[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT07123155":3,"trial-entities:NCT07123155":306,"trial-summary:NCT07123155":311},{"id":4,"nct_id":4,"org_study_id":5,"brief_title":6,"official_title":7,"overall_status":8,"completion_date":9,"status_verified_date":10,"last_update_date":11,"start_date":12,"sponsor_name":13,"lead_sponsor_class":14,"has_dmc":15,"brief_summary":16,"detailed_description":17,"conditions":18,"keywords":20,"study_type":34,"primary_purpose":35,"phases":36,"enrollment_info":38,"interventions":41,"primary_outcomes":52,"secondary_outcomes":56,"sex":83,"minimum_age":84,"maximum_age":17,"healthy_volunteers":85,"eligibility_criteria":86,"std_ages":104,"locations":107,"central_contacts":297,"overall_officials":303,"references":304,"see_also_links":305},"NCT07123155","2405N1221","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","RECRUITING","2027-08-08","2026-06","2026-06-23","2025-10-30","Shionogi","INDUSTRY",true,"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.",null,[19],"Pompe Disease",[21,22,23,24,25,26,27,28,29,30,31,32,33],"Late-onset Pompe disease","Enzyme replacement therapy","LOPD","ERT","S-606001","Muscle glycogen synthase","Liver glycogen synthase","Rare disease","Autosomal disease","Acid alpha-glucosidase","GAA","Glycogen storage disorder","GSD","INTERVENTIONAL","TREATMENT",[37],"PHASE2",{"count":39,"type":40},45,"ESTIMATED",[42,48],{"type":43,"name":25,"description":44,"armGroupLabels":45},"DRUG","S-606001 administered orally",[46,47],"S-606001 High Dose","S-606001 Low Dose",{"type":43,"name":49,"description":50,"armGroupLabels":51},"Placebo","S-606001 matching placebo administered orally",[49],[53],{"measure":54,"timeFrame":55},"Change From Baseline in Percent Forced Vital Capacity (%FVC) at Week 52","Baseline, Week 52",[57,60,63,65,67,69,71,73,75,77,79,81],{"measure":58,"timeFrame":59},"Number of Participants With Treatment-emergent Adverse Events (TEAEs)","Baseline up to Week 53",{"measure":61,"timeFrame":62},"Plasma Concentration of S-606001","Up to Week 12",{"measure":64,"timeFrame":55},"Change From Baseline in Serum Creatine Kinase Levels at Week 52",{"measure":66,"timeFrame":55},"Change From Baseline in 6-minute Walk Test (6MWT) at Week 52",{"measure":68,"timeFrame":55},"Change From Baseline in Pulmonary Function Parameter: Maximal Inspiratory Pressure (MIP) at Week 52",{"measure":70,"timeFrame":55},"Change From Baseline in Pulmonary Function Parameter: Maximal Expiratory Pressure (MEP) at Week 52",{"measure":72,"timeFrame":55},"Change From Baseline in Motor Function Parameter: Gait, Stair, Gower's Maneuver, Chair (GSGC) Score at Week 52",{"measure":74,"timeFrame":55},"Change From Baseline in Patient-Reported Outcomes Measurement Information System Fatigue Short Form 8a (PROMIS-Fatigue-8a) Score at Week 52",{"measure":76,"timeFrame":55},"Change From Baseline in Patient-Reported Outcomes Measurement Information System Physical Function 20-item short form (PROMIS-PF-20) Score at Week 52",{"measure":78,"timeFrame":55},"Change From Baseline in Patient-Reported Outcomes Measurement Information System v2.0 Pain Intensity 3a (PROMIS v2.0 PAIN) Score at Week 52",{"measure":80,"timeFrame":55},"Change From Baseline in 36-item Short Form Health Survey (SF-36) Score at Week 52",{"measure":82,"timeFrame":55},"Change From Baseline in Patient Global Impression of Severity (PGI-S) Score at Week 52","ALL","18 Years",false,{"inclusion":87,"exclusion":93,"raw_text":103},[88,89,90,91,92],"Participant must be ≥18 years of age and ≥40 kilograms (kg) of body weight at the time of signing the informed consent.","Participant must have a diagnosis of LOPD based on documentation of 1 of the following:","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.","Participant