior to signing of informed consent.\n* Patient has the required data available (DMD diagnosis, givinostat administration \\[dose and schedule\\], laboratory results \\[hematology and triglycerides\\]) for addressing the study objectives for the period between index date and study entry (for patients enrolled after the index date).\n\nExclusion Criteria:\n\n* Patient previously received givinostat (commercial or investigational product) and permanently discontinued treatment or patient started commercial givinostat for \\> 6 months before signing of informed consent.","ALL","6 Years",{"count":80,"type":22},300,"OBSERVATIONAL","This is a prospective observational study conducted to evaluate safety, tolerability, and functional outcomes of patients with DMD newly initiating oral givinostat or having started therapy within 6 months as part of routine clinical care in the US. The study has a planned maximum duration of 5 years for the first enrolled patients, including a 24-month enrollment period and a minimum of 2 years of follow-up.",[39],[41,85,86,87,88,89,90,91,92,93,94,95,96,97,98,99,100,101],"DUVYZAT","U.S study","duchenne muscular dystrophy","givinostat","non-interventional study","pediatric neuromuscular disease","rare disease","quality of life","effectiveness","post-marketing requirement","safety","motor function","muscular dystrophy","standard of care","real-world evidence","observational study","post-marketing study","RECRUITING","2026-07-01",{"date":105,"type":61},"2026-07-02",{"date":107,"type":61},"2025-10-23",{"date":109,"type":22},"2030-07-30",{"name":111,"class":68},"ITF Therapeutics LLC",9,{"id":114,"slug":115,"hasResults":11,"nctId":116,"briefTitle":117,"officialTitle":118,"acronym":119,"eligibilityCriteria":120,"healthyVolunteers":121,"sex":17,"minAge":122,"maxAge":123,"enrollmentInfo":124,"targetDuration":4,"studyType":81,"phases":4,"briefSummary":126,"conditions":127,"keywords":129,"overallStatus":102,"whyStopped":4,"lastUpdateSubmitDate":134,"lastUpdatePostDateStruct":135,"startDateStruct":137,"completionDateStruct":139,"leadSponsor":141,"locationsCount":144},"100622048","multiparametric-ultrafast-ultrasound-biomarkers-for-duchenne-and-becker-muscular-dystrophies-100622048","NCT07378553","Multiparametric Ultrafast Ultrasound Biomarkers for Duchenne and Becker Muscular Dystrophies","Multiparametric Ultrafast Ultrasound Evaluation of Skeletal Muscle in Duchenne and Becker Muscular Dystrophies","INNOVAN","Inclusion Criteria:\n\n* Inclusion Criteria for patients with Duchenne Muscular Dystrophy: Ambulatory and non-ambulatory males (ages 5-30 at baseline testing) previously diagnosed with Duchenne Muscular Dystrophy based on absence of dystrophin expression.\n* Inclusion Criteria for patients with Becker Muscular Dystrophy: Ambulatory and non-ambulatory males (ages 5-60 at baseline testing) previously diagnosed with Becker Muscular Dystrophy based on genetically confirmed, reduced or dysfunctional dystrophin.\n* Inclusion Criteria for Aged-matched controls: Ambulatory males (ages 5-60 years) without disease or injury to the lower and\u002For upper extremities\n\nExclusion Criteria:\n\n* Exclusion Criteria for patients with Duchenne and Becker Muscular Dystrophies:\n\nInability to undergo static exam, missing measurement site (resection\u002Famputation), neurocognitive impairment preventing informed consent\n\n* Exclusion Criteria for Age-matched controls: Any condition affecting muscle metabolism\u002Ffunction, neuromuscular disease, or injury to the lower and\u002For upper extremities in the past 5 years",true,"5 Years","60 Years",{"count":125,"type":22},60,"The purpose of this research study is to determine the potential of a multiparametric ultrasound approach to non-invasively monitor disease progression and to serve as an objective outcome measure for future clinical trials in Duchenne and Becker Muscular Dystrophies.\n\nThe investigators will compare the muscles of ambulatory or non-ambulatory boys\u002Fmen with Duchenne and Becker Dystrophies with muscles of healthy age-matched individuals of the same age and monitor disease progression in those with muscular dystrophies over a 12-month year period.\n\nThe ultrafast ultrasound technology used in this study allows the simultaneous assessment of muscle structure, mechanics and physiology, including stiffness, anisotropy, viscosity, intramuscular fat, muscle volume, and microvascular perfusion. The amount of muscle alteration measured will be related to performance in daily activities, such as walking and muscle strength, in order to identify sensitive and objective markers of disease progression.",[39,128],"Becker Muscular Dystrophy",[130,128,131,132,133],"Duchenne Muscular Dystrophy","Ultrasound Imaging","Ultrafast Ultrasound","Muscle","2026-06-23",{"date":136,"type":61},"2026-06-24",{"date":138,"type":61},"2026-03-03",{"date":140,"type":22},"2029-03-03",{"name":142,"class":143},"Nantes University Hospital","OTHER",1,{"id":146,"slug":4,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":147,"targetDuration":4,"studyType":23,"phases":148,"briefSummary":26,"conditions":149,"keywords":150,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":151,"lastUpdatePostDateStruct":152,"startDateStruct":154,"completionDateStruct":155,"leadSponsor":156,"locationsCount":4},"100638788",{"count":21,"type":22},[25],[28,29,30,31,32,33,34,35,36,37,38,39,40,41],[43,44,45,46,47,48,49,50,15,51,52,53,54,55,56,41],"2026-05-08",{"date":153,"type":61},"2026-05-14",{"date":63,"type":22},{"date":65,"type":22},{"name":67,"class":68},{"id":158,"slug":159,"hasResults":11,"nctId":160,"briefTitle":161,"officialTitle":162,"acronym":4,"eligibilityCriteria":163,"healthyVolunteers":11,"sex":77,"minAge":78,"maxAge":164,"enrollmentInfo":165,"targetDuration":4,"studyType":23,"phases":167,"briefSummary":169,"conditions":170,"keywords":4,"overallStatus":57,"whyStopped":4,"lastUpdateSubmitDate":171,"lastUpdatePostDateStruct":172,"startDateStruct":174,"completionDateStruct":176,"leadSponsor":178,"locationsCount":144},"100638831","how-can-different-forms-of-strengthening-exercises-affect-the-muscular-tissue-and-its-chemical-markers-in-children-with-duchenne-muscular-dystrophy-100638831","NCT07581743","How Can Different Forms of Strengthening Exercises Affect the Muscular Tissue and Its Chemical Markers in Children With Duchenne Muscular Dystrophy","Different Forms of Strengthening Exercises and Chemical Markers in Children With Duchenne Muscular Dystrophy","Inclusion Criteria:\n\n* DD,\n* aging from 6 To 10\n\nExclusion Criteria:\n\n* able to understand order","10 Years",{"count":166,"type":22},45,[168],"NA","The treatment of patients diagnosed with DMD needs to be multidisciplinary, judicious, and always focused on the patient's well-being. Physiotherapy and occupational therapy are very often standard care. Physiotherapy is regarded as crucial for the success of treatment, since it has achieved good results in the short term, such as maintaining the autonomy of these individuals . Physical activity is another method that has been used to treat DMD. Studies have used physical exercise in individuals with DMD in order to decrease muscle deterioration, muscle contractures, and bone fractures, and to increase the time of functional independence",[39],"2026-05-06",{"date":173,"type":61},"2026-05-12",{"date":175,"type":22},"2026-06-01",{"date":177,"type":22},"2026-09-01",{"name":179,"class":143},"Cairo University"]