[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT07008469":3,"trial-entities:NCT07008469":353,"trial-summary:NCT07008469":358},{"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":27,"study_type":39,"primary_purpose":40,"phases":41,"enrollment_info":43,"interventions":46,"primary_outcomes":52,"secondary_outcomes":57,"sex":58,"minimum_age":59,"maximum_age":60,"healthy_volunteers":61,"eligibility_criteria":62,"std_ages":78,"locations":82,"central_contacts":349,"overall_officials":350,"references":351,"see_also_links":352},"NCT07008469","AOC 1001-CS4","Global Open-Label Extension Study of Del-desiran for the Treatment of DM1","A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1","ENROLLING_BY_INVITATION","2030-10","2026-07","2026-07-23","2025-06-26","Avidity Biosciences, Inc.","INDUSTRY",true,"A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1","The study consists of a Screening Period of up to either 4-weeks or 8-weeks depending on the prior parent trial, and up to a 4-year Treatment Period. The anticipated duration is 50 months and 2 weeks (4 years and 2.5 months).\n\nParticipants will receive an intravenous infusion of del-desiran at the clinical study site every 8 weeks for a total of 7 doses per year. The final dose will occur at Year 4, Visit 7, followed by a final assessment 8 weeks after the last dose.\n\nAn additional subgroup of de novo participants will also be included in a Fixed-Dose PK cohort.\n\nAn Independent Data Monitoring Committee (IDMC) comprised of members independent and external to the Sponsor will review safety, tolerability, and efficacy (as needed) data of this study at regular intervals.",[19,20,21,22,23,24,25,26],"Myotonic Dystrophy Type 1","DM1","Myotonic Dystrophy","Myotonia","Myotonic Dystrophy 1","Myotonic Disorders","Steinert Myotonic Dystrophy","Steinert Disease",[20,21,23,22,28,29,24,26,30,31,32,33,34,35,36,37,38],"Myotonic Dystrophy Type 1 (DM1)","Dystrophy Myotonic","Steinert","Myotonic Muscular Dystrophy","HARBOR-OLE","Avidity Biosciences","Avidity","AOC 1001","Del-desiran","Delpacibart etedesiran","HARBOR","INTERVENTIONAL","TREATMENT",[42],"PHASE3",{"count":44,"type":45},230,"ESTIMATED",[47],{"type":48,"name":49,"description":50,"armGroupLabels":51},"DRUG","Del-desiran (AOC 1001)","Del-desiran will be administered by intravenous (IV) infusion.",[36],[53],{"measure":54,"description":55,"timeFrame":56},"TEAEs","Incidence of treatment emergent adverse events","From enrollment to the end of treatment at 4 years",[],"ALL","16 Years",null,false,{"inclusion":63,"exclusion":66,"raw_text":77},[64,65],"Completion of any prior AOC 1001 studies with satisfactory completion of dosing and follow-up assessments and satisfactory compliance with the protocol requirements of the parent study, as determined by the Investigator.","Clinical and genetic diagnosis of DM1 and clinically significant hand myotonia.",[67,68,69,70,71,72,73,74,75,76],"Breastfeeding, pregnancy, or intent to become pregnant during the study.","Unwilling to comply with contraceptive requirements.","Any new conditions or worsening of existing condition that in the opinion of the Investigator would make the participant unsuitable for the study.","Breastfeeding, pregnancy, or intent to become pregnant during the study","Unwilling to comply with contraceptive requirements","Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study","Diabetes that is not adequately controlled","History of decompensated heart failure within 3 months of screening. Participants with preexisting pacemaker\u002FICD are not excluded","Body Mass Index \\> 35 kg\u002Fm2 at Screening","Recently treated with an investigational drug or biological agent","FROM A PARENT STUDY\n\nKey Inclusion Criteria:\n\n* Completion of any prior AOC 1001 studies with satisfactory completion of dosing and follow-up assessments and satisfactory compliance with the protocol requirements of the parent study, as determined by the Investigator.