[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"pompe-disease\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:pompe-disease":26},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,10,0,[8,54,82,111,141,161,179,201,219,256],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":24,"conditions":25,"keywords":27,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":42,"lastUpdatePostDateStruct":43,"startDateStruct":46,"completionDateStruct":48,"leadSponsor":50,"locationsCount":53},"100602411","phase-2-study-of-s-606001-as-an-add-on-to-enzyme-replacement-therapy-ert-in-participants-with-late-onset-pompe-disease-lopd-100602411",false,"NCT07123155","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","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.","ALL","18 Years",{"count":19,"type":20},45,"ESTIMATED","INTERVENTIONAL",[23],"PHASE2","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.",[26],"Pompe Disease",[28,29,30,31,32,33,34,35,36,37,38,39,40],"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","RECRUITING","2026-06-19",{"date":44,"type":45},"2026-06-23","ACTUAL",{"date":47,"type":45},"2025-10-30",{"date":49,"type":20},"2027-08-08",{"name":51,"class":52},"Shionogi","INDUSTRY",28,{"id":55,"slug":56,"hasResults":11,"nctId":57,"briefTitle":58,"officialTitle":59,"acronym":4,"eligibilityCriteria":60,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":61,"targetDuration":63,"studyType":64,"phases":4,"briefSummary":65,"conditions":66,"keywords":68,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":42,"lastUpdatePostDateStruct":74,"startDateStruct":75,"completionDateStruct":77,"leadSponsor":79,"locationsCount":81},"100075056","pompe-disease-registry-protocol-100075056","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",{"count":62,"type":20},2000,"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.",[67,26],"Glycogen Storage Disease Type II",[69,70,26,71,72,73],"Glycogen Storage Disease Type II (GSD-II)","GSD-II","Pompe Disease (late-onset)","Acid Maltase Deficiency Disease","Glycogenosis II",{"date":44,"type":45},{"date":76,"type":45},"2004-09-15",{"date":78,"type":20},"2034-01-31",{"name":80,"class":52},"Genzyme, a Sanofi Company",272,{"id":83,"slug":84,"hasResults":11,"nctId":85,"briefTitle":86,"officialTitle":87,"acronym":4,"eligibilityCriteria":88,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":89,"targetDuration":4,"studyType":64,"phases":4,"briefSummary":91,"conditions":92,"keywords":93,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":100,"lastUpdatePostDateStruct":101,"startDateStruct":103,"completionDateStruct":105,"leadSponsor":107,"locationsCount":110},"100644279","phrenic-nerve-and-diaphragm-electrophysiology-in-pompe-disease-100644279","NCT07664930","Phrenic Nerve and Diaphragm Electrophysiology in Pompe Disease","Electrophysiological Study of the Phrenic Nerve and Diaphragm in Pompe Disease: Retrospective and Prospective Cohort Analysis","Inclusion Criteria:\n\nAge ≥ 18 years.\n\nFor the prospective cohort:\n\n* Genetically confirmed diagnosis of Pompe disease.\n* Ability to undergo routine neurophysiological and respiratory assessments.\n* Written informed consent provided.\n\nFor the retrospective cohort:\n\n* History of restrictive respiratory failure or unexplained hypoventilation.\n* Availability of previous phrenic nerve conduction studies and\u002For diaphragm electromyography performed as part of routine clinical evaluation.\n\nExclusion Criteria:\n\n\\- Age \\\u003C 18 years.\n\nFor the prospective cohort:\n\n* Conditions preventing completion of neurophysiological assessments (e.g., inability to maintain required positioning or relevant clinical contraindications).\n* Known primary phrenic nerve injury (e.g., postsurgical phrenic palsy or documented traumatic phrenic neuropathy).\n* Presence of other neuromuscular disorders potentially confounding data interpretation.\n* Refusal or inability to provide informed consent.\n\nFor the retrospective cohort:\n\n* Incomplete or technically non-interpretable neurophysiological examinations.