[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100476280":3},{"organization":4,"armGroups":7,"interventions":44,"overallOfficials":50,"centralContacts":54,"locations":60,"responsibleParty":385,"collaborators":50,"id":387,"slug":50,"hasResults":388,"nctId":389,"briefTitle":390,"officialTitle":391,"acronym":392,"eligibilityCriteria":393,"healthyVolunteers":388,"sex":394,"minAge":395,"maxAge":396,"enrollmentInfo":397,"targetDuration":50,"studyType":400,"phases":401,"briefSummary":404,"conditions":405,"keywords":50,"overallStatus":63,"whyStopped":50,"lastUpdateSubmitDate":407,"lastUpdatePostDateStruct":408,"startDateStruct":411,"completionDateStruct":413,"leadSponsor":415,"locationsCount":416},{"fullName":5,"class":6},"Dyne Therapeutics","INDUSTRY",[8,14,20,24,28,32,36,40],{"label":9,"type":10,"description":11,"interventionNames":12},"MAD Cohort: Placebo-Controlled Period: DYNE-101","EXPERIMENTAL","Participants will be randomized to receive ascending doses of DYNE-101, once every 4 weeks (Q4W) or once every 8 weeks (Q8W) for up to 24 weeks.",[13],"Drug: DYNE-101",{"label":15,"type":16,"description":17,"interventionNames":18},"MAD Cohort: Placebo-Controlled Period: Placebo","PLACEBO_COMPARATOR","Participants will be randomized to receive DYNE-101 matching placebo, Q4W or Q8W for up to 24 weeks.",[19],"Drug: Placebo",{"label":21,"type":10,"description":22,"interventionNames":23},"MAD Cohort: Treatment Period: DYNE-101","Participants who receive DYNE-101 in Placebo-Controlled Period will continue to receive DYNE-101, Q4W or Q8W for up to 24 weeks.\n\nParticipants who receive placebo in Placebo-Controlled Period will receive DYNE-101, Q4W or Q8W for up to 24 weeks.",[13,19],{"label":25,"type":10,"description":26,"interventionNames":27},"MAD Cohort: Long-Term Extension Period: DYNE-101","Participants will receive DYNE-101, Q4W or Q8W for up to 168 weeks.",[13],{"label":29,"type":10,"description":30,"interventionNames":31},"Dose Expansion Cohort: Placebo-Controlled Period: DYNE-101","Participants will receive DYNE-101, Q8W for up to 24 weeks.",[13],{"label":33,"type":10,"description":34,"interventionNames":35},"Dose Expansion Cohort: Placebo-Controlled Period: Placebo","Participants will receive DYNE-101 matching placebo, Q8W for up to 24 weeks.",[19],{"label":37,"type":10,"description":38,"interventionNames":39},"Dose Expansion Cohort: Treatment Period: DYNE-101","Participants who receive DYNE-101 in Placebo-Controlled Period will continue to receive DYNE-101, Q8W for up to 24 weeks.\n\nParticipants who receive placebo in Placebo-Controlled Period will receive DYNE-101, Q8W for up to 24 weeks.",[13,19],{"label":41,"type":10,"description":42,"interventionNames":43},"Dose Expansion Cohort: Long-Term Extension Period: DYNE-101","Participants will receive DYNE-101, Q8W for up to 168 weeks.",[13],[45,51],{"type":46,"name":47,"description":48,"armGroupLabels":49,"otherNames":50},"DRUG","DYNE-101","Administered by IV infusion",[41,29,37,25,9,21],null,{"type":46,"name":52,"description":48,"armGroupLabels":53,"otherNames":50},"Placebo",[33,37,15,21],[55],{"name":56,"role":57,"phone":58,"phoneExt":50,"email":59},"Dyne Clinical Trials","CONTACT","+1-781-317-1919","clinicaltrials@dyne-tx.com",[61,81,97,113,129,145,161,177,196,213,229,244,260,278,294,309,324,340,356,370],{"facility":62,"status":63,"city":64,"state":65,"zip":66,"country":67,"cosmosGeoPoint":68,"geoPoint":73,"contacts":74},"Stanford