[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100526745":3},{"organization":4,"armGroups":7,"interventions":30,"overallOfficials":51,"centralContacts":55,"locations":61,"responsibleParty":372,"collaborators":41,"id":374,"slug":375,"hasResults":376,"nctId":377,"briefTitle":378,"officialTitle":379,"acronym":41,"eligibilityCriteria":380,"healthyVolunteers":376,"sex":381,"minAge":382,"maxAge":383,"enrollmentInfo":384,"targetDuration":41,"studyType":387,"phases":388,"briefSummary":391,"conditions":392,"keywords":394,"overallStatus":64,"whyStopped":41,"lastUpdateSubmitDate":397,"lastUpdatePostDateStruct":398,"startDateStruct":401,"completionDateStruct":403,"leadSponsor":405,"locationsCount":406},{"fullName":5,"class":6},"Sarepta Therapeutics, Inc.","INDUSTRY",[8,14,20,25],{"label":9,"type":10,"description":11,"interventionNames":12},"SRP-1003 IV Infusion","EXPERIMENTAL","Single or multiple doses of SRP-1003 by intravenous (IV) infusion",[13],"Drug: SRP-1003 IV Infusion",{"label":15,"type":16,"description":17,"interventionNames":18},"Placebo by IV Infusion","PLACEBO_COMPARATOR","Single or multiple doses of placebo by IV infusion",[19],"Drug: Placebo IV Infusion",{"label":21,"type":10,"description":22,"interventionNames":23},"SRP-1003 SC Injection","Single or multiple doses of SRP-1003 by subcutaneous (SC) injection",[24],"Drug: SRP-1003 SC Injection",{"label":26,"type":16,"description":27,"interventionNames":28},"Placebo by SC Injection","Single or multiple doses of placebo by SC injection",[29],"Drug: Placebo SC Injection",[31,37,42,47],{"type":32,"name":9,"description":33,"armGroupLabels":34,"otherNames":35},"DRUG","SRP-1003 by IV infusion",[9],[36],"ARO-DM1 IV Infusion",{"type":32,"name":38,"description":39,"armGroupLabels":40,"otherNames":41},"Placebo IV Infusion","0.9% sodium chloride (NaCl) calculated volume to match active treatment by IV infusion",[15],null,{"type":32,"name":21,"description":43,"armGroupLabels":44,"otherNames":45},"SRP-1003 by SC injection(s)",[21],[46],"ARO-DM1 SC Injection",{"type":32,"name":48,"description":49,"armGroupLabels":50,"otherNames":41},"Placebo SC Injection","0.9% NaCl calculated volume to match active treatment by SC injection(s)",[26],[52],{"name":53,"affiliation":5,"role":54},"Medical Director","STUDY_DIRECTOR",[56],{"name":57,"role":58,"phone":59,"phoneExt":41,"email":60},"Sarepta Therapeutics Inc. For Clinical Trial Information, Select Option 4","CONTACT","1-888-SAREPTA (1-888-727-3782)","SareptAlly@sarepta.com",[62,76,86,94,104,109,117,127,135,145,153,161,171,179,187,195,203,213,221,231,239,247,255,266,276,284,292,303,311,316,328,337,346,356,364],{"facility":63,"status":64,"city":65,"state":66,"zip":67,"country":68,"countryCode":69,"cosmosGeoPoint":70,"geoPoint":75,"contacts":41},"Liverpool Hospital","RECRUITING","Liverpool","New South Wales","2170","Australia","AU",{"type":71,"coordinates":72},"Point",[73,74],150.92588,-33.91938,{"lat":74,"lon":73},{"facility":77,"status":64,"city":78,"state":79,"zip":80,"country":68,"countryCode":69,"cosmosGeoPoint":81,"geoPoint":85,"contacts":41},"Sunshine Coast University Hospital","Birtinya","Queensland","4575",{"type":71,"coordinates":82},[83,84],153.11913,-26.74322,{"lat":84,"lon":83},{"facility":87,"status":64,"city":88,"state":79,"zip":41,"country":68,"countryCode":69,"cosmosGeoPoint":89,"geoPoint":93,"contacts":41},"Royal Brisbane And Women's Hospital","Herston",{"type":71,"coordinates":90},[91,92],153.01852,-27.44453,{"lat":92,"lon":91},{"facility":95,"status":64,"city":96,"state":97,"zip":98,"country":68,"countryCode":69,"cosmosGeoPoint":99,"geoPoint":103,"contacts":41},"Alfred