\n* Previously established respiratory or neuromuscular diagnoses fully explaining respiratory impairment.\n* Cases requiring additional clinical information for study purposes when patient consent for contact or data completion cannot be obtained.",{"count":90,"type":20},20,"Pompe disease is traditionally considered a lysosomal myopathy. However, increasing experimental and clinical evidence suggests involvement of the entire motor unit, including motor neurons, peripheral nerves, neuromuscular junctions, and skeletal muscle. Respiratory impairment is a major cause of morbidity and mortality, and diaphragm dysfunction is frequently observed.\n\nClinical observations at IRCCS Fondazione Mondino have highlighted neurophysiological abnormalities of the phrenic nerve and diaphragm in patients with Pompe disease and respiratory involvement, sometimes occurring even in the absence of clinically significant limb muscle weakness. These findings suggest that respiratory motor unit dysfunction may represent an important component of the disease phenotype.\n\nThis observational study aims to systematically characterize phrenic nerve conduction parameters and diaphragm electromyographic findings in adult patients with genetically confirmed Pompe disease and in patients with unexplained respiratory failure. Retrospective and prospective clinical, neurophysiological, and respiratory data collected during routine clinical care will be analyzed to explore whether phrenic nerve and diaphragm abnormalities may serve as markers of respiratory motor unit involvement in Pompe disease.",[26],[94,95,96,97,98,99],"Pompe disease","Phrenic nerve","Diaphragm","Respiratory muscle weakness","Neuromuscular respiratory failure","Motor unit","2026-06-17",{"date":102,"type":45},"2026-06-24",{"date":104,"type":45},"2026-03-30",{"date":106,"type":20},"2029-02-28",{"name":108,"class":109},"IRCCS National Neurological Institute \"C. Mondino\" Foundation","OTHER",1,{"id":112,"slug":113,"hasResults":11,"nctId":114,"briefTitle":115,"officialTitle":116,"acronym":4,"eligibilityCriteria":117,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":118,"targetDuration":4,"studyType":64,"phases":4,"briefSummary":120,"conditions":121,"keywords":122,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":132,"lastUpdatePostDateStruct":133,"startDateStruct":135,"completionDateStruct":137,"leadSponsor":139,"locationsCount":110},"100183587","determination-of-crim-status-and-longitudinal-follow-up-of-individuals-with-pompe-disease-100183587","NCT01665326","Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease","Determination of Cross-Reactive Immunological Material (CRIM) Status and Longitudinal Follow-up of Individuals With Pompe Disease","Inclusion Criteria:\n\n* Confirmed diagnosis of infantile, atypical or juvenile onset Pompe disease\n* Must provide a written informed consent",{"count":119,"type":20},400,"This is a longitudinal natural history study of Infantile Pompe disease. The investigators will regularly collect and review medical information regarding the diagnosis of Pompe disease, response to enzyme replacement (ERT) using alglucosidase alfa (Lumizyme\u002FMyozyme) and response to immunosuppressive therapy in cases at risk for developing or those who have developed high and sustained antibodies to ERT. To follow the long-term outcomes, we will collect medical records including but not limited to the diagnosis, clinical parameters, assessments for clinical monitoring, and laboratory values including antibody testing results.",[26],[94,67,123,124,125,126,127,29,128,129,130,131],"Acid Maltase Deficiency","CRIM Status","Acid Alpha-Glucosidase Deficiency","Alglucosidase alfa","Myozyme","Immune Tolerance Induction","Lumizyme","Immunomodulation","Anti-drug antibodies","2026-04-06",{"date":134,"type":45},"2026-04-08",{"date":136,"type":4},"2009-09",{"date":138,"type":20},"2029-03",{"name":140,"class":109},"Duke