University","RECRUITING","Stanford","California","94305","United States",{"type":69,"coordinates":70},"Point",[71,72],-122.16608,37.42411,{"lat":72,"lon":71},[75,78],{"name":76,"role":57,"phone":50,"phoneExt":50,"email":77},"Lidia Choniawko","lidiacho@stanford.edu",{"name":79,"role":80,"phone":50,"phoneExt":50,"email":50},"Dr. John Day, MD, PhD","PRINCIPAL_INVESTIGATOR",{"facility":82,"status":63,"city":83,"state":84,"zip":85,"country":67,"cosmosGeoPoint":86,"geoPoint":90,"contacts":91},"University of Florida College of Medicine","Gainesville","Florida","32610",{"type":69,"coordinates":87},[88,89],-82.32483,29.65163,{"lat":89,"lon":88},[92,95],{"name":93,"role":57,"phone":50,"phoneExt":50,"email":94},"Debbye Landers","debralanders@peds.ufl.edu",{"name":96,"role":80,"phone":50,"phoneExt":50,"email":50},"Dr. Sub Subramony, MD",{"facility":98,"status":63,"city":99,"state":100,"zip":101,"country":67,"cosmosGeoPoint":102,"geoPoint":106,"contacts":107},"Indiana University School of Medicine","Indianapolis","Indiana","46202",{"type":69,"coordinates":103},[104,105],-86.15804,39.76838,{"lat":105,"lon":104},[108,111],{"name":109,"role":57,"phone":50,"phoneExt":50,"email":110},"Patti Hogan, BSN RN, CCRA","hoganpr@iu.edu",{"name":112,"role":80,"phone":50,"phoneExt":50,"email":50},"Laurie Gutmann, MD",{"facility":114,"status":63,"city":115,"state":116,"zip":117,"country":67,"cosmosGeoPoint":118,"geoPoint":122,"contacts":123},"University of Iowa","Iowa City","Iowa","52242",{"type":69,"coordinates":119},[120,121],-91.53017,41.66113,{"lat":121,"lon":120},[124,127],{"name":125,"role":57,"phone":50,"phoneExt":50,"email":126},"Heena Olade, RN, MSN","heena-olalde@uiowa.edu",{"name":128,"role":80,"phone":50,"phoneExt":50,"email":50},"Andrea Swenson, MD",{"facility":130,"status":63,"city":131,"state":132,"zip":133,"country":67,"cosmosGeoPoint":134,"geoPoint":138,"contacts":139},"Washington University in St. Louis","St Louis","Missouri","63110",{"type":69,"coordinates":135},[136,137],-90.19789,38.62727,{"lat":137,"lon":136},[140,143],{"name":141,"role":57,"phone":50,"phoneExt":50,"email":142},"Natalie Goedeker","ngoedeker@wustl.edu",{"name":144,"role":80,"phone":50,"phoneExt":50,"email":50},"Dr. Craig Zaidman, MD",{"facility":146,"status":63,"city":147,"state":148,"zip":149,"country":67,"cosmosGeoPoint":150,"geoPoint":154,"contacts":155},"University of Rochester Medical Center","Rochester","New York","14642",{"type":69,"coordinates":151},[152,153],-77.61556,43.15478,{"lat":153,"lon":152},[156,159],{"name":157,"role":57,"phone":50,"phoneExt":50,"email":158},"Jim Hilbert","james_hilbert@urmc.rochester.edu",{"name":160,"role":80,"phone":50,"phoneExt":50,"email":50},"Dr. Joahnna Hamel, MD",{"facility":162,"status":63,"city":163,"state":164,"zip":165,"country":67,"cosmosGeoPoint":166,"geoPoint":170,"contacts":171},"Neurology Rare Disease Center","Denton","Texas","76208",{"type":69,"coordinates":167},[168,169],-97.13307,33.21484,{"lat":169,"lon":168},[172,175],{"name":173,"role":57,"phone":50,"phoneExt":50,"email":174},"Isabella Herman","Research@neuromdcenter.com",{"name":176,"role":80,"phone":50,"phoneExt":50,"email":50},"Diana Castro, MD",{"facility":178,"status":63,"city":179,"state":180,"zip":181,"country":67,"cosmosGeoPoint":182,"geoPoint":186,"contacts":187},"Virginia