[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT06270719":3,"trial-entities:NCT06270719":301,"trial-summary:NCT06270719":304},{"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":17,"conditions":18,"keywords":20,"study_type":26,"primary_purpose":17,"phases":27,"enrollment_info":28,"interventions":31,"primary_outcomes":47,"secondary_outcomes":54,"sex":78,"minimum_age":79,"maximum_age":17,"healthy_volunteers":15,"eligibility_criteria":80,"std_ages":97,"locations":101,"central_contacts":297,"overall_officials":298,"references":299,"see_also_links":300},"NCT06270719","SRP-9001-401","An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy","A Long-term Multicenter Prospective Observational Study Evaluating the Comparative Effectiveness and Safety of Sarepta Gene Transfer Therapy vs. Standard of Care in Participants With Duchenne Muscular Dystrophy Under Conditions of Routine Clinical Practice","ENROLLING_BY_INVITATION","2038-12-31","2026-04","2026-05-04","2024-02-07","Sarepta Therapeutics, Inc.","INDUSTRY",false,"This is a multicenter, prospective, observational Phase 4 study including a post marketing safety requirement, designed to collect both medical history data and prospective data on Duchenne muscular dystrophy (DMD) treatment outcomes in participants receiving delandistrogene moxeparvovec (ELEVIDYS) as part of clinical care, compared to participants with DMD receiving or prescribed to start chronic glucocorticoid treatment at the time of study enrollment in routine clinical practice.",null,[19],"Duchenne Muscular Dystrophy",[19,21,22,23,24,25],"DMD","Observational","Standard of Care","Phase 4","ELEVIDYS","OBSERVATIONAL",[],{"count":29,"type":30},500,"ESTIMATED",[32,41],{"type":33,"name":34,"description":35,"armGroupLabels":36,"otherNames":38},"GENETIC","Delandistrogene Moxeparvovec","No study medication will be provided by the sponsor during this study.",[37],"Cohort 1 (Treated)",[39,40,25],"SRP-9001","delandistrogene moxeparvovec-rokl",{"type":42,"name":23,"description":35,"armGroupLabels":43,"otherNames":45},"DRUG",[44],"Cohort 2 (Standard of Care)",[46],"Chronic glucocorticoid treatment",[48,51],{"measure":49,"timeFrame":50},"Mean Change From Baseline in Time to Walk\u002FRun 10 Meters (10MWR) (Calculated Velocity) at Month 12","Baseline, Month 12",{"measure":52,"timeFrame":53},"Number of Participants Experiencing Acute Liver Injury (ALI)","Baseline through Month 12",[55,58,60,62,64,66,68,70,72,74,76],{"measure":56,"timeFrame":57},"Time to Rise From Floor (Supine to Stand)","Up to 10 years",{"measure":59,"timeFrame":57},"Loss of Ambulation (LOA)",{"measure":61,"timeFrame":57},"Performance of Upper Limb (PUL) Version 2.0 Entry Item A Score or Brooke Upper Extremity Scale Score",{"measure":63,"timeFrame":57},"Patient-reported Outcomes Measurement Information (PROMIS) Domain Scores of Mobility, Upper Extremity, and Fatigue",{"measure":65,"timeFrame":57},"Pulmonary Function as Measured by Forced Vital Capacity (FVC)",{"measure":67,"timeFrame":57},"Pulmonary Function as Measured by FVC Percent Predicted (FVC%p)",{"measure":69,"timeFrame":57},"Cardiac Function, Including Left Ventricular Ejection Fraction (LVEF), as Measured by Echocardiogram (ECHO) or Cardiac MRI (cMRI)",{"measure":71,"timeFrame":53},"Number of Participants Experiencing Serious ALI and Acute Liver Failure (ALF)",{"measure":73,"timeFrame":57},"Number of Participants Experiencing Complications Associated with ALI and ALF",{"measure":75,"timeFrame":57},"Survival Time",{"measure":77,"timeFrame":57},"Time to First Vertebral Body (Spine) Fracture","MALE","4 Years",{"inclusion":81,"exclusion":88,"raw_text":96},[82,83,84,85,86,87],"Has an established clinical diagnosis of DMD based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test.","Is currently receiving or has been prescribed to start chronic glucocorticoid therapy at the time of this observational study enrollment.","Is at least 4 years of age at the time of infusion","Will