[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Dyne Therapeutics\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":136},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,54,112],{"id":9,"slug":4,"hasResults":10,"nctId":11,"briefTitle":12,"officialTitle":13,"acronym":4,"eligibilityCriteria":14,"healthyVolunteers":10,"sex":15,"minAge":16,"maxAge":4,"enrollmentInfo":17,"targetDuration":4,"studyType":20,"phases":21,"briefSummary":23,"conditions":24,"keywords":30,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":43,"lastUpdatePostDateStruct":44,"startDateStruct":47,"completionDateStruct":49,"leadSponsor":51,"locationsCount":53},"100630381",false,"NCT07486934","Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1","A Phase 3, Randomized, Double-Blind, 48-Week Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of DYNE-101 Administered to Participants With Myotonic Dystrophy Type 1","Inclusion Criteria:\n\n* Diagnosis of DM1 confirmed by molecular genetics with trinucleotide repeat size greater than (\\>) 100. Historical results from clinical testing are acceptable.\n* Able to walk 10 meters and complete 5 times sit to stand independently (inserts or supports that don't go above the ankle are allowed).\n* Body mass index (BMI) less than (\\\u003C) 35 kilograms per meter square (kg\u002Fm\\^2).\n\nExclusion Criteria:\n\n* A known diagnosis of congenital DM1.\n* History of major surgical procedure (based on Investigator judgment) within 12 weeks prior to the start of screening, with the exception of implanted pacemaker or defibrillator.\n* Use of glucagon-like peptide 1 (GLP-1) agonist\u002Fincretin medications including semaglutide, dulaglutide, liraglutide, exenatide, or tirzepatide within a period of 5 half-lives of the medication prior to performing screening assessments.\n\nNote: Other inclusion and exclusion criteria may apply.","ALL","16 Years",{"count":18,"type":19},150,"ESTIMATED","INTERVENTIONAL",[22],"PHASE3","The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment basivarsen (DYNE-101) for the treatment of myotonic dystrophy 1 (DM1).",[25,26,27,28,29],"Myotonic Dystrophy Type 1 (DM1)","DM1","Myotonic Dystrophy","Steinert Disease","Steinert",[26,27,31,32,25,33,34,28,29,35,36,37,38,39,40,41],"Myotonic Dystrophy 1","Myotonia","Dystrophy Myotonic","Myotonic Disorders","Myotonic Muscular Dystrophy","HARMONIA","Dyne Therapeutics","Dyne","DYNE-101","zeleciment basivarsen","z-basivarsen","RECRUITING","2026-06-26",{"date":45,"type":46},"2026-06-30","ACTUAL",{"date":48,"type":46},"2026-05-14",{"date":50,"type":19},"2029-01",{"name":37,"class":52},"INDUSTRY",14,{"id":55,"slug":4,"hasResults":10,"nctId":56,"briefTitle":57,"officialTitle":58,"acronym":59,"eligibilityCriteria":60,"healthyVolunteers":10,"sex":61,"minAge":62,"maxAge":63,"enrollmentInfo":64,"targetDuration":4,"studyType":20,"phases":66,"briefSummary":67,"conditions":68,"keywords":82,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":103,"lastUpdatePostDateStruct":104,"startDateStruct":106,"completionDateStruct":108,"leadSponsor":110,"locationsCount":111},"100638348","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":65,"type":19},90,[22],"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.",[69,70,71,72,73,74,75,76,77,78,79,80,81],"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)",[83,72,84,85,38,37,86,87,88,89,59,90,91,92,93,94,95,96,97,98,99,100,101,102],"Ambulatory","Duchenne Muscular Dystrophy","Duchenne","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","2026-05-20",{"date":105,"type":46},"2026-05-27",{"date":107,"type":19},"2026-06",{"date":109,"type":19},"2032-10",{"name":37,"class":52},1,{"id":113,"slug":4,"hasResults":10,"nctId":114,"briefTitle":115,"officialTitle":116,"acronym":117,"eligibilityCriteria":118,"healthyVolunteers":10,"sex":15,"minAge":63,"maxAge":119,"enrollmentInfo":120,"targetDuration":4,"studyType":20,"phases":122,"briefSummary":125,"conditions":126,"keywords":4,"overallStatus":42,"whyStopped":4,"lastUpdateSubmitDate":127,"lastUpdatePostDateStruct":128,"startDateStruct":130,"completionDateStruct":132,"leadSponsor":134,"locationsCount":135},"100476280","NCT05481879","Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1","A Randomized, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-101 Administered to Participants With Myotonic Dystrophy Type 1","ACHIEVE","Inclusion Criteria:\n\n* Diagnosis of DM1 with trinucleotide repeat size \\>100.\n* Age of onset of DM1 muscle symptoms ≥12 years.\n* Clinically apparent myotonia equivalent to hand opening time of at least 2 seconds in the opinion of the Investigator.\n* Hand grip strength and ankle dorsiflexion strength.\n* Able to complete 10-MWRT, stair ascend\u002Fdescend (MAD cohorts only), and 5×STS at screening without the use of assistive devices such as canes, walkers, or orthoses.\n\nExclusion Criteria:\n\n* History of major surgical procedure within 12 weeks prior to the start of investigative product administration or an expectation of a major surgical procedure (eg, implantation of cardiac defibrillator) during the study.\n* History of anaphylaxis.\n* Medical condition other than DM1 that would significantly impact ambulation or participation in functional assessments.\n* Treatment with medications that can improve myotonia within a period of 5 half-lives of the medication prior to performing screening assessments.\n* Electrocardiogram (ECG) with the corrected QT interval by Fridericia's Formula (QTcF) ≥450 milliseconds (ms) in men and QTcF ≥460 ms in women, PR ≥240 ms, left bundle-branch block, or a conduction defect, which is clinically significant in the opinion of the Investigator.\n* Percent predicted forced vital capacity (FVC) \\\u003C50%.\n* History of tibialis anterior biopsy within 3 months of Day 1 or planning to undergo tibialis anterior biopsies during study period for reasons unrelated to the study.\n* Participant has a history of suicide attempt, suicidal behavior, or has any suicidal ideation within 6 months prior to Screening that meets criteria at a level of 4 or 5 of the Columbia Suicide Severity Rating Scale (C-SSRS) or who, in the opinion of the Investigator, is at significant risk to commit suicide.\n* Use of glucagon-like peptide 1 (GLP-1) agonist medications including semaglutide, dulaglutide, liraglutide, exenatide, or tirzepatide within a period of 5 half-lives of the medication prior to performing screening assessments.\n* Significant weight loss during study participation may impact weight-based dosing, performance on muscle function assessments, and pharmacodynamic (PD) biomarkers.\n\nNote: Other inclusion and exclusion criteria may apply.","65 Years",{"count":121,"type":19},116,[123,124],"PHASE1","PHASE2","The primary purpose of the study is to evaluate the safety and tolerability of multiple intravenous (IV) doses of DYNE-101 administered to participants with Myotonic Dystrophy Type 1 (DM1).\n\nThe study consists of 4 periods: A Screening Period (up to 8 weeks), a Placebo-Controlled Period (24 weeks), a Treatment Period (24 weeks) and a Long-Term Extension (LTE) Period (168 weeks) in both multiple-ascending dose (MAD) and dose expansion cohorts.",[25],"2026-05-08",{"date":129,"type":46},"2026-05-12",{"date":131,"type":46},"2022-09-05",{"date":133,"type":19},"2029-07",{"name":37,"class":52},20,""]