Commonwealth University (VCU)","Richmond","Virginia","23298",{"type":69,"coordinates":183},[184,185],-77.46026,37.55376,{"lat":185,"lon":184},[188,191,194],{"name":189,"role":57,"phone":50,"phoneExt":50,"email":190},"Jodie Howell","Jodie.howell@vcuhealth.org",{"name":192,"role":57,"phone":50,"phoneExt":50,"email":193},"Anarosa Rezeq","anarosa.rezeq@vcuhealth.org",{"name":195,"role":80,"phone":50,"phoneExt":50,"email":50},"Nicholas Johnson, M.D., M.Sci., FAAN",{"facility":197,"status":63,"city":198,"state":199,"zip":200,"country":201,"cosmosGeoPoint":202,"geoPoint":206,"contacts":207},"St. Vincent's Hospital","Fitzroy","Victoria","3065","Australia",{"type":69,"coordinates":203},[204,205],144.97833,-37.79839,{"lat":205,"lon":204},[208,211],{"name":209,"role":57,"phone":50,"phoneExt":50,"email":210},"Alan Lai","alan.lai@svha.org.au",{"name":212,"role":80,"phone":50,"phoneExt":50,"email":50},"Dr. Gayatri Jain",{"facility":214,"status":63,"city":215,"state":50,"zip":216,"country":217,"cosmosGeoPoint":218,"geoPoint":222,"contacts":223},"CHU de Nantes","Nantes","44093","France",{"type":69,"coordinates":219},[220,221],-1.55336,47.21725,{"lat":221,"lon":220},[224,227],{"name":225,"role":57,"phone":50,"phoneExt":50,"email":226},"Marie-Agnès Sourdril","marieagnes.sourdril@chu-nantes.fr",{"name":228,"role":80,"phone":50,"phoneExt":50,"email":50},"Yann Péréon, MD, PhD",{"facility":230,"status":63,"city":231,"state":50,"zip":232,"country":217,"cosmosGeoPoint":233,"geoPoint":237,"contacts":238},"Institut de Myologie","Paris","75013",{"type":69,"coordinates":234},[235,236],2.3488,48.85341,{"lat":236,"lon":235},[239,242],{"name":240,"role":57,"phone":50,"phoneExt":50,"email":241},"Asma Balloumi","a.balloumi@institut-myologie.org",{"name":243,"role":80,"phone":50,"phoneExt":50,"email":50},"Dr. Guillaume Bassez",{"facility":245,"status":63,"city":246,"state":50,"zip":247,"country":248,"cosmosGeoPoint":249,"geoPoint":253,"contacts":254},"Charité - Universitätsmedizin Berlin","Berlin","10117","Germany",{"type":69,"coordinates":250},[251,252],13.41053,52.52437,{"lat":252,"lon":251},[255,258],{"name":256,"role":57,"phone":50,"phoneExt":50,"email":257},"Susanne Behr-Perst","susanne.behr-perst@charite.de",{"name":259,"role":80,"phone":50,"phoneExt":50,"email":50},"Elisabetta Gazzerro, MD, PhD",{"facility":261,"status":63,"city":262,"state":50,"zip":263,"country":248,"cosmosGeoPoint":264,"geoPoint":268,"contacts":269},"Ludwig Maximilians University, Munich - Friedrich Baur Institut","Munich","80336",{"type":69,"coordinates":265},[266,267],11.57549,48.13743,{"lat":267,"lon":266},[270,273,275],{"name":271,"role":57,"phone":50,"phoneExt":50,"email":272},"Corinna Wirner-Piotrowski","Corinna.Wirner@med.uni-muenchen.de",{"name":274,"role":80,"phone":50,"phoneExt":50,"email":50},"Benedikt Schoser, MD",{"name":276,"role":277,"phone":50,"phoneExt":50,"email":50},"Stephan Wenninger, MD","SUB_INVESTIGATOR",{"facility":279,"status":63,"city":280,"state":50,"zip":281,"country":282,"cosmosGeoPoint":283,"geoPoint":287,"contacts":288},"Centro Clinico Nemo","Milan","20162","Italy",{"type":69,"coordinates":284},[285,286],9.18951,45.46427,{"lat":286,"lon":285},[289,292],{"name":290,"role":57,"phone":50,"phoneExt":50,"email":291},"Maribel Evoli","maribel.evoli@centrocliniconemo.it",{"name":293,"role":80,"phone":50,"phoneExt":50,"email":50},"Valeria