University",{"id":142,"slug":143,"hasResults":11,"nctId":144,"briefTitle":145,"officialTitle":145,"acronym":4,"eligibilityCriteria":146,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":147,"targetDuration":63,"studyType":64,"phases":4,"briefSummary":149,"conditions":150,"keywords":4,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":151,"lastUpdatePostDateStruct":152,"startDateStruct":154,"completionDateStruct":156,"leadSponsor":158,"locationsCount":160},"100525382","a-global-prospective-observational-registry-of-patients-with-pompe-disease-100525382","NCT06121011","A Global Prospective Observational Registry of Patients With Pompe Disease","Inclusion Criteria:\n\n* Diagnosis of LOPD or IOPD based on documented deficiency of GAA enzyme activity and\u002For GAA genotyping\n\nExclusion Criteria:\n\n* Patients who are currently receiving investigational therapy for Pompe disease in a clinical trial, a compassionate use program, or an expanded access program (EAP)",{"count":148,"type":20},500,"This is a global, multicenter, prospective, observational registry of patients with Pompe disease, including those with late-onset pompe disease (LOPD) and infantile-onset pompe disease (IOPD). Both untreated patients and those being treated with an approved therapy for Pompe disease are eligible to participate.\n\nThe objectives of the registry are:\n\n* To evaluate the long-term safety of Pompe disease treatments through collection of data that describe the frequency of adverse events (AEs)\u002Fserious adverse events (SAEs) occurring in Pompe disease patients\n* To evaluate the long-term real-world effectiveness of Pompe disease treatments\n* To evaluate the long-term real-world impact of Pompe disease treatments on quality of life (QOL) and patient-reported outcomes (PROs)\n* To describe the natural history of untreated Pompe disease",[26],"2026-03-09",{"date":153,"type":45},"2026-03-10",{"date":155,"type":45},"2024-02-16",{"date":157,"type":20},"2034-12-20",{"name":159,"class":52},"Amicus Therapeutics",41,{"id":162,"slug":163,"hasResults":11,"nctId":164,"briefTitle":165,"officialTitle":165,"acronym":166,"eligibilityCriteria":167,"healthyVolunteers":11,"sex":16,"minAge":168,"maxAge":17,"enrollmentInfo":169,"targetDuration":4,"studyType":64,"phases":4,"briefSummary":170,"conditions":171,"keywords":4,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":172,"lastUpdatePostDateStruct":173,"startDateStruct":175,"completionDateStruct":177,"leadSponsor":178,"locationsCount":110},"100411577","cognitive-and-neurological-pathologies-in-pompe-disease-100411577","NCT04639336","Cognitive and Neurological Pathologies in Pompe Disease","CNS","Inclusion Criteria:\n\n* Subject has a confirmed and documented diagnosis of infantile Pompe Disease (IPD) or Late onset Pompe Disease (LOPD)\n\nExclusion Criteria:\n\n* Refusal to give informed consent","0 Years",{"count":19,"type":20},"The purpose of this study is to better understand the long-term health effects of Pompe disease and to determine if there are any abnormal changes in the brain and peripheral nerves. Additionally, the investigators will study the relationship between the abnormal changes in brain, nervous system findings, and developmental outcomes.\n\nThe investigators will collect clinical information from clinic visits as well as assessments such as neuroimaging (magnetic resonance imaging (MRI), magnetic resonance spectroscopy (MRS), and diffusion tensor imaging (DTI)), cognition, academic skills, speech and language function, physical therapy and quantitative muscle ultrasound. Subjects will be in this study for at least 3 years and up to 6 years.",[26],"2026-03-03",{"date":174,"type":45},"2026-03-04",{"date":176,"type":45},"2016-08-03",{"date":138,"type":20},{"name":140,"class":109},{"id":180,"slug":181,"hasResults":11,"nctId":182,"briefTitle":183,"officialTitle":184,"acronym":4,"eligibilityCriteria":185,"healthyVolunteers":11,"sex":186,"minAge":4,"maxAge":4,"enrollmentInfo":187,"targetDuration":4,"studyType":64,"phases":4,"briefSummary":189,"conditions":190,"keywords":4,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":192,"lastUpdatePostDateStruct":193,"startDateStruct":195,"completionDateStruct":197,"leadSponsor":199,"locationsCount":110},"100495693","avalglucosidase-alfa-pregnancy-study-100495693","NCT05734521","Avalglucosidase