performs the 6MWT at screening, as determined by the clinical evaluator, and meets all of the following criteria:","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.",[94,95,96,97,98,99,100,101,102],"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.","Has active infections at screening.","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.","Current or chronic history of liver disease.","Known biallelic loss of function mutations whether in glycogenin gene (GYG) or in glycogen phosphorylase muscle associated gene(PYGM) .","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.","Has received gene therapy or small interfering ribonucleic acid (RNA) therapy for Pompe disease.","Participant, if female, is pregnant or breastfeeding at screening.","Participant, whether male or female, is planning to conceive a child during the study.","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.",[105,106],"ADULT","OLDER_ADULT",[108,117,125,133,141,149,157,165,172,180,187,194,201,207,213,220,227,233,239,246,252,256,263,269,276,282,285,291],{"facility":109,"status":8,"city":110,"state":111,"zip":112,"country":113,"geoPoint":114},"University of California - Irvine Medical Center","Irvine","California","92868","United States",{"lat":115,"lon":116},33.66946,-117.82311,{"facility":118,"status":8,"city":119,"state":120,"zip":121,"country":113,"geoPoint":122},"University of Florida (UF) - Gainesville","Gainesville","Florida","32611",{"lat":123,"lon":124},29.65163,-82.32483,{"facility":126,"status":8,"city":127,"state":128,"zip":129,"country":113,"geoPoint":130},"Emory University Hospital","Atlanta","Georgia","30322",{"lat":131,"lon":132},33.749,-84.38798,{"facility":134,"status":8,"city":135,"state":136,"zip":137,"country":113,"geoPoint":138},"Washington University in St. Louis","St Louis","Missouri","63130",{"lat":139,"lon":140},38.62727,-90.19789,{"facility":142,"status":8,"city":143,"state":144,"zip":145,"country":113,"geoPoint":146},"Duke University Medical Center","Durham","North Carolina","27710",{"lat":147,"lon":148},35.99403,-78.89862,{"facility":150,"status":8,"city":151,"state":152,"zip":153,"country":113,"geoPoint":154},"Cincinnati Children's Hospital Medical Center","Cincinnati","Ohio","45229",{"lat":155,"lon":156},39.12711,-84.51439,{"facility":158,"status":8,"city":159,"state":160,"zip":161,"country":113,"geoPoint":162},"University of Pennsylvania","Philadelphia","Pennsylvania","19104",{"lat":163,"lon":164},39.95238,-75.16362,{"facility":166,"status":8,"city":167,"state":160,"zip":168,"country":113,"geoPoint":169},"University of Pittsburgh School of Medicine","Pittsburgh","15213",{"lat":170,"lon":171},40.44062,-79.99589,{"facility":173,"status":8,"city":174,"state":175,"zip":176,"country":113,"geoPoint":177},"Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)","Fairfax","Virginia","22030",{"lat":178,"lon":179},38.84622,-77.30637,{"facility":181,"status":8,"city":182,"country":183,"geoPoint":184},"UZ Leuven","Leuven","Belgium",{"lat":185,"lon":186},50.87959,4.70093,{"facility":188,"status":8,"city":189,"country":190,"geoPoint":191},"Aarhus University Hospital","Aarhus","Denmark",{"lat":192,"lon":193},56.15674,10.21076,{"facility":195,"status":8,"city":196,"country":197,"geoPoint":198},"HLC Hopital Pierre Wertheimer","Bron","France",{"lat":199,"lon":200},45.73865,4.91303,{"facility":202,"status":8,"city":203,"country":197,"geoPoint":204},"AP-HP Hopital Raymond Poincare","Garches",{"lat":205,"lon":206},48.84226,2.18232,{"facility":208,"status":8,"city":209,"country":197,"geoPoint":210},"Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone","Marseille",{"lat":211,"lon":212},43.29695,5.38107,{"facility":214,"status":215,"city":216,"country":197,"geoPoint":217},"CHU