either: a) be initiating or has initiated ELEVIDYS within the last 30 days in routine clinical practice at the time of this observational study enrollment, or b) was administered ELEVIDYS in routine clinical practice and has the required minimum dataset for entry into the observational study per Sponsor approval","Is at least 4 years of age at the time of enrollment","Is unexposed to DMD gene therapy at the time of this observational study enrollment",[89,90,91,92,93,94,95],"Has any deletion of exon 8 and\u002For exon 9 in the DMD gene.","Is currently participating in any DMD interventional study at the time of this observational study enrollment.","Has any prior exposure to DMD gene therapy other than that described for Cohort 1c (ELEVIDYS Retrospectively Treated Cohort).","Has a medical condition or confounding circumstances (for example, prior traumatic limitation for mobility or significant behavioral comorbidity) that, in the opinion of the Investigator, might compromise:","The participant's ability to comply with the protocol-required procedures,","The participant's wellbeing or safety, and\u002For","The clinical interpretability of the data collected from the participant.","Inclusion Criteria:\n\n* Has an established clinical diagnosis of DMD based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test.\n* Is currently receiving or has been prescribed to start chronic glucocorticoid therapy at the time of this observational study enrollment.\n\nFor ELEVIDYS-treated Participants (Cohorts 1a, 1b, and 1c):\n\n* Is at least 4 years of age at the time of infusion\n* Will either: a) be initiating or has initiated ELEVIDYS within the last 30 days in routine clinical practice at the time of this observational study enrollment, or b) was administered ELEVIDYS in routine clinical practice and has the required minimum dataset for entry into the observational study per Sponsor approval\n\nFor Standard of Care Comparators (Cohort 2):\n\n* Is at least 4 years of age at the time of enrollment\n* Is unexposed to DMD gene therapy at the time of this observational study enrollment\n\nExclusion Criteria:\n\n* Has any deletion of exon 8 and\u002For exon 9 in the DMD gene.\n* Is currently participating in any DMD interventional study at the time of this observational study enrollment.\n* Has any prior exposure to DMD gene therapy other than that described for Cohort 1c (ELEVIDYS Retrospectively Treated Cohort).\n* Has a medical condition or confounding circumstances (for example, prior traumatic limitation for mobility or significant behavioral comorbidity) that, in the opinion of the Investigator, might compromise:\n\n  * The participant's ability to comply with the protocol-required procedures,\n  * The participant's wellbeing or safety, and\u002For\n  * The clinical interpretability of the data collected from the participant.\n\nOther inclusion\u002Fexclusion criteria may apply.",[98,99,100],"CHILD","ADULT","OLDER_ADULT",[102,111,119,127,135,143,151,158,165,173,181,189,197,205,213,221,229,236,244,251,259,267,274,282,290],{"facility":103,"city":104,"state":105,"zip":106,"country":107,"geoPoint":108},"Arkansas Children's Hospital","Little Rock","Arkansas","72202","United States",{"lat":109,"lon":110},34.74648,-92.28959,{"facility":112,"city":113,"state":114,"zip":115,"country":107,"geoPoint":116},"Children's Hospital Los Angeles - PIN","Los Angeles","California","90027",{"lat":117,"lon":118},34.05223,-118.24368,{"facility":120,"city":121,"state":122,"zip":123,"country":107,"geoPoint":124},"University of Colorado - PPDS","Aurora","Colorado","80045",{"lat":125,"lon":126},39.72943,-104.83192,{"facility":128,"city":129,"state":130,"zip":131,"country":107,"geoPoint":132},"Connecticut Children's Medical Center - Hartford","Hartford","Connecticut","06106",{"lat":133,"lon":134},41.76371,-72.68509,{"facility":136,"city":137,"state":138,"zip":139,"country":107,"geoPoint":140},"Children's National Medical Center","Washington