Health","Melbourne","Victoria","3004",{"type":71,"coordinates":100},[101,102],144.96332,-37.814,{"lat":102,"lon":101},{"facility":105,"status":64,"city":88,"state":41,"zip":41,"country":68,"countryCode":69,"cosmosGeoPoint":106,"geoPoint":108,"contacts":41},"Nucleus Network Brisbane Clinic",{"type":71,"coordinates":107},[91,92],{"lat":92,"lon":91},{"facility":110,"status":64,"city":111,"state":41,"zip":41,"country":68,"countryCode":69,"cosmosGeoPoint":112,"geoPoint":116,"contacts":41},"Mater Hospital Brisbane","South Brisbane",{"type":71,"coordinates":113},[114,115],153.02049,-27.48034,{"lat":115,"lon":114},{"facility":118,"status":64,"city":119,"state":41,"zip":41,"country":120,"countryCode":121,"cosmosGeoPoint":122,"geoPoint":126,"contacts":41},"Universitair Ziekenhuis Gent","Ghent","Belgium","BE",{"type":71,"coordinates":123},[124,125],3.71667,51.05,{"lat":125,"lon":124},{"facility":128,"status":64,"city":129,"state":41,"zip":41,"country":120,"countryCode":121,"cosmosGeoPoint":130,"geoPoint":134,"contacts":41},"UZ Leuven","Leuven",{"type":71,"coordinates":131},[132,133],4.70093,50.87959,{"lat":133,"lon":132},{"facility":136,"status":64,"city":137,"state":41,"zip":41,"country":138,"countryCode":139,"cosmosGeoPoint":140,"geoPoint":144,"contacts":41},"University Of Calgary","Calgary","Canada","CA",{"type":71,"coordinates":141},[142,143],-114.08529,51.05011,{"lat":143,"lon":142},{"facility":146,"status":64,"city":147,"state":41,"zip":41,"country":138,"countryCode":139,"cosmosGeoPoint":148,"geoPoint":152,"contacts":41},"University Of Alberta","Edmonton",{"type":71,"coordinates":149},[150,151],-113.46871,53.55014,{"lat":151,"lon":150},{"facility":154,"status":64,"city":155,"state":41,"zip":41,"country":138,"countryCode":139,"cosmosGeoPoint":156,"geoPoint":160,"contacts":41},"McGill University","Montreal",{"type":71,"coordinates":157},[158,159],-73.58781,45.50884,{"lat":159,"lon":158},{"facility":162,"status":64,"city":163,"state":41,"zip":41,"country":164,"countryCode":165,"cosmosGeoPoint":166,"geoPoint":170,"contacts":41},"University Hospital of Angers","Angers","France","FR",{"type":71,"coordinates":167},[168,169],-0.55202,47.47156,{"lat":169,"lon":168},{"facility":172,"status":64,"city":173,"state":41,"zip":41,"country":164,"countryCode":165,"cosmosGeoPoint":174,"geoPoint":178,"contacts":41},"Centre Hospitalier Universitaire De Bordeaux","Bordeaux",{"type":71,"coordinates":175},[176,177],-0.58046,44.84124,{"lat":177,"lon":176},{"facility":180,"status":64,"city":181,"state":41,"zip":41,"country":164,"countryCode":165,"cosmosGeoPoint":182,"geoPoint":186,"contacts":41},"Centre Hospitalier Universitaire De Lille","Lille",{"type":71,"coordinates":183},[184,185],3.05512,50.63391,{"lat":185,"lon":184},{"facility":188,"status":64,"city":189,"state":41,"zip":41,"country":164,"countryCode":165,"cosmosGeoPoint":190,"geoPoint":194,"contacts":41},"CHU de Nice - Hôpital Pasteur 2","Nice",{"type":71,"coordinates":191},[192,193],7.26608,43.70313,{"lat":193,"lon":192},{"facility":196,"status":64,"city":197,"state":41,"zip":41,"country":164,"countryCode":165,"cosmosGeoPoint":198,"geoPoint":202,"contacts":41},"Assistance Publique Hôpitaux De Paris","Paris",{"type":71,"coordinates":199},[200,201],2.3488,48.85341,{"lat":201,"lon":200},{"facility":204,"status":64,"city":205,"state":41,"zip":41,"country":206,"countryCode":207,"cosmosGeoPoint":208,"geoPoint":212,"contacts":41},"Klinikum der Universität München AöR","Munich","Germany","DE",{"type":71,"coordinates":209},[210,211],11.57549,48.13743,{"lat":211,"lon":210},{"facility":214,"status":64,"city":215,"state":41,"zip":41,"country":206,"countryCode":207,"cosmosGeoPoint":216,"geoPoint":220,"contacts":41},"Deutsches Zentrum für Neurodegenerative Erkrankungen