[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT06606340":3,"trial-entities:NCT06606340":245,"trial-summary:NCT06606340":249},{"id":4,"nct_id":4,"org_study_id":5,"brief_title":6,"official_title":7,"overall_status":8,"completion_date":9,"status_verified_date":10,"last_update_date":11,"start_date":12,"sponsor_name":13,"lead_sponsor_class":14,"has_dmc":15,"brief_summary":16,"detailed_description":15,"conditions":17,"keywords":19,"study_type":27,"primary_purpose":15,"phases":28,"enrollment_info":29,"interventions":32,"primary_outcomes":51,"secondary_outcomes":65,"sex":66,"minimum_age":15,"maximum_age":15,"healthy_volunteers":67,"eligibility_criteria":68,"std_ages":78,"locations":82,"central_contacts":237,"overall_officials":238,"references":239,"see_also_links":244},"NCT06606340","4658-403","A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice","A Long-term Observational Study Evaluating Sarepta Therapeutics, Inc.'s Exon-Skipping Therapies in Patients With Duchenne Muscular Dystrophy Under Conditions of Routine Clinical Practice","ENROLLING_BY_INVITATION","2033-12-31","2025-08","2025-09-10","2019-01-07","Sarepta Therapeutics, Inc.","INDUSTRY",null,"This is a phase 4, multicenter, prospective, observational study designed to collect both medical history data and prospective data on Duchenne Muscular Dystrophy (DMD) treatment outcomes in participants receiving eteplirsen, golodirsen, and casimersen in routine clinical practice. Participants in this study will have been prescribed eteplirsen, golodirsen, or casimersen commercially prior to entry into the study.",[18],"Duchenne Muscular Dystrophy",[18,20,21,22,23,24,25,26],"DMD","Post-marketing","Observational","Phase 4","Eteplirsen","Golodirsen","Casimersen","OBSERVATIONAL",[],{"count":30,"type":31},300,"ESTIMATED",[33,41,46],{"type":34,"name":24,"description":35,"armGroupLabels":36,"otherNames":37},"DRUG","No study medication will be provided by the Sponsor as a condition to participate in this observational study, and all decisions concerning each participant's treatment are at the discretion of the participant's treating physician. Participants will receive treatment as prescribed by the treating physician as part of standard of care.",[24],[38,39,40],"AVI-4658","EXONDYS 51","EXONDYS",{"type":34,"name":25,"description":35,"armGroupLabels":42,"otherNames":43},[25],[44,45],"SRP-4053","VYONDYS 53",{"type":34,"name":26,"description":35,"armGroupLabels":47,"otherNames":48},[26],[49,50],"SRP-4045","AMONDYS 45",[52,55,57,59,61,63],{"measure":53,"timeFrame":54},"Loss of Ambulation (LOA)","Up to 5 years",{"measure":56,"timeFrame":54},"Time to Rise From the Floor (Supine to Stand)",{"measure":58,"timeFrame":54},"Time to Walk\u002FRun 10 Meters",{"measure":60,"timeFrame":54},"Performance of Upper Limb Module for DMD 2.0 (PUL 2.0) Entry Item A",{"measure":62,"timeFrame":54},"Pulmonary Function, as Measured by Forced Vital Capacity (FVC) (% Predicted)",{"measure":64,"timeFrame":54},"Cardiac Function, Including Left Ventricular Ejection Fraction (LVEF) as Measured by Echocardiogram (ECHO)",[],"MALE",false,{"inclusion":69,"exclusion":73,"raw_text":77},[70,71,72],"Is willing to provide informed assent or consent (if applicable) and has a parent(s) or legal guardian(s) or is a participant ≥18 years of age who is (are) willing to provide informed consent for the participant to participate in the study and comply with study data collection procedures.","Has an established clinical diagnosis of DMD, as documented prior to screening by a genetic report.","Receiving, or initiating treatment with, eteplirsen, golodirsen, or casimersen at the time of observational study enrollment. Note: Participants with a prescription for eteplirsen, golodirsen, or casimersen at enrollment must initiate the exon-skipping therapy within 6 months of the date of enrollment or will no longer be eligible for this study. Note: Enrollment of eteplirsen participants has been completed, no additional participants will be enrolled.",[74,75,76],"Is currently participating in any DMD interventional study at the time of this study enrollment.","Has declined to provide consent for collection of their genetic data.","Has a medical condition or confounding circumstances that, in the opinion of the Investigator, might compromise:","Key Inclusion Criteria:\n\n* Is willing to provide informed assent or consent (if applicable) and has a parent(s) or legal guardian(s) or is a participant ≥18 years of age who is (are) willing to provide informed consent for the participant to participate in the study and comply with study data collection procedures.\n* Has an established clinical diagnosis of DMD, as documented prior to screening by a genetic report.\n* Receiving, or initiating treatment with, eteplirsen, golodirsen, or casimersen at the time of observational study enrollment. Note: Participants with a prescription for eteplirsen, golodirsen, or casimersen at enrollment must initiate the exon-skipping therapy within 6 months of the date of enrollment or will no longer be eligible for this study. Note: Enrollment of eteplirsen participants has been completed, no additional participants will be enrolled.