[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100602411":3},{"organization":4,"armGroups":7,"interventions":24,"overallOfficials":30,"centralContacts":34,"locations":40,"responsibleParty":286,"collaborators":30,"id":288,"slug":30,"hasResults":289,"nctId":290,"briefTitle":291,"officialTitle":292,"acronym":30,"eligibilityCriteria":293,"healthyVolunteers":289,"sex":294,"minAge":295,"maxAge":30,"enrollmentInfo":296,"targetDuration":30,"studyType":299,"phases":300,"briefSummary":302,"conditions":303,"keywords":305,"overallStatus":43,"whyStopped":30,"lastUpdateSubmitDate":318,"lastUpdatePostDateStruct":319,"startDateStruct":322,"completionDateStruct":324,"leadSponsor":326,"locationsCount":328},{"fullName":5,"class":6},"Shionogi Inc.","INDUSTRY",[8,14,18],{"label":9,"type":10,"description":11,"interventionNames":12},"S-606001 Low Dose","EXPERIMENTAL","Participants will receive S-606001 at a low dose level twice daily (BID) after a meal for 52 weeks.",[13],"Drug: S-606001",{"label":15,"type":10,"description":16,"interventionNames":17},"S-606001 High Dose","Participants will receive S-606001 at a high dose level BID after a meal for 52 weeks.",[13],{"label":19,"type":20,"description":21,"interventionNames":22},"Placebo","PLACEBO_COMPARATOR","Participants will receive S-606001 matching placebo BID after a meal for 52 weeks.",[23],"Drug: Placebo",[25,31],{"type":26,"name":27,"description":28,"armGroupLabels":29,"otherNames":30},"DRUG","S-606001","S-606001 administered orally",[15,9],null,{"type":26,"name":19,"description":32,"armGroupLabels":33,"otherNames":30},"S-606001 matching placebo administered orally",[19],[35],{"name":36,"role":37,"phone":38,"phoneExt":30,"email":39},"Shionogi Clinical Trials Administrator Clinical Support Help Line","CONTACT","800-849-9707","Shionogiclintrials-admin@shionogi.co.jp",[41,54,64,74,84,94,104,114,123,133,142,151,160,168,176,185,194,202,210,219,227,231,240,248,257,265,270,278],{"facility":42,"status":43,"city":44,"state":45,"zip":46,"country":47,"cosmosGeoPoint":48,"geoPoint":53,"contacts":30},"University of California - Irvine Medical Center","RECRUITING","Irvine","California","92868","United States",{"type":49,"coordinates":50},"Point",[51,52],-117.82311,33.66946,{"lat":52,"lon":51},{"facility":55,"status":43,"city":56,"state":57,"zip":58,"country":47,"cosmosGeoPoint":59,"geoPoint":63,"contacts":30},"University of Florida (UF) - Gainesville","Gainesville","Florida","32611",{"type":49,"coordinates":60},[61,62],-82.32483,29.65163,{"lat":62,"lon":61},{"facility":65,"status":43,"city":66,"state":67,"zip":68,"country":47,"cosmosGeoPoint":69,"geoPoint":73,"contacts":30},"Emory University Hospital","Atlanta","Georgia","30322",{"type":49,"coordinates":70},[71,72],-84.38798,33.749,{"lat":72,"lon":71},{"facility":75,"status":43,"city":76,"state":77,"zip":78,"country":47,"cosmosGeoPoint":79,"geoPoint":83,"contacts":30},"Washington University in St. Louis","St Louis","Missouri","63130",{"type":49,"coordinates":80},[81,82],-90.19789,38.62727,{"lat":82,"lon":81},{"facility":85,"status":43,"city":86,"state":87,"zip":88,"country":47,"cosmosGeoPoint":89,"geoPoint":93,"contacts":30},"Duke University Medical Center","Durham","North Carolina","27710",{"type":49,"coordinates":90},[91,92],-78.89862,35.99403,{"lat":92,"lon":91},{"facility":95,"status":43,"city":96,"state":97,"zip":98,"country":47,"cosmosGeoPoint":99,"geoPoint":103,"contacts":30},"Cincinnati Children's Hospital Medical Center","Cincinnati","Ohio","45229",{"type":49,"coordinates":100},[101,102],-84.51439,39.12711,{"lat":102,"lon":101},{"facility":105,"status":43,"city":106,"state":107,"zip":108,"country":47,"cosmosGeoPoint":109,"geoPoint":113,"contacts":30},"University of Pennsylvania","Philadelphia","Pennsylvania","19104",{"type":49,"coordinates":110},[111,112],-75.16362,39.95238,{"lat":112,"lon":111},{"facility":115,"status":43,"city":116,"state":107,"zip":117,"country":47,"cosmosGeoPoint":118,"geoPoint":122,"contacts":30},"University of Pittsburgh School of