[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100439579":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":20,"centralContacts":19,"locations":25,"responsibleParty":244,"collaborators":19,"id":246,"slug":247,"hasResults":248,"nctId":249,"briefTitle":250,"officialTitle":251,"acronym":252,"eligibilityCriteria":253,"healthyVolunteers":248,"sex":254,"minAge":255,"maxAge":256,"enrollmentInfo":257,"targetDuration":19,"studyType":260,"phases":261,"briefSummary":264,"conditions":265,"keywords":267,"overallStatus":28,"whyStopped":19,"lastUpdateSubmitDate":277,"lastUpdatePostDateStruct":278,"startDateStruct":281,"completionDateStruct":283,"leadSponsor":285,"locationsCount":286},{"fullName":5,"class":6},"AMO Pharma Limited","INDUSTRY",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"Tideglusib","EXPERIMENTAL","Weight adjusted or weight banded tideglusib, orally, once daily",[13],"Drug: Tideglusib",[15],{"type":16,"name":9,"description":17,"armGroupLabels":18,"otherNames":19},"DRUG","Tideglusib dosing will be weight-adjusted at 400 mg, 600 mg, or 1000 mg dose levels, or weight banded fixed doses of 400 mg, 600 mg, 800 mg or 1000 mg, with each subject starting at a weight-adjusted 400 mg dose level for 2 weeks, then up titrating to a weight-adjusted 600 mg dose level for the next 2 weeks.",[9],null,[21],{"name":22,"affiliation":23,"role":24},"Harriet Gray-Stephens, BM BCh, MA (Oxon), MFPM","AMO Pharma","STUDY_DIRECTOR",[26,52,63,72,90,108,126,145,163,174,184,204,216,233],{"facility":27,"status":28,"city":29,"state":30,"zip":31,"country":32,"countryCode":33,"cosmosGeoPoint":34,"geoPoint":39,"contacts":40},"Arkansas Children's Hospital","RECRUITING","Little Rock","Arkansas","72202","United States","US",{"type":35,"coordinates":36},"Point",[37,38],-92.28959,34.74648,{"lat":38,"lon":37},[41,46,49],{"name":42,"role":43,"phone":44,"phoneExt":19,"email":45},"Annette Guy","CONTACT","501-364-3380","GuyEA@archildrens.org",{"name":47,"role":43,"phone":19,"phoneExt":19,"email":48},"Caitlyn Daley","caitlyn.daley@reachcdm.com",{"name":50,"role":51,"phone":19,"phoneExt":19,"email":19},"Aravindhan Veerapandiyan, MD","PRINCIPAL_INVESTIGATOR",{"facility":53,"status":54,"city":55,"state":56,"zip":57,"country":32,"countryCode":33,"cosmosGeoPoint":58,"geoPoint":62,"contacts":19},"University of California, Los Angeles (UCLA)","ENROLLING_BY_INVITATION","Los Angeles","California","90095",{"type":35,"coordinates":59},[60,61],-118.24368,34.05223,{"lat":61,"lon":60},{"facility":64,"status":54,"city":65,"state":56,"zip":66,"country":32,"countryCode":33,"cosmosGeoPoint":67,"geoPoint":71,"contacts":19},"Stanford University","Palo Alto","94304",{"type":35,"coordinates":68},[69,70],-122.14302,37.44188,{"lat":70,"lon":69},{"facility":73,"status":28,"city":74,"state":75,"zip":76,"country":32,"countryCode":33,"cosmosGeoPoint":77,"geoPoint":81,"contacts":82},"Lurie's Children's Hospital","Chicago","Illinois","60611",{"type":35,"coordinates":78},[79,80],-87.65005,41.85003,{"lat":80,"lon":79},[83,87,88],{"name":84,"role":43,"phone":85,"phoneExt":19,"email":86},"Joseph Alberts","312-227-4449","jalberts@luriechildrens.org",{"name":47,"role":43,"phone":19,"phoneExt":19,"email":48},{"name":89,"role":51,"phone":19,"phoneExt":19,"email":19},"Nancy Kuntz, MD",{"facility":91,"status":28,"city":92,"state":93,"zip":94,"country":32,"countryCode":33,"cosmosGeoPoint":95,"geoPoint":99,"contacts":100},"University of Iowa Hospitals and Clinics","Iowa City","Iowa","52242",{"type":35,"coordinates":96},[97,98],-91.53017,41.66113,{"lat":98,"lon":97},[101,105,106],{"name":102,"role":43,"phone":103,"phoneExt":19,"email":104},"Laura Knosp","319-384-6277","laura-knosp@uiowa.edu",{"name":47,"role":43,"phone":19,"phoneExt":19,"email":48},{"name":107,"role":51,"phone":19,"phoneExt":19,"email":19},"Katherine Mathews, MD",{"facility":109,"status":28,"city":110,"state":111,"zip":112,"country":32,"countryCode":33,"cosmosGeoPoint":113,"geoPoint":117,"contacts":118},"University of Rochester - Medical Center","Rochester","New