\n\nKey Exclusion Criteria:\n\n* Is currently participating in any DMD interventional study at the time of this study enrollment.\n* Has declined to provide consent for collection of their genetic data.\n* Has a medical condition or confounding circumstances that, in the opinion of the Investigator, might compromise:\n\n  1. The participant's ability to comply with the protocol-required procedures\n  2. The participant's wellbeing or safety, and\u002For\n  3. The clinical interpretability of the data collected from the participant.\n\nOther inclusion\u002Fexclusion criteria may apply.",[79,80,81],"CHILD","ADULT","OLDER_ADULT",[83,92,100,107,115,123,131,139,147,154,162,169,177,184,192,199,206,214,221,229],{"facility":84,"city":85,"state":86,"zip":87,"country":88,"geoPoint":89},"Arkansas Children's Hospital Research Institute","Little Rock","Arkansas","72202","United States",{"lat":90,"lon":91},34.74648,-92.28959,{"facility":93,"city":94,"state":95,"zip":96,"country":88,"geoPoint":97},"Children's Hospital Los Angeles","Los Angeles","California","90027",{"lat":98,"lon":99},34.05223,-118.24368,{"facility":101,"city":102,"state":95,"zip":103,"country":88,"geoPoint":104},"University of California Davis Medical Center","Sacramento","95817",{"lat":105,"lon":106},38.58157,-121.4944,{"facility":108,"city":109,"state":110,"zip":111,"country":88,"geoPoint":112},"Children's National Hospital","Washington D.C.","District of Columbia","20010",{"lat":113,"lon":114},38.89511,-77.03637,{"facility":116,"city":117,"state":118,"zip":119,"country":88,"geoPoint":120},"University of Florida","Gainesville","Florida","32610",{"lat":121,"lon":122},29.65163,-82.32483,{"facility":124,"city":125,"state":126,"zip":127,"country":88,"geoPoint":128},"University Of Iowa Hospitals And Clinics","Iowa City","Iowa","52242",{"lat":129,"lon":130},41.66113,-91.53017,{"facility":132,"city":133,"state":134,"zip":135,"country":88,"geoPoint":136},"Kennedy Krieger Institute","Baltimore","Maryland","21287",{"lat":137,"lon":138},39.29038,-76.61219,{"facility":140,"city":141,"state":142,"zip":143,"country":88,"geoPoint":144},"Washington University","St Louis","Missouri","63110",{"lat":145,"lon":146},38.62727,-90.19789,{"facility":148,"city":149,"state":149,"zip":150,"country":88,"geoPoint":151},"Columbia University Medical Center - PIN","New York","10032",{"lat":152,"lon":153},40.71427,-74.00597,{"facility":155,"city":156,"state":157,"zip":158,"country":88,"geoPoint":159},"Duke Lenox Baker Children's Hospital","Durham","North Carolina","27705",{"lat":160,"lon":161},35.99403,-78.89862,{"facility":163,"city":164,"state":157,"zip":165,"country":88,"geoPoint":166},"Atrium Health Wake Forest University Health Sciences","Winston-Salem","27157",{"lat":167,"lon":168},36.09986,-80.24422,{"facility":170,"city":171,"state":172,"zip":173,"country":88,"geoPoint":174},"Children's Hospital Medical Center","Cincinnati","Ohio","45229",{"lat":175,"lon":176},39.12711,-84.51439,{"facility":178,"city":179,"state":172,"zip":180,"country":88,"geoPoint":181},"Nationwide Children's Hospital","Columbus","43205",{"lat":182,"lon":183},39.96118,-82.99879,{"facility":185,"city":186,"state":187,"zip":188,"country":88,"geoPoint":189},"Penn State Health Milton S. Hershey Medical Center","Hershey","Pennsylvania","17033-0850",{"lat":190,"lon":191},40.28592,-76.65025,{"facility":193,"city":194,"state":187,"zip":195,"country":88,"geoPoint":196},"Children's Hospital of Philadelphia","Philadelphia","19104",{"lat":197,"lon":198},39.95238,-75.16362,{"facility":200,"city":201,"state":187,"zip":202,"country":88,"geoPoint":203},"UPMC Children's Hospital of Pittsburgh","Pittsburgh","15213",{"lat":204,"lon":205},40.44062,-79.99589,{"facility":207,"city":208,"state":209,"zip":210,"country":88,"geoPoint":211},"Cook Children's Hospital","Fort Worth","Texas","76104",{"lat":212,"lon":213},32.72541,-97.32085,{"facility":215,"city":216,"state":209,"zip":217,"country":88,"geoPoint":218},"Texas Children's Hospital","Houston","77030",{"lat":219,"lon":220},29.76328,-95.36327,{"facility":222,"city":223,"state":224,"zip":225,"country":88,"geoPoint":226},"UVA Children's Hospital","Charlottesville","Virginia","22908",{"lat":227,"lon":228},38.02931,-78.47668,{"facility":230,"city":231,"state":232,"zip":233,"country":88,"geoPoint":234},"Seattle Children's Hospital","Seattle","Washington","98105",{"lat":235,"lon":236},47.60621,-122.33207,[],[],[240],{"pmid":241,"type":242,"citation":243},"41961051","DERIVED","Tian C, Grabich S, Veerapandiyan A, Scharf RJ, Santra S, Hornibrook S, Drummond K, Sehinovych I, Waldrop M. Advancements from the EVOLVE study for assessing real-world experience with eteplirsen, golodirsen and casimersen for the treatment of DMD. J Comp Eff Res. 2026 May;15(5):e250108. doi: 10.57264\u002Fcer-2025-0108. Epub 2026 Apr 10.",[],{"nct_id":4,"conditions":246,"biomarkers":247},[18],[248],"DMD Gene",{"nct_id":4,"found":67,"summary":15,"prompt_version":15}]