e.V.","Ulm",{"type":71,"coordinates":217},[218,219],9.99155,48.39841,{"lat":219,"lon":218},{"facility":222,"status":64,"city":223,"state":41,"zip":41,"country":224,"countryCode":225,"cosmosGeoPoint":226,"geoPoint":230,"contacts":41},"Fondazione Serena Onlus - Centro Clinico Nemo Brescia","Gussago","Italy","IT",{"type":71,"coordinates":227},[228,229],10.15717,45.58358,{"lat":229,"lon":228},{"facility":232,"status":64,"city":233,"state":41,"zip":41,"country":224,"countryCode":225,"cosmosGeoPoint":234,"geoPoint":238,"contacts":41},"Centro Clinico Nemo (Milan)","Milan",{"type":71,"coordinates":235},[236,237],9.18951,45.46427,{"lat":237,"lon":236},{"facility":240,"status":64,"city":241,"state":41,"zip":41,"country":224,"countryCode":225,"cosmosGeoPoint":242,"geoPoint":246,"contacts":41},"Azienda Ospedaliera Universitaria Pisana (AOUP)","Pisa",{"type":71,"coordinates":243},[244,245],10.4036,43.70853,{"lat":245,"lon":244},{"facility":248,"status":64,"city":249,"state":41,"zip":41,"country":224,"countryCode":225,"cosmosGeoPoint":250,"geoPoint":254,"contacts":41},"Fondazione Policlinico Universitario Agostino Gemelli IRCCS","Roma",{"type":71,"coordinates":251},[252,253],11.10642,44.99364,{"lat":253,"lon":252},{"facility":256,"status":64,"city":257,"state":41,"zip":258,"country":259,"countryCode":260,"cosmosGeoPoint":261,"geoPoint":265,"contacts":41},"New Zealand Clinical Research Christchurch","Christchurch","8011","New Zealand","NZ",{"type":71,"coordinates":262},[263,264],172.63333,-43.53333,{"lat":264,"lon":263},{"facility":267,"status":64,"city":268,"state":41,"zip":41,"country":269,"countryCode":270,"cosmosGeoPoint":271,"geoPoint":275,"contacts":41},"Hospital Universitario Torrecárdenas","Almería","Spain","ES",{"type":71,"coordinates":272},[273,274],-2.45974,36.83814,{"lat":274,"lon":273},{"facility":277,"status":64,"city":278,"state":41,"zip":41,"country":269,"countryCode":270,"cosmosGeoPoint":279,"geoPoint":283,"contacts":41},"Hospital Universitari Vall D Hebron","Barcelona",{"type":71,"coordinates":280},[281,282],2.15899,41.38879,{"lat":282,"lon":281},{"facility":285,"status":64,"city":286,"state":41,"zip":41,"country":269,"countryCode":270,"cosmosGeoPoint":287,"geoPoint":291,"contacts":41},"Hospital Universitario Infanta Sofia","Madrid",{"type":71,"coordinates":288},[289,290],-3.70256,40.4165,{"lat":290,"lon":289},{"facility":293,"status":64,"city":294,"state":41,"zip":295,"country":296,"countryCode":297,"cosmosGeoPoint":298,"geoPoint":302,"contacts":41},"China Medical University Hospital","Taichung","40447","Taiwan","TW",{"type":71,"coordinates":299},[300,301],120.6839,24.1469,{"lat":301,"lon":300},{"facility":304,"status":64,"city":305,"state":41,"zip":41,"country":296,"countryCode":297,"cosmosGeoPoint":306,"geoPoint":310,"contacts":41},"National Taiwan University Hospital","Taipei",{"type":71,"coordinates":307},[308,309],121.52639,25.05306,{"lat":309,"lon":308},{"facility":312,"status":64,"city":305,"state":41,"zip":41,"country":296,"countryCode":297,"cosmosGeoPoint":313,"geoPoint":315,"contacts":41},"Taipei Veterans General Hospital",{"type":71,"coordinates":314},[308,309],{"lat":309,"lon":308},{"facility":317,"status":64,"city":318,"state":319,"zip":320,"country":321,"countryCode":322,"cosmosGeoPoint":323,"geoPoint":327,"contacts":41},"Songklanagarind Hospital","Hat Yai","Changwat Songkhla","90110","Thailand","TH",{"type":71,"coordinates":324},[325,326],100.47668,7.00836,{"lat":326,"lon":325},{"facility":329,"status":64,"city":330,"state":41,"zip":331,"country":321,"countryCode":322,"cosmosGeoPoint":332,"geoPoint":336,"contacts":41},"Siriraj Hospital","Bangkok","10700",{"type":71,"coordinates":333},[334,335],100.50144,13.75398,{"lat":335,"lon":334},{"facility":338,"status":64,"city":339,"state":41,"zip":340,"country":321,"countryCode":322,"cosmosGeoPoint":341,"geoPoint":345,"contacts":41},"Lampang