York","14642",{"type":35,"coordinates":114},[115,116],-77.61556,43.15478,{"lat":116,"lon":115},[119,123,124],{"name":120,"role":43,"phone":121,"phoneExt":19,"email":122},"James Hilbert","585-273-5590","james_hilbert@URMC.Rochester.eud",{"name":47,"role":43,"phone":19,"phoneExt":19,"email":48},{"name":125,"role":51,"phone":19,"phoneExt":19,"email":19},"Bo Hoon Lee, MD",{"facility":127,"status":28,"city":128,"state":129,"zip":130,"country":32,"countryCode":33,"cosmosGeoPoint":131,"geoPoint":135,"contacts":136},"University of Pittsburgh Medical Center","Pittsburgh","Pennsylvania","15213",{"type":35,"coordinates":132},[133,134],-79.99589,40.44062,{"lat":134,"lon":133},[137,141,143],{"name":138,"role":43,"phone":139,"phoneExt":19,"email":140},"Heather DiCostanzo","412-692-7660","follhl@upmc.edu",{"name":47,"role":43,"phone":19,"phoneExt":19,"email":142},"catilyn.daley@reachcdm.com",{"name":144,"role":51,"phone":19,"phoneExt":19,"email":19},"Hoda Abdel-Hamid, MD",{"facility":146,"status":28,"city":147,"state":148,"zip":149,"country":32,"countryCode":33,"cosmosGeoPoint":150,"geoPoint":154,"contacts":155},"University of Utah Clinical Neurosciences Center","Salt Lake City","Utah","84132",{"type":35,"coordinates":151},[152,153],-111.89105,40.76078,{"lat":153,"lon":152},[156,160,161],{"name":157,"role":43,"phone":158,"phoneExt":19,"email":159},"Domink May","801-587-4864","dominik.may@hsc.utah.edu",{"name":47,"role":43,"phone":19,"phoneExt":19,"email":142},{"name":162,"role":51,"phone":19,"phoneExt":19,"email":19},"Stephanie Manberg, DO",{"facility":164,"status":165,"city":166,"state":167,"zip":168,"country":32,"countryCode":33,"cosmosGeoPoint":169,"geoPoint":173,"contacts":19},"Children's Hospital of The King's Daughters","WITHDRAWN","Norfolk","Virginia","23507",{"type":35,"coordinates":170},[171,172],-76.28522,36.84681,{"lat":172,"lon":171},{"facility":175,"status":176,"city":177,"state":167,"zip":178,"country":32,"countryCode":33,"cosmosGeoPoint":179,"geoPoint":183,"contacts":19},"Virginia Commonwealth University-Department of Neurology - Muscular Dystrophy Translational Research Program","COMPLETED","Richmond","23219",{"type":35,"coordinates":180},[181,182],-77.46026,37.55376,{"lat":182,"lon":181},{"facility":185,"status":28,"city":186,"state":187,"zip":188,"country":189,"countryCode":190,"cosmosGeoPoint":191,"geoPoint":195,"contacts":196},"The Bright Alliance","Randwick","New South Wales","2031","Australia","AU",{"type":35,"coordinates":192},[193,194],151.24895,-33.91439,{"lat":194,"lon":193},[197,201,202],{"name":198,"role":43,"phone":199,"phoneExt":19,"email":200},"AMO Study Coordinator","02 9382 5534","SCHN-SCHClinicalTrials@health.nsw.gov.au",{"name":47,"role":43,"phone":19,"phoneExt":19,"email":48},{"name":203,"role":51,"phone":19,"phoneExt":19,"email":19},"Michelle Farrar,, MD",{"facility":205,"status":54,"city":206,"state":207,"zip":208,"country":209,"countryCode":210,"cosmosGeoPoint":211,"geoPoint":215,"contacts":19},"Children's Hospital London Health Sciences Centre (LHSC)","London","Ontario","N6A 4G5","Canada","CA",{"type":35,"coordinates":212},[213,214],-81.23304,42.98339,{"lat":214,"lon":213},{"facility":217,"status":28,"city":218,"state":207,"zip":219,"country":209,"countryCode":210,"cosmosGeoPoint":220,"geoPoint":224,"contacts":225},"Children's Hospital of Eastern Ontario","Ottawa","K1H 8L1",{"type":35,"coordinates":221},[222,223],-75.69812,45.41117,{"lat":223,"lon":222},[226,230,231],{"name":227,"role":43,"phone":228,"phoneExt":19,"email":229},"Emilie Hill-Smith","613-737-7600 x4017","ehillsmith@cheo.on.ca",{"name":47,"role":43,"phone":19,"phoneExt":19,"email":48},{"name":232,"role":51,"phone":19,"phoneExt":19,"email":19},"Hanns Lochmüller, MD",{"facility":234,"status":176,"city":235,"state":19,"zip":236,"country":237,"countryCode":238,"cosmosGeoPoint":239,"geoPoint":243,"contacts":19},"New Zealand Clinical Research (NZCR)","Auckland","1010","New