Medicine","Pittsburgh","15213",{"type":49,"coordinates":119},[120,121],-79.99589,40.44062,{"lat":121,"lon":120},{"facility":124,"status":43,"city":125,"state":126,"zip":127,"country":47,"cosmosGeoPoint":128,"geoPoint":132,"contacts":30},"Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)","Fairfax","Virginia","22030",{"type":49,"coordinates":129},[130,131],-77.30637,38.84622,{"lat":131,"lon":130},{"facility":134,"status":43,"city":135,"state":30,"zip":30,"country":136,"cosmosGeoPoint":137,"geoPoint":141,"contacts":30},"UZ Leuven","Leuven","Belgium",{"type":49,"coordinates":138},[139,140],4.70093,50.87959,{"lat":140,"lon":139},{"facility":143,"status":43,"city":144,"state":30,"zip":30,"country":145,"cosmosGeoPoint":146,"geoPoint":150,"contacts":30},"Aarhus University Hospital","Aarhus","Denmark",{"type":49,"coordinates":147},[148,149],10.21076,56.15674,{"lat":149,"lon":148},{"facility":152,"status":43,"city":153,"state":30,"zip":30,"country":154,"cosmosGeoPoint":155,"geoPoint":159,"contacts":30},"HLC Hopital Pierre Wertheimer","Bron","France",{"type":49,"coordinates":156},[157,158],4.91303,45.73865,{"lat":158,"lon":157},{"facility":161,"status":43,"city":162,"state":30,"zip":30,"country":154,"cosmosGeoPoint":163,"geoPoint":167,"contacts":30},"AP-HP Hopital Raymond Poincare","Garches",{"type":49,"coordinates":164},[165,166],2.18232,48.84226,{"lat":166,"lon":165},{"facility":169,"status":43,"city":170,"state":30,"zip":30,"country":154,"cosmosGeoPoint":171,"geoPoint":175,"contacts":30},"Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone","Marseille",{"type":49,"coordinates":172},[173,174],5.38107,43.29695,{"lat":174,"lon":173},{"facility":177,"status":178,"city":179,"state":30,"zip":30,"country":154,"cosmosGeoPoint":180,"geoPoint":184,"contacts":30},"CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires","NOT_YET_RECRUITING","Nice",{"type":49,"coordinates":181},[182,183],7.26608,43.70313,{"lat":183,"lon":182},{"facility":186,"status":43,"city":187,"state":30,"zip":30,"country":188,"cosmosGeoPoint":189,"geoPoint":193,"contacts":30},"Universitaetsklinikum Halle (Saale)","Halle","Germany",{"type":49,"coordinates":190},[191,192],11.97947,51.48158,{"lat":192,"lon":191},{"facility":195,"status":43,"city":196,"state":30,"zip":30,"country":188,"cosmosGeoPoint":197,"geoPoint":201,"contacts":30},"SphinCS GmbH","Höchheim",{"type":49,"coordinates":198},[199,200],10.45,50.36667,{"lat":200,"lon":199},{"facility":203,"status":43,"city":204,"state":30,"zip":30,"country":188,"cosmosGeoPoint":205,"geoPoint":209,"contacts":30},"Klinikum der Ludwig-Maximilians-Universitaet Muenchen","München",{"type":49,"coordinates":206},[207,208],13.31243,51.60698,{"lat":208,"lon":207},{"facility":211,"status":43,"city":212,"state":30,"zip":30,"country":213,"cosmosGeoPoint":214,"geoPoint":218,"contacts":30},"A.O.U. Policlinico \"G. Martino\"","Messina","Italy",{"type":49,"coordinates":215},[216,217],15.55256,38.19394,{"lat":217,"lon":216},{"facility":220,"status":43,"city":221,"state":30,"zip":30,"country":213,"cosmosGeoPoint":222,"geoPoint":226,"contacts":30},"AOU Citta della Salute e della Scienza di Torino - Ospedale le Molinette","Torino",{"type":49,"coordinates":223},[224,225],11.99138,44.88856,{"lat":225,"lon":224},{"facility":228,"status":43,"city":229,"state":229,"zip":30,"country":230,"cosmosGeoPoint":30,"geoPoint":30,"contacts":30},"Erasmus MC","GE Rotterdam","Netherlands",{"facility":232,"status":43,"city":233,"state":30,"zip":30,"country":234,"cosmosGeoPoint":235,"geoPoint":239,"contacts":30},"Hospital Universitario 12 de Octubre","Madrid","Spain",{"type":49,"coordinates":236},[237,238],-3.70256,40.4165,{"lat":238,"lon":237},{"facility":241,"status":43,"city":242,"state":30,"zip":30,"country":234,"cosmosGeoPoint":243,"geoPoint":247,"contacts":30},"Hospital Universitari i Politecnic La Fe","Valencia",{"type":49,"coordinates":244},[245,246],-0.37966,39.47391,{"lat":246,"lon":245},{"facility":249,"status":43,"city":250,"state":30,"zip":30,"country":251,"cosmosGeoPoint":252,"geoPoint":256,"contacts":30},"Queen Elizabeth Hospital Birmingham","Birmingham","United Kingdom",{"type":49,"coordinates":253},[254,255],-1.89983,52.48142,{"lat":255,"lon":254},{"facility":258,"status":43,"city":259,"state":30,"zip":30,"country":251,"cosmosGeoPoint":260,"geoPoint":264,"contacts":30},"National