Hospital","Lampang","52000",{"type":71,"coordinates":342},[343,344],99.49277,18.29232,{"lat":344,"lon":343},{"facility":347,"status":64,"city":348,"state":41,"zip":41,"country":349,"countryCode":350,"cosmosGeoPoint":351,"geoPoint":355,"contacts":41},"The National Hospital for Neurology and Neurosurgery, NHS Foundation Trust","London","United Kingdom","UK",{"type":71,"coordinates":352},[353,354],-0.12574,51.50853,{"lat":354,"lon":353},{"facility":357,"status":64,"city":358,"state":41,"zip":41,"country":349,"countryCode":350,"cosmosGeoPoint":359,"geoPoint":363,"contacts":41},"Nottingham University Hospitals NHS Trust","Nottingham",{"type":71,"coordinates":360},[361,362],-1.15047,52.9536,{"lat":362,"lon":361},{"facility":365,"status":64,"city":366,"state":41,"zip":41,"country":349,"countryCode":350,"cosmosGeoPoint":367,"geoPoint":371,"contacts":41},"Southampton General Hospital, NHS Foundation Trust","Southampton",{"type":71,"coordinates":368},[369,370],-1.40428,50.90395,{"lat":370,"lon":369},{"type":373,"investigatorFullName":41,"investigatorTitle":41,"investigatorAffiliation":41,"oldNameTitle":41,"oldOrganization":41},"SPONSOR","100526745","phase-1-study-of-aro-dm1-in-subjects-with-type-1-myotonic-dystrophy-100526745",false,"NCT06138743","Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy","A Phase 1\u002F2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DM1 (SRP-1003) in Subjects With Type 1 Myotonic Dystrophy Who Are ≥18 to ≤ 65 Years","Key Inclusion Criteria:\n\n* Genetically confirmed diagnosis of DM1\n* Clinician-assessed signs of DM1 including clinically apparent myotonia\n* Onset of DM1 symptoms occurred after the age of 12 years\n* Walk for at least 10 meters independently at screening\n* Participants of childbearing potential must agree to use highly effective contraception in addition to a condom during the study and for at least 90 days following the end of study or last dose of study drug, whichever is later. Participants must not donate sperm or eggs during the study and for at least 90 days following the end of study or last dose of study drug whichever is later.\n\nKey Exclusion Criteria:\n\n* Inadequately controlled diabetes\n* Confirmed diagnosis of congenital DM1\n* Uncontrolled hypertension\n* History of tibialis anterior (TA) biopsy within 3 months of Day 1 or planning to undergo TA biopsies during the study period\n* Clinically significant cardiac, liver or renal disease\n* Human immunodeficiency virus infection (seropositive) at screening\n* Seropositive for hepatitis B or hepatitis C at screening\n* Untreated or poorly controlled epilepsy\n* Treatment with anti-myotonia medication within a period of 5 half-lives of the medication prior to screening.\n* Abnormal coagulation parameters at screening including platelet count, international normalized ratio, prothrombin time, and activated partial thromboplastin time\n\nNote: Additional inclusion\u002Fexclusion criteria may apply per protocol","ALL","18 Years","65 Years",{"count":385,"type":386},78,"ESTIMATED","INTERVENTIONAL",[389,390],"PHASE1","PHASE2","This is a phase 1\u002F2a double-blinded, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of single and multiple ascending doses of SRP-1003 compared to placebo in male and female participants with type 1 myotonic dystrophy (DM1). Participants who have provided written informed consent and met all protocol eligibility requirements will be randomized to receive single (Part 1) or multiple (Part 2) doses of SRP-1003 or placebo.",[393],"Myotonic Dystrophy 1",[393,395,396],"SRP-1003","ARO-DM1","2026-08-07",{"date":399,"type":400},"2026-08-11","ACTUAL",{"date":402,"type":400},"2024-03-04",{"date":404,"type":386},"2026-12-31",{"name":5,"class":6},35]