[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100476280":3},{"organization":4,"armGroups":7,"interventions":44,"overallOfficials":50,"centralContacts":54,"locations":60,"responsibleParty":393,"collaborators":50,"id":395,"slug":396,"hasResults":397,"nctId":398,"briefTitle":399,"officialTitle":400,"acronym":401,"eligibilityCriteria":402,"healthyVolunteers":397,"sex":403,"minAge":404,"maxAge":405,"enrollmentInfo":406,"targetDuration":50,"studyType":409,"phases":410,"briefSummary":413,"conditions":414,"keywords":50,"overallStatus":63,"whyStopped":50,"lastUpdateSubmitDate":416,"lastUpdatePostDateStruct":417,"startDateStruct":420,"completionDateStruct":422,"leadSponsor":424,"locationsCount":425},{"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",[61,82,98,114,130,146,162,178,197,215,232,247,264,282,299,314,330,347,364,378],{"facility":62,"status":63,"city":64,"state":65,"zip":66,"country":67,"countryCode":68,"cosmosGeoPoint":69,"geoPoint":74,"contacts":75},"Stanford University","RECRUITING","Stanford","California","94305","United States","US",{"type":70,"coordinates":71},"Point",[72,73],-122.16608,37.42411,{"lat":73,"lon":72},[76,79],{"name":77,"role":57,"phone":50,"phoneExt":50,"email":78},"Lidia Choniawko","lidiacho@stanford.edu",{"name":80,"role":81,"phone":50,"phoneExt":50,"email":50},"Dr. John Day, MD, PhD","PRINCIPAL_INVESTIGATOR",{"facility":83,"status":63,"city":84,"state":85,"zip":86,"country":67,"countryCode":68,"cosmosGeoPoint":87,"geoPoint":91,"contacts":92},"University of Florida College of Medicine","Gainesville","Florida","32610",{"type":70,"coordinates":88},[89,90],-82.32483,29.65163,{"lat":90,"lon":89},[93,96],{"name":94,"role":57,"phone":50,"phoneExt":50,"email":95},"Debbye Landers","debralanders@peds.ufl.edu",{"name":97,"role":81,"phone":50,"phoneExt":50,"email":50},"Dr. Sub Subramony, MD",{"facility":99,"status":63,"city":100,"state":101,"zip":102,"country":67,"countryCode":68,"cosmosGeoPoint":103,"geoPoint":107,"contacts":108},"Indiana University School of Medicine","Indianapolis","Indiana","46202",{"type":70,"coordinates":104},[105,106],-86.15804,39.76838,{"lat":106,"lon":105},[109,112],{"name":110,"role":57,"phone":50,"phoneExt":50,"email":111},"Patti Hogan, BSN RN, CCRA","hoganpr@iu.edu",{"name":113,"role":81,"phone":50,"phoneExt":50,"email":50},"Laurie Gutmann, MD",{"facility":115,"status":63,"city":116,"state":117,"zip":118,"country":67,"countryCode":68,"cosmosGeoPoint":119,"geoPoint":123,"contacts":124},"University of Iowa","Iowa City","Iowa","52242",{"type":70,"coordinates":120},[121,122],-91.53017,41.66113,{"lat":122,"lon":121},[125,128],{"name":126,"role":57,"phone":50,"phoneExt":50,"email":127},"Heena Olade, RN, MSN","heena-olalde@uiowa.edu",{"name":129,"role":81,"phone":50,"phoneExt":50,"email":50},"Andrea Swenson, MD",{"facility":131,"status":63,"city":132,"state":133,"zip":134,"country":67,"countryCode":68,"cosmosGeoPoint":135,"geoPoint":139,"contacts":140},"Washington University in St. Louis","St Louis","Missouri","63110",{"type":70,"coordinates":136},[137,138],-90.19789,38.62727,{"lat":138,"lon":137},[141,144],{"name":142,"role":57,"phone":50,"phoneExt":50,"email":143},"Natalie Goedeker","ngoedeker@wustl.edu",{"name":145,"role":81,"phone":50,"phoneExt":50,"email":50},"Dr. Craig Zaidman, MD",{"facility":147,"status":63,"city":148,"state":149,"zip":150,"country":67,"countryCode":68,"cosmosGeoPoint":151,"geoPoint":155,"contacts":156},"University of Rochester Medical Center","Rochester","New York","14642",{"type":70,"coordinates":152},[153,154],-77.61556,43.15478,{"lat":154,"lon":153},[157,160],{"name":158,"role":57,"phone":50,"phoneExt":50,"email":159},"Jim Hilbert","james_hilbert@urmc.rochester.edu",{"name":161,"role":81,"phone":50,"phoneExt":50,"email":50},"Dr. Joahnna