[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100653553":3},{"organization":4,"outcomesModule":7,"designInfo":31,"detailedDescription":11,"studyPopulation":11,"armGroups":37,"interventions":44,"overallOfficials":49,"centralContacts":53,"locations":11,"responsibleParty":59,"collaborators":11,"id":61,"slug":62,"hasResults":63,"nctId":64,"briefTitle":65,"officialTitle":66,"acronym":11,"eligibilityCriteria":67,"healthyVolunteers":63,"sex":68,"minAge":69,"maxAge":11,"enrollmentInfo":70,"targetDuration":11,"studyType":73,"phases":74,"briefSummary":76,"conditions":77,"keywords":79,"overallStatus":90,"whyStopped":11,"lastUpdateSubmitDate":91,"lastUpdatePostDateStruct":92,"startDateStruct":95,"completionDateStruct":97,"leadSponsor":99,"locationsCount":11},{"fullName":5,"class":6},"Shionogi Inc.","INDUSTRY",{"primaryOutcomes":8,"secondaryOutcomes":13,"otherOutcomes":11},[9],{"measure":10,"description":11,"timeFrame":12},"Number of Participants with Treatment-emergent Adverse Events",null,"Baseline (Day 1) through Week 49",[14,17,19,21,23,25,28],{"measure":15,"description":11,"timeFrame":16},"Change From Baseline in Percent Forced Vital Capacity (%FVC)","Baseline (Day 1), Week 48",{"measure":18,"description":11,"timeFrame":16},"Change From Baseline in Maximum Expiratory Capacity (MEP)",{"measure":20,"description":11,"timeFrame":16},"Change From Baseline in Minimum Inspiratory Capacity (MIP)",{"measure":22,"description":11,"timeFrame":16},"Change From Baseline in 6-minute Walk Test (6MWT)",{"measure":24,"description":11,"timeFrame":16},"Change From Baseline in Gait, Stair, Gower's Maneuver, and Chair (GSGC) Score",{"measure":26,"description":11,"timeFrame":27},"Change from Baseline in Daily Living Activity Levels as Assessed by the Investigator","Baseline (2-4 weeks prior to Day 1), Week 48",{"measure":29,"description":11,"timeFrame":30},"Plasma Concentration of S-606001 and Metabolites","Up to Week 28",{"allocation":32,"interventionModel":33,"interventionModelDescription":11,"primaryPurpose":34,"observationalModel":11,"timePerspective":11,"maskingInfo":35},"NA","SINGLE_GROUP","TREATMENT",{"masking":36,"maskingDescription":11,"whoMasked":11},"NONE",[38],{"label":39,"type":40,"description":41,"interventionNames":42},"S-606001","EXPERIMENTAL","Participants will receive S-606001.",[43],"Drug: S-606001",[45],{"type":46,"name":39,"description":47,"armGroupLabels":48,"otherNames":11},"DRUG","S-606001 will be administered orally as a tablet.",[39],[50],{"name":51,"affiliation":5,"role":52},"Medical Director","STUDY_DIRECTOR",[54],{"name":55,"role":56,"phone":57,"phoneExt":11,"email":58},"Shionogi Clinical Trials Administrator Clinical Support Help Line","CONTACT","1-800-849-9707","Shionogiclintrials-admin@shionogi.co.jp",{"type":60,"investigatorFullName":11,"investigatorTitle":11,"investigatorAffiliation":11,"oldNameTitle":11,"oldOrganization":11},"SPONSOR","100653553","phase-1-a-study-of-s-606001-as-monotherapy-in-participants-with-late-onset-pompe-disease-lopd-100653553",false,"NCT07787650","A Study of S-606001 as Monotherapy in Participants With Late-onset Pompe Disease (LOPD)","A Phase 1b, Multicenter, Single-arm, Open-label Study to Investigate the Safety, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of S-606001 as a Monotherapy in Adult Patients With Late-onset Pompe Disease","Key Inclusion Criteria:\n\n* Participant must be ≥40 kilograms of body weight at the time of signing the informed consent form\n* Participant must have a diagnosis of LOPD based on documentation of the following: deficiency of acid alpha-glucosidase (GAA) enzyme; GAA genotype\n* Participant has a %FVC ≥30% and ≤80% in an upright position without mechanical ventilation at screening or participant has \\>80% FVC in upright position and ≥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: screening values of 6-minute walk distance (6MWD) are ≥75 meters; screening values of 6MWD are ≤90% of the predicted value for healthy adults\n* Participant must not have received enzyme replacement therapy (ERT) for at least 3 months prior to providing informed consent and enrollment and agrees not receive any type of ERT while participating in the study\n\nKey Exclusion Criteria:\n\n* Has a medical condition or any other extenuating circumstance that may, in the opinion of the investigator or medical monitor, 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 the muscle glycogen synthase gene or in the glycogen phosphorylase muscle associated gene\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 for Pompe disease within 2 years of screening or small interfering RNA therapy for Pompe disease within 6 months of screening\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":71,"type":72},15,"ESTIMATED","INTERVENTIONAL",[75],"PHASE1","The primary purpose of this study is to evaluate the safety and tolerability profile of S-606001 in participants with LOPD.",[78],"Pompe Disease",[39,80,81,82,83,84,85,86,87,88,89],"Late-onset Pompe Disease","LOPD","Muscle glycogen synthase","Liver glycogen synthase","Rare disease","Autosomal disease","Acid alpha-glucosidase","GAA","Glycogen storage disorder","GSD","NOT_YET_RECRUITING","2026-08-24",{"date":93,"type":94},"2026-08-26","ACTUAL",{"date":96,"type":72},"2026-09-30",{"date":98,"type":72},"2028-10-31",{"name":100,"class":6},"Shionogi"]