Hospital for Neurology & Neurosurgery","London",{"type":49,"coordinates":261},[262,263],-0.12574,51.50853,{"lat":263,"lon":262},{"facility":266,"status":43,"city":259,"state":30,"zip":30,"country":251,"cosmosGeoPoint":267,"geoPoint":269,"contacts":30},"Royal Free London NHS Foundation Trust",{"type":49,"coordinates":268},[262,263],{"lat":263,"lon":262},{"facility":271,"status":43,"city":272,"state":30,"zip":30,"country":251,"cosmosGeoPoint":273,"geoPoint":277,"contacts":30},"Royal Victoria Infirmary","Newcastle upon Tyne",{"type":49,"coordinates":274},[275,276],-1.61396,54.97328,{"lat":276,"lon":275},{"facility":279,"status":43,"city":280,"state":30,"zip":30,"country":251,"cosmosGeoPoint":281,"geoPoint":285,"contacts":30},"Salford Royal Hospital","Salford",{"type":49,"coordinates":282},[283,284],-2.29042,53.48771,{"lat":284,"lon":283},{"type":287,"investigatorFullName":30,"investigatorTitle":30,"investigatorAffiliation":30,"oldNameTitle":30,"oldOrganization":30},"SPONSOR","100602411",false,"NCT07123155","Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)","A Phase 2, Multicenter, Randomized, Placebo-controlled, Double-blind Study to Investigate the Safety, Pharmacodynamics, and Preliminary Efficacy of S-606001 as an Add-on to Enzyme Replacement Therapy in Patients With Late-onset Pompe Disease","Key Inclusion Criteria:\n\n* Participant must be ≥18 years of age and ≥40 kilograms (kg) of body weight at the time of signing the informed consent.\n* Participant must have a diagnosis of LOPD based on documentation of 1 of the following:\n\n  1. Deficiency of acid alpha-glucosidase (GAA) enzyme\n  2. GAA genotype\n* Participant has a %FVC ≥30% and ≤80% in an upright position without mechanical ventilation at screening; or Participant has ≥10% %FVC drop from upright position to supine position and %FVC ≥20% in a supine position.\n* Participant performs the 6MWT at screening, as determined by the clinical evaluator, and meets all of the following criteria:\n\n  1. Screening values of 6-minute walk distance (6MWD) are ≥75 meters\n  2. Screening values of 6MWD are ≤90% of the predicted value for healthy adults\n* Participants must be ERT-experienced, defined as currently receiving ERT and having been receiving ERT for ≥24 months, with no regimen change in the last 6 months.\n\nKey Exclusion Criteria:\n\n* Has a medical condition or any other extenuating circumstance that may pose an undue safety risk to the participant or may compromise his\u002Fher ability to comply with or adversely impact protocol requirements.\n* Has active infections at screening.\n* Malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 3 years.\n* Current or chronic history of liver disease.\n* Known biallelic loss of function mutations whether in glycogenin gene (GYG) or in glycogen phosphorylase muscle associated gene(PYGM) .\n* Has received any investigational therapy or pharmacological treatment for Pompe disease, within 30 days or 5 half-lives of the therapy or treatment, whichever is longer, before day 1 or is anticipated to do so during the study.\n* Has received gene therapy or small interfering ribonucleic acid (RNA) therapy for Pompe disease.\n* Participant, if female, is pregnant or breastfeeding at screening.\n* Participant, whether male or female, is planning to conceive a child during the study.\n\nNote: Other protocol-specified inclusion and exclusion criteria may apply.","ALL","18 Years",{"count":297,"type":298},45,"ESTIMATED","INTERVENTIONAL",[301],"PHASE2","The purpose of this study is to evaluate the safety, pharmacodynamics (PD), and exploratory clinical efficacy of S-606001 in adult participants with LOPD as an add-on to ERT.",[304],"Pompe Disease",[306,307,308,309,27,310,311,312,313,314,315,316,317],"Late-onset Pompe disease","Enzyme replacement therapy","LOPD","ERT","Muscle glycogen synthase","Liver glycogen synthase","Rare disease","Autosomal disease","Acid alpha-glucosidase","GAA","Glycogen storage disorder","GSD","2026-06-19",{"date":320,"type":321},"2026-06-23","ACTUAL",{"date":323,"type":321},"2025-10-30",{"date":325,"type":298},"2027-08-08",{"name":327,"class":6},"Shionogi",28]