[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"glycogen-storage-disease-type-ii\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:glycogen-storage-disease-type-ii":24},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,45,70,98],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":17,"targetDuration":20,"studyType":21,"phases":4,"briefSummary":22,"conditions":23,"keywords":26,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":33,"lastUpdatePostDateStruct":34,"startDateStruct":37,"completionDateStruct":39,"leadSponsor":41,"locationsCount":44},"100075056","pompe-disease-registry-protocol-100075056",false,"NCT00231400","Pompe Disease Registry Protocol","Pompe Disease Registry","Inclusion Criteria:\n\nAll patients with a confirmed diagnosis of Pompe disease who have signed the informed consent and authorization form(s) are eligible for inclusion. Confirmed diagnosis is defined as documented GAA enzyme deficiency from blood, skin, or muscle tissue and\u002For documentation of 2 GAA gene mutations.\n\nExclusion Criteria:\n\nThere are no exclusion criteria in this Registry","ALL",{"count":18,"type":19},2000,"ESTIMATED","5 Years","OBSERVATIONAL","The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to track the disease's natural history and outcomes in patients, both treated and not. Data from the Registry are also used to fulfill various global regulatory commitments, to support product development\u002Freimbursement, and for other research and non-research related purposes.\n\nThe objectives of the Registry are:\n\n* To enhance understanding of the variability, progression, identification, and natural history of Pompe disease, with the ultimate goal of better guiding and assessing therapeutic intervention.\n* To assist the Pompe medical community with the development of recommendations for monitoring patients, and to provide reports on patient outcomes, to optimize patient care.\n* To characterize the Pompe disease population.\n* To evaluate the long-term effectiveness of alglucosidase alfa.",[24,25],"Glycogen Storage Disease Type II","Pompe Disease",[27,28,25,29,30,31],"Glycogen Storage Disease Type II (GSD-II)","GSD-II","Pompe Disease (late-onset)","Acid Maltase Deficiency Disease","Glycogenosis II","RECRUITING","2026-06-19",{"date":35,"type":36},"2026-06-23","ACTUAL",{"date":38,"type":36},"2004-09-15",{"date":40,"type":19},"2034-01-31",{"name":42,"class":43},"Genzyme, a Sanofi Company","INDUSTRY",272,{"id":46,"slug":47,"hasResults":11,"nctId":48,"briefTitle":49,"officialTitle":50,"acronym":4,"eligibilityCriteria":51,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":52,"enrollmentInfo":53,"targetDuration":4,"studyType":55,"phases":56,"briefSummary":58,"conditions":59,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":61,"lastUpdatePostDateStruct":62,"startDateStruct":64,"completionDateStruct":66,"leadSponsor":68,"locationsCount":69},"100567301","phase-4-china-post-approval-commitment-pac-study-of-avalglucosidase-alfa-in-participants-with-iopd-100567301","NCT06666413","China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD","A Single-arm, Open-label, Phase 4 Study to Evaluate the Safety and Efficacy of Avalglucosidase Alfa in Chinese Participants With Infantile-onset Pompe Disease (IOPD)","Inclusion Criteria:\n\n* Participant must be \\\u003C18 years of age, at the time of signing the informed consent.\n* Participants have documented onset of Pompe disease symptoms before 12 months of age (corrected for gestation if born before 40 weeks); and diagnosis of IOPD is confirmed by GAA enzyme deficiency from any tissue source and GAA gene pathogenic mutations.\n* Participants must have documented cardiomyopathy at the time of diagnosis.\n* Contraceptive use should be consistent with local regulations Participant's parent\u002Flegally authorized representative (LAR) must be capable of giving signed informed consent.\n\nExclusion Criteria: Participants are excluded from the study if any of the following criteria apply:\n\n* Participants with major congenital abnormality that, in the opinion of the Investigator, would preclude participation in the study or potentially decrease survival.\n* Participants with clinically significant organic disease (with the exception of symptoms relating to Pompe disease).\n* Participants who have received an ERT other than alglucosidase alfa or avalglucosidase alfa, or any other treatment for Pompe disease, including gene therapy prior to the enrollment.\n* Participants who have received alglucosidase alfa or avalglucosidase alfa less than 1 week prior to the first dose of avalglucosidase alfa given as IMP Participants who are anticipated to take prohibited therapy (ie, any other treatment for Pompe disease) during this study.\n* Participants who have taken other investigational drugs (not Pompe disease specific) within 30 days or 5 elimination half-lives in blood of that drug before enrollment, whichever is longer, or are anticipated to take any other concurrent investigational treatments.\n* Participants not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures.