[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"muscular-dystrophy-in-children\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:muscular-dystrophy-in-children":33},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,76],{"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":41,"overallStatus":64,"whyStopped":4,"lastUpdateSubmitDate":65,"lastUpdatePostDateStruct":66,"startDateStruct":69,"completionDateStruct":71,"leadSponsor":73,"locationsCount":75},"100638348","phase-3-efficacy-safety-and-tolerability-of-zeleciment-rostudirsen-dyne-251-administered-intravenously-every-4-weeks-in-ambulatory-participants-with-duchenne-muscular-dystrophy-forzetto-100638348",false,"NCT07608432","Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)","A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy, Safety, and Tolerability of DYNE-251 Administered Intravenously in Ambulatory Male Participants 4 to 18 Years of Age With Duchenne Muscular Dystrophy Amenable to Exon-51 Skipping","FORZETTO","Inclusion Criteria:\n\n* Ambulatory male with confirmed diagnosis of DMD and with a mutation in the dystrophin gene characterized by exon deletion amenable to exon 51 skipping .\n* Rise From Floor (RFF) time must be \\\u003C 10 seconds for both screening assessments .\n* Receiving a stable daily or weekend dosage of glucocorticoids for at least 24 weeks prior to randomization with the expectation of maintaining a stable dose during the Placebo-Controlled Period of the study (unless dose adjustment is required by weight change)\n\nExclusion Criteria:\n\n* Receipt of ongoing immunosuppressive therapy (other than glucocorticoids) within 12 weeks prior to randomization\n* Use of any pharmacologic treatment (other than glucocorticoids) that may have an effect on muscle strength or function within 12 weeks prior to randomization\n* Any change in prophylaxis\u002Ftreatment for congestive heart failure (CHF) within 12 weeks prior to randomization\n* Receipt of eteplirsen within 1 week prior to randomization\n* Receipt of alternative exon-skipping or dystrophin-modifying therapy or zeleciment rostudirsen within 24 weeks prior to randomization\n* Receipt of givinostat within 12 weeks prior to randomization\n* Receipt of gene therapy at any time\n\nNote: Other inclusion or exclusion criteria may apply","MALE","4 Years","18 Years",{"count":21,"type":22},90,"ESTIMATED","INTERVENTIONAL",[25],"PHASE3","The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment rostudirsen (DYNE-251) administered intravenously (IV) every 4 weeks to ambulatory Duchenne muscular dystrophy (DMD) participants, 4 to 18 years of age, with dystrophin mutations amenable to exon 51 skipping.",[28,29,30,31,32,33,34,35,36,37,38,39,40],"Duchenne Muscular Dystrophy (DMD)","Muscular Dystrophy, Duchenne","Muscular Dystrophy (DMD)","DMD","Muscular Dystrophies","Muscular Dystrophy in Children","Muscular Dystrophy, Duchenne Type","Muscular Dystrophy, Duchenne and Becker Types","Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy)","Genetic Disease, Inborn","Genetic Disease, X-Linked","Congenital, Hereditary, and Neonatal Diseases and Abnormalities","Neuromuscular Diseases (NMD)",[42,31,43,44,45,46,47,48,49,50,15,51,52,53,54,55,56,57,58,59,60,61,62,63],"Ambulatory","Duchenne Muscular Dystrophy","Duchenne","Dyne","Dyne Therapeutics","DYNE-251","Dystrophy","Exon Skipping","Exon 51","Pediatric","PMO","Muscle Function","Muscular Dystropy, Duchenne","Rise From Floor","RFF","RFF Velocity","Rostudirsen","Time to rise","TTR","TTR Velocity","Zeleciment rostudirsen","Z-rostudirsen","RECRUITING","2026-05-20",{"date":67,"type":68},"2026-05-27","ACTUAL",{"date":70,"type":22},"2026-06",{"date":72,"type":22},"2032-10",{"name":46,"class":74},"INDUSTRY",1,{"id":77,"slug":78,"hasResults":11,"nctId":79,"briefTitle":80,"officialTitle":81,"acronym":4,"eligibilityCriteria":82,"healthyVolunteers":11,"sex":17,"minAge":83,"maxAge":4,"enrollmentInfo":84,"targetDuration":4,"studyType":23,"phases":86,"briefSummary":88,"conditions":89,"keywords":94,"overallStatus":64,"whyStopped":4,"lastUpdateSubmitDate":98,"lastUpdatePostDateStruct":99,"startDateStruct":101,"completionDateStruct":103,"leadSponsor":105,"locationsCount":75},"100538429","phase-2-vasodilator-and-exercise-study-for-dmd-vaso-rex-100538429","NCT06290713","Vasodilator and Exercise Study for DMD (VASO-REx)","Vasodilators and Exercise as Adjuvant Therapy for Duchenne Muscular Dystrophy (VASO-REx Study)","Inclusion Criteria:\n\n* Diagnosis of DMD confirmed by genetic report\n* Minimum entry age of 6.0 years old\n* Ambulatory\n* On stable glucocorticoid regimen (for \\> 3 months)\n\nExclusion Criteria:\n\n* Contraindication to a Magnetic resonance Imaging examination (e.g. severe claustrophobia, magnetic implants, unable\u002Funwilling to perform test)\n* Presence of unstable medical problems, including severe cardiomyopathy, left ventricular ejection fraction \\\u003C45%, cardiac conduction abnormalities as evidenced on ECG, uncontrolled seizure disorder, uncontrolled hypo or hypertension\n* Presence of a secondary condition that impacts muscle function or muscle metabolism (e.g., myasthenia gravis, endocrine disorder, mitochondrial disease)\n* Presence of a secondary condition leading to developmental delay or impaired motor control (e.g., cerebral palsy) or previous history of unprovoked rhabdomyolysis\n* Contraindications to phosphodiesterase 5 inhibitors (use of nitrates, alpha-adrenergic blockers, other phosphodiesterase 5 inhibitors) or other medications known to modulate blood flow or muscle metabolism\n* Participation in currently approved FDA trials or other investigational clinical trials during the period of the study","6 Years",{"count":85,"type":22},50,[87],"PHASE2","Examining two strategies as potential adjuvant therapies for Duchenne muscular dystrophy (DMD); aerobic exercise training (to induce adaptations in skeletal muscle and improve cardiovascular health) and tadalafil, an FDA-approved vasodilator (to optimize blood flow and muscle perfusion which is impaired and often overlooked in DMD). Target: improved muscle function, vascular health, and DMD treatment.",[43,90,91,33,92,93,31],"Duchenne Disease","Muscular Dystrophy","Vasodilation","Exercise",[31,95,96,97],"Tadalafil","Drug and Exercise Intervention","Treatment Strategy","2026-05-12",{"date":100,"type":68},"2026-05-15",{"date":102,"type":68},"2024-06-05",{"date":104,"type":22},"2026-11",{"name":106,"class":107},"University of Florida","OTHER"]