Sansone, MD, PhD",{"facility":295,"status":63,"city":296,"state":50,"zip":297,"country":282,"cosmosGeoPoint":298,"geoPoint":302,"contacts":303},"Fondazione Policlinico Universitario A Gemelli-Rome","Rome","00168",{"type":69,"coordinates":299},[300,301],12.51133,41.89193,{"lat":301,"lon":300},[304,307],{"name":305,"role":57,"phone":50,"phoneExt":50,"email":306},"Maria Pirozzoli","mariaceleste.pirozzoli@policlinicogemelli.it",{"name":308,"role":80,"phone":50,"phoneExt":50,"email":50},"Marika Pane",{"facility":310,"status":63,"city":311,"state":50,"zip":50,"country":312,"cosmosGeoPoint":313,"geoPoint":317,"contacts":318},"Radboud Medical Center","Nijmegen","Netherlands",{"type":69,"coordinates":314},[315,316],5.85278,51.8425,{"lat":316,"lon":315},[319,322],{"name":320,"role":57,"phone":50,"phoneExt":50,"email":321},"Eline Sanders","clinicalresearchunit@radboudumc.nl",{"name":323,"role":80,"phone":50,"phoneExt":50,"email":50},"Karien Mul",{"facility":325,"status":63,"city":326,"state":50,"zip":327,"country":328,"cosmosGeoPoint":329,"geoPoint":333,"contacts":334},"NZCR Auckland","Auckland","1023","New Zealand",{"type":69,"coordinates":330},[331,332],174.76349,-36.84853,{"lat":332,"lon":331},[335,338],{"name":336,"role":57,"phone":50,"phoneExt":50,"email":337},"Miriam Rodrigues","MRodrigues@adhb.govt.nz",{"name":339,"role":80,"phone":50,"phoneExt":50,"email":50},"Dr. Richard Roxburgh",{"facility":341,"status":63,"city":342,"state":50,"zip":343,"country":344,"cosmosGeoPoint":345,"geoPoint":349,"contacts":350},"University College London Hospitals","London","NW1 2BU","United Kingdom",{"type":69,"coordinates":346},[347,348],-0.12574,51.50853,{"lat":348,"lon":347},[351,354],{"name":352,"role":57,"phone":50,"phoneExt":50,"email":353},"Nikoletta Nikolenko","n.nikolenko@nhs.net",{"name":355,"role":80,"phone":50,"phoneExt":50,"email":50},"Dr. Chris Turner",{"facility":357,"status":63,"city":358,"state":50,"zip":50,"country":344,"cosmosGeoPoint":359,"geoPoint":363,"contacts":364},"John Walton Muscular Dystrophy Research Centre","Newcastle upon Tyne",{"type":69,"coordinates":360},[361,362],-1.61396,54.97328,{"lat":362,"lon":361},[365,368],{"name":366,"role":57,"phone":50,"phoneExt":50,"email":367},"Nicola McLarty","nicola.mclarty1@nhs.net",{"name":369,"role":80,"phone":50,"phoneExt":50,"email":50},"Jordi Diaz-Manera",{"facility":371,"status":63,"city":372,"state":50,"zip":373,"country":344,"cosmosGeoPoint":374,"geoPoint":378,"contacts":379},"Salford Royal Hospital","Salford","M6 8HD",{"type":69,"coordinates":375},[376,377],-2.29042,53.48771,{"lat":377,"lon":376},[380,383],{"name":381,"role":57,"phone":50,"phoneExt":50,"email":382},"Bethan Blackledge","Bethan.blackledge@nca.nhs.uk",{"name":384,"role":80,"phone":50,"phoneExt":50,"email":50},"James Lilleker",{"type":386,"investigatorFullName":50,"investigatorTitle":50,"investigatorAffiliation":50,"oldNameTitle":50,"oldOrganization":50},"SPONSOR","100476280",false,"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.","ALL","18 Years","65 Years",{"count":398,"type":399},116,"ESTIMATED","INTERVENTIONAL",[402,403],"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.",[406],"Myotonic Dystrophy Type 1 (DM1)","2026-05-08",{"date":409,"type":410},"2026-05-12","ACTUAL",{"date":412,"type":410},"2022-09-05",{"date":414,"type":399},"2029-07",{"name":5,"class":6},20]