\n\nThe above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.","17 Years",{"count":54,"type":19},13,"INTERVENTIONAL",[57],"PHASE4","This is a single group, 52-week treatment, Phase 4, open-label, single-arm study to assess the safety and efficacy of avalglucosidase alfa IV infusion in male and female Chinese participants with IOPD who are treatment-naïve or were previously treated with ERT.\n\nStudy details include:\n\n* The study duration: total study duration is approximately 64 weeks.\n\n  * Screening period of up to 8 weeks\n  * Treatment period of 52 weeks\n  * Follow-up period of 4 weeks. (if the participant enrolls in another study or receives commercially available ERT, the follow-up period may be reduced from 4 to 2 weeks)\n* The number of visits will be 30, including 29 site visits and 1 phone call follow-up visit.",[24,60],"Pompe's Disease","2026-03-19",{"date":63,"type":36},"2026-03-23",{"date":65,"type":36},"2025-05-07",{"date":67,"type":19},"2028-05-02",{"name":42,"class":43},1,{"id":71,"slug":72,"hasResults":11,"nctId":73,"briefTitle":74,"officialTitle":75,"acronym":76,"eligibilityCriteria":77,"healthyVolunteers":11,"sex":16,"minAge":78,"maxAge":4,"enrollmentInfo":79,"targetDuration":4,"studyType":55,"phases":81,"briefSummary":82,"conditions":83,"keywords":4,"overallStatus":87,"whyStopped":4,"lastUpdateSubmitDate":88,"lastUpdatePostDateStruct":89,"startDateStruct":91,"completionDateStruct":93,"leadSponsor":95,"locationsCount":4},"100560337","phase-4-treatment-frequency-reduction-in-pompe-disease-100560337","NCT06575829","Treatment Frequency Reduction in Pompe Disease","Open Label, Single-center Pilot Study to Investigate Alglucosidase Alfa (20 mg\u002Fkg) Frequency Reduction From 2 to 4 Weeks in a Subgroup of Elderly Patients With Late-onset Pompe Disease (TRIPOD-Study)","TRIPOD","Inclusion Criteria:\n\n* LOPD (confirmed diagnosis: enzyme deficiency in any tissue source and\u002For 2 confirmed disease-causing variants in the GAA gene)\n* Age ≥50 years\n* Current treatment with alglucosidase alfa at a standard dose of 20 mg\u002Fkg once every 2 weeks for ≥4 years\n* Relatively stable clinical condition over the past year\n* Able to walk ≥150 m within 6 minutes (6MWT)\n* (Forced) vital capacity (FVC) in sitting position: \\>55% of expected value and in supine position: \\>45% of expected value\n* Willing and able to adhere to the study procedures\n\nExclusion Criteria:\n\n* Rapidly progressive muscle weakness\n* Severely limited muscle strength almost requiring\u002Frequiring daily wheelchair use\n* Requiring respiratory support (non-invasive\u002Finvasive ventilation) or being at high risk to require respiratory support (ventilation) due to further deterioration of current pulmonary function. Using continuous positive airway pressure (CPAP) support only for obstructive sleep apnea syndrome (OSAS) is permitted.\n* Comorbidities which are expected to influence the primary outcome measures within the next 2 years","50 Years",{"count":80,"type":19},10,[57],"The aim of this study is to assess if dosing frequency reduction of alglucosidase alfa 20 mg\u002Fkg once every 2 weeks to once every 4 weeks is safe and does not lead to increased progression of disease in a selected group of patients with late-onset Pompe disease.",[84,85,24,86],"Pompe Disease (Late-onset)","GAA Deficiency","Acid Maltase Deficiency","NOT_YET_RECRUITING","2024-08-26",{"date":90,"type":36},"2024-08-28",{"date":92,"type":19},"2024-10-01",{"date":94,"type":19},"2027-12-31",{"name":96,"class":97},"Erasmus Medical Center","OTHER",{"id":99,"slug":100,"hasResults":11,"nctId":101,"briefTitle":102,"officialTitle":103,"acronym":4,"eligibilityCriteria":104,"healthyVolunteers":11,"sex":16,"minAge":105,"maxAge":106,"enrollmentInfo":107,"targetDuration":4,"studyType":21,"phases":4,"briefSummary":109,"conditions":110,"keywords":4,"overallStatus":87,"whyStopped":4,"lastUpdateSubmitDate":111,"lastUpdatePostDateStruct":112,"startDateStruct":114,"completionDateStruct":116,"leadSponsor":118,"locationsCount":4},"100440600","higher-dose-of-alglucosidase-alpha-for-pompe-disease-100440600","NCT05017402","Higher Dose of Alglucosidase Alpha for Pompe Disease","Higher Dose of Alglucosidase Alpha for Pompe Disease: Long-term Follow-up Study","Inclusion Criteria:\n\n1. Diagnosis of Pompe disease\n2. Currently receiving ERT with regular clinic visits\n3. Agree to sign informed consent\n\nExclusion Criteria:\n\n1. Lost follow-up for clinical visits\n2. Allergy to Myozyme\n3. Refuse to sign informed consent","0 Years","60 Years",{"count":108,"type":19},36,"This study is aimed to investigate that whether the higher dose ERT improve safety and clinical outcomes of Pompe disease patients. Also, wish to develop a new therapeutic recommendation and hope that it could improve the long-term outcomes of Pompe diesease patients.",[24],"2021-08-17",{"date":113,"type":36},"2021-08-23",{"date":115,"type":19},"2021-09-01",{"date":117,"type":19},"2026-12-31",{"name":119,"class":120},"Taipei Veterans General Hospital, Taiwan","OTHER_GOV"]