Alfa Pregnancy Study","A Descriptive Safety Study Based on Data Collected From Women and Their Offspring Exposed to Nexviazyme\u002FNexviadyme (Avalglucosidase Alfa-ngpt\u002FAvalglucosidase Alfa) During Pregnancy and\u002For Lactation in the Postmarketing Setting","Inclusion Criteria:\n\n* Women and their offspring exposed to avalglucosidase alfa during pregnancy and\u002For lactation for whom an Individual Case Safety Report (ICSR) has been submitted to the Sanofi Global Pharmacovigilance (PV) department, and\u002For,\n* Women exposed to avalglucosidase alfa during pregnancy and\u002For lactation who have provided informed consent to enroll in the Pompe Pregnancy Sub-registry.\n\nExclusion Criteria:\n\nThere are no exclusion criteria in this study.\n\nThe above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.","FEMALE",{"count":188,"type":20},100,"This is a worldwide, descriptive safety study collecting data on women and their offspring exposed to avalglucosidase alfa during pregnancy and\u002For lactation, to assess the risks of avalglucsodiase alfa on pregnancy and maternal complications and adverse effects in the developing fetus, neonate, and infant.\n\n* Outcomes in exposed infants, including growth and development, will be assessed through at least the first year of life.\n* Data will be collected for approximately 10 years.",[26,191],"Pregnancy","2026-01-14",{"date":194,"type":45},"2026-01-16",{"date":196,"type":45},"2022-10-26",{"date":198,"type":20},"2032-10-31",{"name":200,"class":52},"Sanofi",{"id":202,"slug":203,"hasResults":11,"nctId":204,"briefTitle":205,"officialTitle":206,"acronym":4,"eligibilityCriteria":207,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":4,"targetDuration":4,"studyType":208,"phases":4,"briefSummary":209,"conditions":210,"keywords":211,"overallStatus":214,"whyStopped":4,"lastUpdateSubmitDate":215,"lastUpdatePostDateStruct":216,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":218,"locationsCount":4},"100352205","expanded-access-for-atb200at2221-for-the-treatment-of-pompe-disease-100352205","NCT03865836","Expanded Access for ATB200\u002FAT2221 for the Treatment of Pompe Disease","Named Patient or Compassionate Use for Treatment Use of ATB200\u002FAT2221 for Patients With Pompe Disease","Inclusion Criteria:\n\n1. Subject must have a diagnosis of Pompe disease based on documentation of one of the following:\n\n   1. deficiency of GAA enzyme\n   2. GAA genotyping\n2. Patient does not currently qualify for an Amicus sponsored on-going clinical trial or is declining on currently approved ERT (e.g. Myozyme)\n3. The patient must be willing to receive treatment with ATB200\u002FAT2221 via this program, which includes signing an authorization form for sharing clinical data with Amicus Therapeutics, and its agent Early Access Care LLC.\n\nExclusion Criteria:\n\n1. Patient, whether male or female, is planning to conceive a child during the treatment program.\n2. Patient has a hypersensitivity to any of the excipients in ATB200, alglucosidase alfa, or AT2221\n3. Patient has a medical or any other extenuating condition or circumstance that may, in the opinion of the investigator, pose an undue safety risk to the subject or compromise his\u002Fher ability to comply with or adversely impact protocol requirements.\n4. Patient has received any gene therapy at any time.\n5. Concomitant use of miglitol (eg, Glyset), non-AT2221 form of miglustat (eg, Zavesca), acarbose (eg, Precose or Glucobay), voglibose (eg, Volix, Vocarb, or Volibo)","EXPANDED_ACCESS","This is an expanded access program (EAP) for eligible participants designed to provide access to ATB200\u002FAT2221.",[26],[212,213],"expanded access","compassionate