Hamel, MD",{"facility":163,"status":63,"city":164,"state":165,"zip":166,"country":67,"countryCode":68,"cosmosGeoPoint":167,"geoPoint":171,"contacts":172},"Neurology Rare Disease Center","Denton","Texas","76208",{"type":70,"coordinates":168},[169,170],-97.13307,33.21484,{"lat":170,"lon":169},[173,176],{"name":174,"role":57,"phone":50,"phoneExt":50,"email":175},"Isabella Herman","Research@neuromdcenter.com",{"name":177,"role":81,"phone":50,"phoneExt":50,"email":50},"Diana Castro, MD",{"facility":179,"status":63,"city":180,"state":181,"zip":182,"country":67,"countryCode":68,"cosmosGeoPoint":183,"geoPoint":187,"contacts":188},"Virginia Commonwealth University (VCU)","Richmond","Virginia","23298",{"type":70,"coordinates":184},[185,186],-77.46026,37.55376,{"lat":186,"lon":185},[189,192,195],{"name":190,"role":57,"phone":50,"phoneExt":50,"email":191},"Jodie Howell","Jodie.howell@vcuhealth.org",{"name":193,"role":57,"phone":50,"phoneExt":50,"email":194},"Anarosa Rezeq","anarosa.rezeq@vcuhealth.org",{"name":196,"role":81,"phone":50,"phoneExt":50,"email":50},"Nicholas Johnson, M.D., M.Sci., FAAN",{"facility":198,"status":63,"city":199,"state":200,"zip":201,"country":202,"countryCode":203,"cosmosGeoPoint":204,"geoPoint":208,"contacts":209},"St. Vincent's Hospital","Fitzroy","Victoria","3065","Australia","AU",{"type":70,"coordinates":205},[206,207],144.97833,-37.79839,{"lat":207,"lon":206},[210,213],{"name":211,"role":57,"phone":50,"phoneExt":50,"email":212},"Alan Lai","alan.lai@svha.org.au",{"name":214,"role":81,"phone":50,"phoneExt":50,"email":50},"Dr. Gayatri Jain",{"facility":216,"status":63,"city":217,"state":50,"zip":218,"country":219,"countryCode":220,"cosmosGeoPoint":221,"geoPoint":225,"contacts":226},"CHU de Nantes","Nantes","44093","France","FR",{"type":70,"coordinates":222},[223,224],-1.55336,47.21725,{"lat":224,"lon":223},[227,230],{"name":228,"role":57,"phone":50,"phoneExt":50,"email":229},"Marie-Agnès Sourdril","marieagnes.sourdril@chu-nantes.fr",{"name":231,"role":81,"phone":50,"phoneExt":50,"email":50},"Yann Péréon, MD, PhD",{"facility":233,"status":63,"city":234,"state":50,"zip":235,"country":219,"countryCode":220,"cosmosGeoPoint":236,"geoPoint":240,"contacts":241},"Institut de Myologie","Paris","75013",{"type":70,"coordinates":237},[238,239],2.3488,48.85341,{"lat":239,"lon":238},[242,245],{"name":243,"role":57,"phone":50,"phoneExt":50,"email":244},"Asma Balloumi","a.balloumi@institut-myologie.org",{"name":246,"role":81,"phone":50,"phoneExt":50,"email":50},"Dr. Guillaume Bassez",{"facility":248,"status":63,"city":249,"state":50,"zip":250,"country":251,"countryCode":252,"cosmosGeoPoint":253,"geoPoint":257,"contacts":258},"Charité - Universitätsmedizin Berlin","Berlin","10117","Germany","DE",{"type":70,"coordinates":254},[255,256],13.41053,52.52437,{"lat":256,"lon":255},[259,262],{"name":260,"role":57,"phone":50,"phoneExt":50,"email":261},"Susanne Behr-Perst","susanne.behr-perst@charite.de",{"name":263,"role":81,"phone":50,"phoneExt":50,"email":50},"Elisabetta Gazzerro, MD, PhD",{"facility":265,"status":63,"city":266,"state":50,"zip":267,"country":251,"countryCode":252,"cosmosGeoPoint":268,"geoPoint":272,"contacts":273},"Ludwig Maximilians University, Munich - Friedrich Baur Institut","Munich","80336",{"type":70,"coordinates":269},[270,271],11.57549,48.13743,{"lat":271,"lon":270},[274,277,279],{"name":275,"role":57,"phone":50,"phoneExt":50,"email":276},"Corinna Wirner-Piotrowski","Corinna.Wirner@med.uni-muenchen.de",{"name":278,"role":81,"phone":50,"phoneExt":50,"email":50},"Benedikt Schoser, MD",{"name":280,"role":281,"phone":50,"phoneExt":50,"email":50},"Stephan Wenninger, MD","SUB_INVESTIGATOR",{"facility":283,"status":63,"city":284,"state":50,"zip":285,"country":286,"countryCode":287,"cosmosGeoPoint":288,"geoPoint":292,"contacts":293},"Centro Clinico