\n\nKey Exclusion Criteria:\n\n* Breastfeeding, pregnancy, or intent to become pregnant during the study.\n* Unwilling to comply with contraceptive requirements.\n* Any new conditions or worsening of existing condition that in the opinion of the Investigator would make the participant unsuitable for the study.\n\nFIXED-DOSE PK COHORT\n\nKey Inclusion Criteria:\n\n* Clinical and genetic diagnosis of DM1 and clinically significant hand myotonia.\n\nKey Exclusion Criteria:\n\n* Breastfeeding, pregnancy, or intent to become pregnant during the study\n* Unwilling to comply with contraceptive requirements\n* Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study\n* Diabetes that is not adequately controlled\n* History of decompensated heart failure within 3 months of screening. Participants with preexisting pacemaker\u002FICD are not excluded\n* Body Mass Index \\> 35 kg\u002Fm2 at Screening\n* Recently treated with an investigational drug or biological agent\n\nNote: Additional protocol defined Inclusion and Exclusion criteria apply",[79,80,81],"CHILD","ADULT","OLDER_ADULT",[83,92,99,107,115,122,130,138,146,154,162,170,178,185,193,200,208,216,224,232,241,249,257,264,272,280,288,296,303,307,314,322,329,337,345],{"facility":84,"city":85,"state":86,"zip":87,"country":88,"geoPoint":89},"University of California, Los Angeles (UCLA)","Los Angeles","California","90095","United States",{"lat":90,"lon":91},34.05223,-118.24368,{"facility":93,"city":94,"state":86,"zip":95,"country":88,"geoPoint":96},"Stanford University","Stanford","94305",{"lat":97,"lon":98},37.42411,-122.16608,{"facility":100,"city":101,"state":102,"zip":103,"country":88,"geoPoint":104},"University of Colorado","Denver","Colorado","80045",{"lat":105,"lon":106},39.73915,-104.9847,{"facility":108,"city":109,"state":110,"zip":111,"country":88,"geoPoint":112},"University of Florida","Gainesville","Florida","32608",{"lat":113,"lon":114},29.65163,-82.32483,{"facility":116,"city":117,"state":110,"zip":118,"country":88,"geoPoint":119},"University Research Center of South Florida","Tampa","33612",{"lat":120,"lon":121},27.94752,-82.45843,{"facility":123,"city":124,"state":125,"zip":126,"country":88,"geoPoint":127},"Indiana University (IU)","Indianapolis","Indiana","46202",{"lat":128,"lon":129},39.76838,-86.15804,{"facility":131,"city":132,"state":133,"zip":134,"country":88,"geoPoint":135},"Kansas University Medical Center","Kansas City","Kansas","66205",{"lat":136,"lon":137},39.11417,-94.62746,{"facility":139,"city":140,"state":141,"zip":142,"country":88,"geoPoint":143},"Kennedy Krieger Institute","Baltimore","Maryland","21205",{"lat":144,"lon":145},39.29038,-76.61219,{"facility":147,"city":148,"state":149,"zip":150,"country":88,"geoPoint":151},"Beth Israel Deaconess Medical Center","Boston","Massachusetts","02215",{"lat":152,"lon":153},42.35843,-71.05977,{"facility":155,"city":156,"state":157,"zip":158,"country":88,"geoPoint":159},"University of Minnesota","Minneapolis","Minnesota","55455",{"lat":160,"lon":161},44.97997,-93.26384,{"facility":163,"city":164,"state":165,"zip":166,"country":88,"geoPoint":167},"University of Rochester Medical Center","Rochester","New York","14642",{"lat":168,"lon":169},43.15478,-77.61556,{"facility":171,"city":172,"state":173,"zip":174,"country":88,"geoPoint":175},"Duke University Medical Center","Durham","North Carolina","27708",{"lat":176,"lon":177},35.99403,-78.89862,{"facility":179,"city":180,"state":173,"zip":181,"country":88,"geoPoint":182},"Wake Forest","Winston-Salem","27157",{"lat":183,"lon":184},36.09986,-80.24422,{"facility":186,"city":187,"state":188,"zip":189,"country":88,"geoPoint":190},"University of Cincinnati Gardner Neuroscience