de Nice - Hopital Pasteur 2 - Centre de reference des Maladies Neuromusculaires","NOT_YET_RECRUITING","Nice",{"lat":218,"lon":219},43.70313,7.26608,{"facility":221,"status":8,"city":222,"country":223,"geoPoint":224},"Universitaetsklinikum Halle (Saale)","Halle","Germany",{"lat":225,"lon":226},51.48158,11.97947,{"facility":228,"status":8,"city":229,"country":223,"geoPoint":230},"SphinCS GmbH","Höchheim",{"lat":231,"lon":232},50.36667,10.45,{"facility":234,"status":8,"city":235,"country":223,"geoPoint":236},"Klinikum der Ludwig-Maximilians-Universitaet Muenchen","München",{"lat":237,"lon":238},51.60698,13.31243,{"facility":240,"status":8,"city":241,"country":242,"geoPoint":243},"A.O.U. Policlinico \"G. Martino\"","Messina","Italy",{"lat":244,"lon":245},38.19394,15.55256,{"facility":247,"status":8,"city":248,"country":242,"geoPoint":249},"AOU Citta della Salute e della Scienza di Torino - Ospedale le Molinette","Torino",{"lat":250,"lon":251},44.88856,11.99138,{"facility":253,"status":8,"city":254,"state":254,"country":255},"Erasmus MC","GE Rotterdam","Netherlands",{"facility":257,"status":8,"city":258,"country":259,"geoPoint":260},"Hospital Universitario 12 de Octubre","Madrid","Spain",{"lat":261,"lon":262},40.4165,-3.70256,{"facility":264,"status":8,"city":265,"country":259,"geoPoint":266},"Hospital Universitari i Politecnic La Fe","Valencia",{"lat":267,"lon":268},39.47391,-0.37966,{"facility":270,"status":8,"city":271,"country":272,"geoPoint":273},"Queen Elizabeth Hospital Birmingham","Birmingham","United Kingdom",{"lat":274,"lon":275},52.48142,-1.89983,{"facility":277,"status":8,"city":278,"country":272,"geoPoint":279},"National Hospital for Neurology & Neurosurgery","London",{"lat":280,"lon":281},51.50853,-0.12574,{"facility":283,"status":8,"city":278,"country":272,"geoPoint":284},"Royal Free London NHS Foundation Trust",{"lat":280,"lon":281},{"facility":286,"status":8,"city":287,"country":272,"geoPoint":288},"Royal Victoria Infirmary","Newcastle upon Tyne",{"lat":289,"lon":290},54.97328,-1.61396,{"facility":292,"status":8,"city":293,"country":272,"geoPoint":294},"Salford Royal Hospital","Salford",{"lat":295,"lon":296},53.48771,-2.29042,[298],{"name":299,"role":300,"phone":301,"email":302},"Shionogi Clinical Trials Administrator Clinical Support Help Line","CONTACT","800-849-9707","Shionogiclintrials-admin@shionogi.co.jp",[],[],[],{"nct_id":4,"conditions":307,"biomarkers":309},[308],"Glycogen storage disease due to acid maltase deficiency, late-onset",[310],"GLUCOSIDASE, ALPHA, ACID",{"nct_id":4,"found":15,"summary":312,"prompt_version":322},{"design":313,"status":314,"heading":315,"summary":316,"follow_up":317,"word_count":318,"commitments":319,"compensation":320,"drugs_mentioned":321},"This interventional study plans to enroll 45 participants. Some participants will receive S-606001, while others will receive a matching placebo.","completed","Study of S-606001 for Late-onset Pompe Disease","This study is testing S-606001, an oral medication, as an add-on treatment to enzyme replacement therapy (ERT) for adults with Late-onset Pompe Disease (LOPD). Researchers want to learn about the safety of S-606001, how it affects the body (pharmacodynamics), and if it helps improve lung function. You may be eligible if you are at least 18 years old, weigh at least 40 kg, and have a confirmed diagnosis of LOPD. The main way they will measure success is by looking at changes in your forced vital capacity (%FVC), which measures how much air you can exhale, after 52 weeks. The current recruitment status is unclear.","The primary endpoint is measured at Week 52, suggesting follow-up for at least this duration.",105,"Not specified in the trial record.","Not stated in the trial record.",[25],"v2"]