[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100657002":3},{"organization":4,"outcomesModule":7,"designInfo":25,"detailedDescription":28,"studyPopulation":29,"armGroups":24,"interventions":24,"overallOfficials":30,"centralContacts":35,"locations":41,"responsibleParty":56,"collaborators":24,"id":59,"slug":60,"hasResults":61,"nctId":62,"briefTitle":63,"officialTitle":64,"acronym":24,"eligibilityCriteria":65,"healthyVolunteers":61,"sex":66,"minAge":24,"maxAge":24,"enrollmentInfo":67,"targetDuration":24,"studyType":70,"phases":24,"briefSummary":71,"conditions":72,"keywords":74,"overallStatus":43,"whyStopped":24,"lastUpdateSubmitDate":75,"lastUpdatePostDateStruct":76,"startDateStruct":79,"completionDateStruct":81,"leadSponsor":83,"locationsCount":84},{"fullName":5,"class":6},"National Taiwan University Hospital","OTHER",{"primaryOutcomes":8,"secondaryOutcomes":13,"otherOutcomes":24},[9],{"measure":10,"description":11,"timeFrame":12},"Urinary Glc4\u002FHex4","the primary endpoints for effectiveness analysis of avalglucosidase alfa. Urinary Glc4\u002FHex4 is usually elevated in Pompe patients and are sensitive though nonspecific biomarkers for diagnosis and monitoring of IOPD or LOPD","3years",[14,17,21],{"measure":15,"description":16,"timeFrame":12},"Creatine kinase","Serum CK is usually elevated in Pompe patients and are sensitive though nonspecific biomarkers for diagnosis and monitoring of IOPD or LOPD (Kishnani et al., 2006; Li et al., 2021; Young et al., 2009).",{"measure":18,"description":19,"timeFrame":20},"motor function (Quick Motor Function Test)","QMFT score is strongly correlated with proximal muscle strength, and significantly differentiated between patients with different levels of mobility (van Capelle et al., 2012), muscle function will be assessed by QMFT annually.","3 years",{"measure":22,"description":23,"timeFrame":20},"respiratory function (forced vital capacity).","Pulmonary function will be assessed by forced vital capacity. For infants, pulmonary function tests are difficult to administer and therefore data might be limited.",null,{"allocation":24,"interventionModel":24,"interventionModelDescription":24,"primaryPurpose":24,"observationalModel":26,"timePerspective":27,"maskingInfo":24},"COHORT","PROSPECTIVE","Clinical characteristics of Pompe patients receiving avalglucosidase alfa will be collected and analyzed. Adverse events occurring during avalglucosidase alfa treatment will be collected and described. Urinary Glc4\u002FHex4 is the primary endpoints for effectiveness analysis of avalglucosidase alfa. Other assessment items include creatine kinase, motor function (Quick Motor Function Test) and respiratory function (forced vital capacity). The evolution of those assessment items after avalglucosidase alfa treatment will be descriptively analyzed and compared to baseline assessments (i.e., the time before initiating avalglucosidase alfa and\u002For one-year preceding avalglucosidase alfa initiation) at the individual patient level depending on prior duration of ERT at time of avalglucosidase alfa switch.","Pompe Patients receiving Avalglucosidase alfa",[31],{"name":32,"affiliation":33,"role":34},"Yin-Hsiu Chien, MD, PhD","National Taiwan University Hospital Taipei, Taiwan, 100","PRINCIPAL_INVESTIGATOR",[36],{"name":32,"role":37,"phone":38,"phoneExt":39,"email":40},"CONTACT","+886223123456","271937","chienyh@ntu.edu.tw",[42],{"facility":5,"status":43,"city":44,"state":45,"zip":46,"country":45,"countryCode":47,"cosmosGeoPoint":48,"geoPoint":53,"contacts":54},"RECRUITING","Taipei","Taiwan","100","TW",{"type":49,"coordinates":50},"Point",[51,52],121.52639,25.05306,{"lat":52,"lon":51},[55],{"name":32,"role":37,"phone":38,"phoneExt":39,"email":40},{"type":34,"investigatorFullName":57,"investigatorTitle":58,"investigatorAffiliation":5,"oldNameTitle":24,"oldOrganization":24},"National Taiwan University Clinical Trial Center","Attending Physician","100657002","pompe-disease-rwe-long-term-monitoring-program-100657002",false,"NCT07834320","Pompe Disease RWE Long-term Monitoring Program","Long Term Follow-up for Patients With Pompe Disease Undergoing Compassionate Use and Transitioning to Medication Listed Under the National Health Insurance.","Inclusion criteria:\n\n-Confirmed GAA enzyme deficiency (any tissue source) and\u002For confirmed 2 pathogenic GAA gene variants Pompe patient\n\nExclusion criteria:\n\n* Having severe allergic reaction to avalglucosidase alfa.\n* Patient\u002Fparent\u002Flegal guardian is not agreeing to provide signed informed consent, and the patient, if \\\u003C18 years of age","ALL",{"count":68,"type":69},40,"ESTIMATED","OBSERVATIONAL","Evaluate short- and long-term biomarker, motor function, respiratory function and safety with avalglucosidase alfa in IOPD and LOPD patients who switched from other ERT.",[73],"Pompe Disease",[73],"2026-09-16",{"date":77,"type":78},"2026-09-22","ACTUAL",{"date":80,"type":78},"2023-10-01",{"date":82,"type":69},"2036-10-01",{"name":5,"class":6},1]