use","AVAILABLE","2025-09-09",{"date":217,"type":45},"2025-09-15",{"name":159,"class":52},{"id":220,"slug":221,"hasResults":11,"nctId":222,"briefTitle":223,"officialTitle":223,"acronym":4,"eligibilityCriteria":224,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":225,"targetDuration":4,"studyType":64,"phases":4,"briefSummary":227,"conditions":228,"keywords":245,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":247,"lastUpdatePostDateStruct":248,"startDateStruct":250,"completionDateStruct":252,"leadSponsor":254,"locationsCount":110},"100557519","flower-following-longitudinal-outcomes-with-epidemiology-for-rare-diseases-100557519","NCT06539169","FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases","Inclusion Criteria:\n\n* Any person with a known or suspected rare disease, defined by their prevalence of fewer than 200,000 individuals nationwide. Diseases include but are not limited to:\n\nAlpha- or Beta- Thalassemia Amyloidosis Amyotrophic Lateral Sclerosis (ALS) Creutzfeldt-Jakob disease (CJD) Cystic Fibrosis (CF) Duchenne Muscular Dystrophy (DMD) Early-onset Alzheimer's Disease Ehlers-Danlos Syndrome (EDS) Huntington's Disease (HD) Gaucher Disease GM1 Gangliosidosis Myasthenia Gravis Pompe Disease Sickle Cell Disease Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Transthyretin Amyloid Polyneuropathy (ATTR-PN)\n\n\\- Patients or their legally-authorized representative must be willing and able to provide informed consent (and assent, if applicable). Deceased persons may participate via consent of their legally-authorized representative in accordance with applicable Federal and state laws\n\nExclusion Criteria:\n\n* Patient or LAR is unable to provide informed consent.\n* Patient resides in a country other than the United States and is unable to provide access to medical records.",{"count":226,"type":20},1000,"FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases. Patients participate in the study by electronic consent (eConsent) and sign a medical records release to permit data collection. Medical records are accessed from institutions directly via eFax or paper fax, online from patient electronic medical record (EMR) portals, direct from DNA\u002FRNA sequencing and molecular profiling vendors, and via electronic health information exchanges. Patients and their treating physicians may also optionally provide medical records. Medical records are received in or converted to electronic\u002Fdigitized formats (CCDA, FHIR, PDF), sorted by medical record type (clinic visit, in-patient hospital, out-patient clinic, infusion and out-patient pharmacies, etc.) and made machine-readable to support data annotation, full text searches, and natural language processing (NLP) algorithms to further facilitate feature identification.",[229,230,231,232,233,234,235,236,237,238,239,240,241,26,242,243,244],"Alpha-Thalassemia","Beta-Thalassemia","Amyloidosis","Amyotrophic Lateral Sclerosis","Creutzfeld-Jakob Disease","Cystic Fibrosis","Duchenne Muscular Dystrophy","Early-Onset Alzheimer Disease","Ehlers-Danlos Syndrome","Huntington Disease","Gaucher Disease","GM1 Gangliosidosis","Myasthenia Gravis","Sickle Cell Disease","Transthyretin Amyloid Cardiomyopathy","Rare Diseases",[246],"rare diseases","2024-11-12",{"date":249,"type":45},"2024-11-14",{"date":251,"type":45},"2024-06-10",{"date":253,"type":20},"2026-06-10",{"name":255,"class":52},"xCures",{"id":257,"slug":258,"hasResults":11,"nctId":259,"briefTitle":260,"officialTitle":260,"acronym":4,"eligibilityCriteria":261,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":262,"targetDuration":4,"studyType":64,"phases":4,"briefSummary":264,"conditions":265,"keywords":4,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":266,"lastUpdatePostDateStruct":267,"startDateStruct":269,"completionDateStruct":271,"leadSponsor":273,"locationsCount":110},"100239795","a-long-term-study-for-the-outcome-of-pompe-disease-100239795","NCT02399748","A Long-term Study for the Outcome of Pompe Disease","Inclusion Criteria:\n\nConfirm diagnosis with Pompe disease, is or is preparing receiving enzyme replacement therapy\n\nExclusion Criteria:\n\nNo",{"count":263,"type":20},50,"Prospectively follow patients with Pompe disease underwent enzyme replacement therapy.",[26],"2017-07-13",{"date":268,"type":45},"2017-07-18",{"date":270,"type":4},"2007-04",{"date":272,"type":20},"2026-12",{"name":274,"class":109},"National Taiwan University Hospital"]