Nemo","Milan","20162","Italy","IT",{"type":70,"coordinates":289},[290,291],9.18951,45.46427,{"lat":291,"lon":290},[294,297],{"name":295,"role":57,"phone":50,"phoneExt":50,"email":296},"Maribel Evoli","maribel.evoli@centrocliniconemo.it",{"name":298,"role":81,"phone":50,"phoneExt":50,"email":50},"Valeria Sansone, MD, PhD",{"facility":300,"status":63,"city":301,"state":50,"zip":302,"country":286,"countryCode":287,"cosmosGeoPoint":303,"geoPoint":307,"contacts":308},"Fondazione Policlinico Universitario A Gemelli-Rome","Rome","00168",{"type":70,"coordinates":304},[305,306],12.51133,41.89193,{"lat":306,"lon":305},[309,312],{"name":310,"role":57,"phone":50,"phoneExt":50,"email":311},"Maria Pirozzoli","mariaceleste.pirozzoli@policlinicogemelli.it",{"name":313,"role":81,"phone":50,"phoneExt":50,"email":50},"Marika Pane",{"facility":315,"status":63,"city":316,"state":50,"zip":50,"country":317,"countryCode":318,"cosmosGeoPoint":319,"geoPoint":323,"contacts":324},"Radboud Medical Center","Nijmegen","Netherlands","NL",{"type":70,"coordinates":320},[321,322],5.85278,51.8425,{"lat":322,"lon":321},[325,328],{"name":326,"role":57,"phone":50,"phoneExt":50,"email":327},"Eline Sanders","clinicalresearchunit@radboudumc.nl",{"name":329,"role":81,"phone":50,"phoneExt":50,"email":50},"Karien Mul",{"facility":331,"status":63,"city":332,"state":50,"zip":333,"country":334,"countryCode":335,"cosmosGeoPoint":336,"geoPoint":340,"contacts":341},"NZCR Auckland","Auckland","1023","New Zealand","NZ",{"type":70,"coordinates":337},[338,339],174.76349,-36.84853,{"lat":339,"lon":338},[342,345],{"name":343,"role":57,"phone":50,"phoneExt":50,"email":344},"Miriam Rodrigues","MRodrigues@adhb.govt.nz",{"name":346,"role":81,"phone":50,"phoneExt":50,"email":50},"Dr. Richard Roxburgh",{"facility":348,"status":63,"city":349,"state":50,"zip":350,"country":351,"countryCode":352,"cosmosGeoPoint":353,"geoPoint":357,"contacts":358},"University College London Hospitals","London","NW1 2BU","United Kingdom","UK",{"type":70,"coordinates":354},[355,356],-0.12574,51.50853,{"lat":356,"lon":355},[359,362],{"name":360,"role":57,"phone":50,"phoneExt":50,"email":361},"Nikoletta Nikolenko","n.nikolenko@nhs.net",{"name":363,"role":81,"phone":50,"phoneExt":50,"email":50},"Dr. Chris Turner",{"facility":365,"status":63,"city":366,"state":50,"zip":50,"country":351,"countryCode":352,"cosmosGeoPoint":367,"geoPoint":371,"contacts":372},"John Walton Muscular Dystrophy Research Centre","Newcastle upon Tyne",{"type":70,"coordinates":368},[369,370],-1.61396,54.97328,{"lat":370,"lon":369},[373,376],{"name":374,"role":57,"phone":50,"phoneExt":50,"email":375},"Nicola McLarty","nicola.mclarty1@nhs.net",{"name":377,"role":81,"phone":50,"phoneExt":50,"email":50},"Jordi Diaz-Manera",{"facility":379,"status":63,"city":380,"state":50,"zip":381,"country":351,"countryCode":352,"cosmosGeoPoint":382,"geoPoint":386,"contacts":387},"Salford Royal Hospital","Salford","M6 8HD",{"type":70,"coordinates":383},[384,385],-2.29042,53.48771,{"lat":385,"lon":384},[388,391],{"name":389,"role":57,"phone":50,"phoneExt":50,"email":390},"Bethan Blackledge","Bethan.blackledge@nca.nhs.uk",{"name":392,"role":81,"phone":50,"phoneExt":50,"email":50},"James Lilleker",{"type":394,"investigatorFullName":50,"investigatorTitle":50,"investigatorAffiliation":50,"oldNameTitle":50,"oldOrganization":50},"SPONSOR","100476280","phase-1-safety-tolerability-pharmacodynamic-efficacy-and-pharmacokinetic-study-of-dyne-101-in-participants-with-myotonic-dystrophy-type-1-100476280",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":407,"type":408},116,"ESTIMATED","INTERVENTIONAL",[411,412],"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.",[415],"Myotonic Dystrophy Type 1 (DM1)","2026-05-08",{"date":418,"type":419},"2026-05-12","ACTUAL",{"date":421,"type":419},"2022-09-05",{"date":423,"type":408},"2029-07",{"name":5,"class":6},20]