[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100560337":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":20,"centralContacts":28,"locations":19,"responsibleParty":38,"collaborators":19,"id":41,"slug":42,"hasResults":43,"nctId":44,"briefTitle":45,"officialTitle":46,"acronym":47,"eligibilityCriteria":48,"healthyVolunteers":43,"sex":49,"minAge":50,"maxAge":19,"enrollmentInfo":51,"targetDuration":19,"studyType":54,"phases":55,"briefSummary":57,"conditions":58,"keywords":19,"overallStatus":63,"whyStopped":19,"lastUpdateSubmitDate":64,"lastUpdatePostDateStruct":65,"startDateStruct":68,"completionDateStruct":70,"leadSponsor":72,"locationsCount":19},{"fullName":5,"class":6},"Erasmus Medical Center","OTHER",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"Algucosidase alfa 20 mg\u002Fkg once every 4 weeks instead of once every 2 weeks","EXPERIMENTAL","Alglucosidase alfa will be administered by intravenous infusion at a dose of 20 mg\u002Fkg once every 4 weeks instead of once every 2 weeks for a duration of 9 months.\n\nAfter 9 months of treatment with the extended interval, it will be determined for each patient whether it is considered safe to discontinue enzyme replacement therapy (ERT). Both, patients who stop ERT after 9 months and those who continue with either the new or the previous dosing schedule, will be closely followed for an additional 12 months, leading to a total study duration of 21 months.",[13],"Drug: Algucosidase alfa 20 mg\u002Fkg once every 4 weeks instead of once every 2 weeks",[15],{"type":16,"name":9,"description":17,"armGroupLabels":18,"otherNames":19},"DRUG","The interval of ERT with alglucosidase alfa will be extended from once every 2 weeks to once every 4 weeks. The dose of 20 mg\u002Fkg per infusion remains the same.",[9],null,[21,24,26],{"name":22,"affiliation":5,"role":23},"Pieter A. van Doorn, Prof. dr.","PRINCIPAL_INVESTIGATOR",{"name":25,"affiliation":5,"role":23},"Nadine A.M.E. van der Beek, Dr.",{"name":27,"affiliation":5,"role":23},"Tim Preijers, Dr.",[29,34],{"name":30,"role":31,"phone":32,"phoneExt":19,"email":33},"Ina Barzel, MSc","CONTACT","+31(0)107031182 |","i.barzel@erasmusmc.nl",{"name":35,"role":31,"phone":36,"phoneExt":19,"email":37},"Lianne H. Potters, MSc","+31(0)0633342010","l.potters@erasmusmc.nl",{"type":23,"investigatorFullName":39,"investigatorTitle":40,"investigatorAffiliation":5,"oldNameTitle":19,"oldOrganization":19},"Ina Barzel","Coordinating investigator","100560337","phase-4-treatment-frequency-reduction-in-pompe-disease-100560337",false,"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","ALL","50 Years",{"count":52,"type":53},10,"ESTIMATED","INTERVENTIONAL",[56],"PHASE4","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.",[59,60,61,62],"Pompe Disease (Late-onset)","GAA Deficiency","Glycogen Storage Disease Type II","Acid Maltase Deficiency","NOT_YET_RECRUITING","2024-08-26",{"date":66,"type":67},"2024-08-28","ACTUAL",{"date":69,"type":53},"2024-10-01",{"date":71,"type":53},"2027-12-31",{"name":5,"class":6}]