Institute","Cincinnati","Ohio","45219",{"lat":191,"lon":192},39.12711,-84.51439,{"facility":194,"city":195,"state":188,"zip":196,"country":88,"geoPoint":197},"Ohio State University","Columbus","43221",{"lat":198,"lon":199},39.96118,-82.99879,{"facility":201,"city":202,"state":203,"zip":204,"country":88,"geoPoint":205},"University of Pennsylvania","Philadelphia","Pennsylvania","19104",{"lat":206,"lon":207},39.95238,-75.16362,{"facility":209,"city":210,"state":211,"zip":212,"country":88,"geoPoint":213},"Houston Methodist Neurological Institute","Houston","Texas","77030",{"lat":214,"lon":215},29.76328,-95.36327,{"facility":217,"city":218,"state":219,"zip":220,"country":88,"geoPoint":221},"Virginia Commonwealth University","Richmond","Virginia","23298",{"lat":222,"lon":223},37.55376,-77.46026,{"facility":225,"city":226,"state":227,"zip":228,"country":88,"geoPoint":229},"University of Washington","Seattle","Washington","98104",{"lat":230,"lon":231},47.60621,-122.33207,{"facility":233,"city":234,"state":235,"zip":236,"country":237,"geoPoint":238},"The Ottawa Hospital","Ottawa","Ontario","K1Y 4E9","Canada",{"lat":239,"lon":240},45.41117,-75.69812,{"facility":242,"city":243,"state":244,"zip":245,"country":237,"geoPoint":246},"Montreal Neurological Institute","Montreal","Quebec","H3A 2B4",{"lat":247,"lon":248},45.50884,-73.58781,{"facility":250,"city":251,"zip":252,"country":253,"geoPoint":254},"Aarhus University Hospital","Aarhus","8200","Denmark",{"lat":255,"lon":256},56.15674,10.21076,{"facility":258,"city":259,"zip":260,"country":253,"geoPoint":261},"Rigshospitalet","Copenhagen","2100",{"lat":262,"lon":263},55.67594,12.56553,{"facility":265,"city":266,"zip":267,"country":268,"geoPoint":269},"AP-HP Hopital Pitie-Salpetriere","Paris","75013","France",{"lat":270,"lon":271},48.85341,2.3488,{"facility":273,"city":274,"zip":275,"country":276,"geoPoint":277},"Klinikum der Ludwig-Maximilians-Universitaet Muenchen","München","80336","Germany",{"lat":278,"lon":279},51.60698,13.31243,{"facility":281,"city":282,"zip":283,"country":284,"geoPoint":285},"Fondazione Serena Onlus - Centro Clinico NeMO Milano","Milan","20162","Italy",{"lat":286,"lon":287},42.78235,12.59836,{"facility":289,"city":290,"state":290,"zip":291,"country":292,"geoPoint":293},"Aomori Hospital","Aomori","038-1331","Japan",{"lat":294,"lon":295},40.81667,140.73333,{"facility":297,"city":298,"zip":299,"country":292,"geoPoint":300},"National Hospital Organization Osaka Toneyama Medical Center","Osaka","560-8552",{"lat":301,"lon":302},34.69379,135.50107,{"facility":304,"city":298,"zip":305,"country":292,"geoPoint":306},"Osaka University Hospital","565-0871",{"lat":301,"lon":302},{"facility":308,"city":309,"zip":310,"country":292,"geoPoint":311},"National Center of Neurology and Psychiatry","Tokyo","187-8551",{"lat":312,"lon":313},35.6895,139.69171,{"facility":315,"city":316,"zip":317,"country":318,"geoPoint":319},"Maastricht University Medical Center","Maastricht","6229 HX","Netherlands",{"lat":320,"lon":321},50.84833,5.68889,{"facility":323,"city":324,"zip":325,"country":318,"geoPoint":326},"Stichting Radboud Universitair Medisch Centrum","Nijmegen","6525 GA",{"lat":327,"lon":328},51.8425,5.85278,{"facility":330,"city":331,"zip":332,"country":333,"geoPoint":334},"Hospital Universitario Donostia","San Sebastián","20014","Spain",{"lat":335,"lon":336},43.56667,-5.9,{"facility":338,"city":339,"zip":340,"country":341,"geoPoint":342},"University College London Hospital","London","NW1 2BU","United Kingdom",{"lat":343,"lon":344},51.50853,-0.12574,{"facility":346,"city":339,"zip":347,"country":341,"geoPoint":348},"St. Georges University Hospitals NHS Foundation Trust","SW17 0QT",{"lat":343,"lon":344},[],[],[],[],{"nct_id":4,"conditions":354,"biomarkers":356},[355],"Dystrophia Myotonica 1",[357],"DMPK Gene",{"nct_id":4,"found":61,"summary":60,"prompt_version":60}]