Zealand","NZ",{"type":35,"coordinates":240},[241,242],174.76349,-36.84853,{"lat":242,"lon":241},{"type":245,"investigatorFullName":19,"investigatorTitle":19,"investigatorAffiliation":19,"oldNameTitle":19,"oldOrganization":19},"SPONSOR","100439579","phase-2-safety-and-efficacy-of-tideglusib-in-congenital-or-childhood-onset-myotonic-dystrophy-100439579",false,"NCT05004129","Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy","An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Tideglusib for the Treatment of Congenital or Childhood Onset DM1 (REACH CDM X)","REACH CDM X","Inclusion Criteria:\n\nSubjects who do not enter this study directly from completing the AMO-02-MD-2-003 study (i.e. subjects who did not complete AMO-02-MD-2-003, subjects who completed AMO-02-MD-2-003 but did not directly rollover or subjects who are re-entering AMO-02-MD-2-004), will not be considered eligible for the study without meeting all of the criteria below:\n\n1. Subjects under study must be individuals with a diagnosis of Congenital or Childhood Onset DM1.\n2. Diagnosis must be genetically confirmed\n3. Subjects must be male or female aged ≥6 years to ≤45 years at Screening\n4. Subjects must have a Clinical Global Impression - Severity (CGI-S) score of 3 or greater at Screening (V-1)\n5. Written, voluntary informed consent must be obtained before any study related procedures are conducted. Where a parent or legally authorized representative (LAR) provides consent, there must also be assent from the subject (as required by local regulations)\n6. Subject's caregiver must be willing and able to support participation for duration of study\n7. Subject must be willing and able to comply with the required food intake restrictions as outlined per protocol\n\nSubjects entering directly from completing the antecedent AMO-02-MD-2-003 study will not be considered eligible for the study without meeting all of the criteria below:\n\n1. Subjects who have completed the antecedent AMO-02-MD-2-003 study through V11\n2. Written, voluntary informed consent must be obtained before any study related procedures are conducted. Where a parent or LAR provides consent, there must also be assent from the subject (as required by local regulations)\n3. Subject's caregiver must be willing and able to support participation for duration of study\n4. Subject must be willing and able to comply with the required food intake restrictions as outlined per protocol\n\nKey Exclusion Criteria:\n\n1. Body mass index (BMI) less than 13.5 kg\u002Fm² or greater than 40 kg\u002Fm²\n2. New or change in medications\u002Ftherapies within 4 weeks prior to Eligibility\u002FBaseline Visit\n3. Use within 4 weeks prior to Eligibility\u002FBaseline Visit of strong CYP3A4 inhibitors (eg.clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole, nefazodone, idinavir and ritonavir)\n4. Concurrent use of drugs metabolized by CYP3A4 with a narrow therapeutic window (e.g. warfarin and digitoxin)\n5. Current enrollment in a clinical trial of an investigational drug or enrollment in a clinical trial of an investigational drug in the last 6 months other than the AMO-02- MD-2-003 study\n6. Existing or historical medical conditions or complications (eg. neurological, cardiovascular, renal, hepatic, gastrointestinal, endocrine or respiratory disease) that may impact the interpretability of the study results\n7. Hypersensitivity to tideglusib or any components of its formulation including allergy to strawberry","ALL","6 Years","45 Years",{"count":258,"type":259},76,"ESTIMATED","INTERVENTIONAL",[262,263],"PHASE2","PHASE3","This is an open-label phase 2\u002F3 study for individuals with Congenital Myotonic Dystrophy (Congenital DM1) who participated in the preceding AMO-02-MD-2-003 study or individuals with either Congenital or Childhood Onset DM1 who are treatment naïve.",[266],"Congenital Myotonic Dystrophy",[9,268,266,269,270,271,272,273,274,275,276],"AMO-02-MD-2-004","Myotonic Dystrophy","Dystrophia Myotonica","Myotonia Atrophica","Myotonia Dystrophica","Myotonic Dystrophy, Congenital","Steinert Disease","Steinert Myotonic Dystrophy","Steinert's Disease","2025-05-22",{"date":279,"type":280},"2025-05-28","ACTUAL",{"date":282,"type":280},"2021-08-23",{"date":284,"type":259},"2026-12-31",{"name":5,"class":6},14]