D.C.","District of Columbia","20010",{"lat":141,"lon":142},38.89511,-77.03637,{"facility":144,"city":145,"state":146,"zip":147,"country":107,"geoPoint":148},"Nicklaus Children's Hospital","Miami","Florida","33155",{"lat":149,"lon":150},25.77427,-80.19366,{"facility":152,"city":153,"state":146,"zip":154,"country":107,"geoPoint":155},"Nemours Children's Hospital - Orlando","Orlando","32827",{"lat":156,"lon":157},28.53834,-81.37924,{"facility":159,"city":160,"state":146,"zip":161,"country":107,"geoPoint":162},"All Children's Research Institute, Inc","St. Petersburg","33701",{"lat":163,"lon":164},27.77086,-82.67927,{"facility":166,"city":167,"state":168,"zip":169,"country":107,"geoPoint":170},"Ann and Robert H Lurie Childrens Hospital of Chicago","Chicago","Illinois","60611-2991",{"lat":171,"lon":172},41.85003,-87.65005,{"facility":174,"city":175,"state":176,"zip":177,"country":107,"geoPoint":178},"Indiana Clinical and Translational Science Institute","Indianapolis","Indiana","46202",{"lat":179,"lon":180},39.76838,-86.15804,{"facility":182,"city":183,"state":184,"zip":185,"country":107,"geoPoint":186},"University of Kansas Medical Center","Kansas City","Kansas","66160",{"lat":187,"lon":188},39.11417,-94.62746,{"facility":190,"city":191,"state":192,"zip":193,"country":107,"geoPoint":194},"University of Michigan","Ann Arbor","Michigan","48109",{"lat":195,"lon":196},42.27756,-83.74088,{"facility":198,"city":199,"state":200,"zip":201,"country":107,"geoPoint":202},"Duke Lenox Baker Children's Hospital","Durham","North Carolina","27704",{"lat":203,"lon":204},35.99403,-78.89862,{"facility":206,"city":207,"state":208,"zip":209,"country":107,"geoPoint":210},"Childrens Hospital Medical Center of Akron","Akron","Ohio","44302",{"lat":211,"lon":212},41.08144,-81.51901,{"facility":214,"city":215,"state":216,"zip":217,"country":107,"geoPoint":218},"OHSU Healthcare (Oregon Health and Science University)","Portland","Oregon","97239",{"lat":219,"lon":220},45.52345,-122.67621,{"facility":222,"city":223,"state":224,"zip":225,"country":107,"geoPoint":226},"Penn State Health Milton S. Hershey Medical Center","Hershey","Pennsylvania","17033",{"lat":227,"lon":228},40.28592,-76.65025,{"facility":230,"city":231,"state":224,"zip":232,"country":107,"geoPoint":233},"Children's Hospital of Philadelphia","Philadelphia","19104",{"lat":234,"lon":235},39.95238,-75.16362,{"facility":237,"city":238,"state":239,"zip":240,"country":107,"geoPoint":241},"Le Bonheur Children's Hospital","Memphis","Tennessee","38103",{"lat":242,"lon":243},35.14953,-90.04898,{"facility":245,"city":246,"state":239,"zip":247,"country":107,"geoPoint":248},"Vanderbilt University Medical Center","Nashville","37232",{"lat":249,"lon":250},36.16589,-86.78444,{"facility":252,"city":253,"state":254,"zip":255,"country":107,"geoPoint":256},"Cook Children's Hospital","Fort Worth","Texas","76104",{"lat":257,"lon":258},32.72541,-97.32085,{"facility":260,"city":261,"state":262,"zip":263,"country":107,"geoPoint":264},"University of Virginia","Charlottesville","Virginia","22903",{"lat":265,"lon":266},38.02931,-78.47668,{"facility":268,"city":269,"state":262,"zip":270,"country":107,"geoPoint":271},"Children's Hospital of the King's Daughters","Norfolk","23507",{"lat":272,"lon":273},36.84681,-76.28522,{"facility":275,"city":276,"state":277,"zip":278,"country":107,"geoPoint":279},"Seattle Children's Hospital","Seattle","Washington","98105",{"lat":280,"lon":281},47.60621,-122.33207,{"facility":283,"city":284,"state":285,"zip":286,"country":107,"geoPoint":287},"The Board of Regents of the University of Wisconsin","Madison","Wisconsin","53715-1218",{"lat":288,"lon":289},43.07305,-89.40123,{"facility":291,"city":292,"state":285,"zip":293,"country":107,"geoPoint":294},"The Medical College of Wisconsin","Milwaukee","53226-4874",{"lat":295,"lon":296},43.0389,-87.90647,[],[],[],[],{"nct_id":4,"conditions":302,"biomarkers":303},[19],[],{"nct_id":4,"found":15,"summary":17,"prompt_version":17}]