{"disease":{"id":"pompe-s-disease","name":"pompe s disease"},"drugs":{"marketed":[],"pipeline":[],"offLabel":[],"totalMarketed":0,"totalPipeline":0},"trials":{"data":[{"nct_id":"NCT05687474","title":"Baby Detect : Genomic Newborn Screening","phase":"","overall_status":"COMPLETED","enrollment_count":6824,"lead_sponsor_name":"Centre Hospitalier Universitaire de Liege","has_results":false},{"nct_id":"NCT06539169","title":"FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases","phase":"","overall_status":"RECRUITING","enrollment_count":1000,"lead_sponsor_name":"xCures","has_results":false},{"nct_id":"NCT05619900","title":"Registry of Patients Diagnosed With Lysosomal Storage Diseases","phase":"","overall_status":"RECRUITING","enrollment_count":250,"lead_sponsor_name":"University of California, San Francisco","has_results":false},{"nct_id":"NCT04758130","title":"Getting Global Rare Disease Insights Through Technology Study","phase":"NA","overall_status":"UNKNOWN","enrollment_count":150,"lead_sponsor_name":"M.A.G.I.C. Clinic LTD","has_results":false},{"nct_id":"NCT02782741","title":"Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease","phase":"PHASE3","overall_status":"COMPLETED","enrollment_count":101,"lead_sponsor_name":"Genzyme, a Sanofi Company","has_results":true},{"nct_id":"NCT03893240","title":"Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease","phase":"NA","overall_status":"COMPLETED","enrollment_count":61,"lead_sponsor_name":"Spark Therapeutics, Inc.","has_results":false},{"nct_id":"NCT04676373","title":"Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen","phase":"PHASE4","overall_status":"COMPLETED","enrollment_count":41,"lead_sponsor_name":"Genzyme, a Sanofi Company","has_results":false},{"nct_id":"NCT05431127","title":"High Dose Inspiratory Muscle Training in LOPD","phase":"NA","overall_status":"COMPLETED","enrollment_count":34,"lead_sponsor_name":"Duke University","has_results":false},{"nct_id":"NCT05943678","title":"Novel Metabolic Muscular Biomarkers in Pompe Disease - a Non-invasive Magnetic Resonance Exploratory Pilot Study.","phase":"","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":30,"lead_sponsor_name":"Medical University of Vienna","has_results":false},{"nct_id":"NCT06130228","title":"Nutritional Therapy in Late-onset Pompe Disease","phase":"PHASE2","overall_status":"UNKNOWN","enrollment_count":28,"lead_sponsor_name":"McMaster University","has_results":false},{"nct_id":"NCT01898364","title":"Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.","phase":"PHASE1","overall_status":"COMPLETED","enrollment_count":24,"lead_sponsor_name":"Genzyme, a Sanofi Company","has_results":false},{"nct_id":"NCT03019406","title":"A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa","phase":"PHASE2","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":22,"lead_sponsor_name":"Genzyme, a Sanofi Company","has_results":true},{"nct_id":"NCT01410890","title":"Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease","phase":"PHASE4","overall_status":"COMPLETED","enrollment_count":21,"lead_sponsor_name":"Genzyme, a Sanofi Company","has_results":true},{"nct_id":"NCT03694561","title":"Developing a Management Approach for Patients With \"Late-Onset\" Pompe Disease","phase":"","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":20,"lead_sponsor_name":"Duke University","has_results":false},{"nct_id":"NCT05083806","title":"MSOT in Pompe Disease","phase":"NA","overall_status":"COMPLETED","enrollment_count":20,"lead_sponsor_name":"University of Erlangen-Nürnberg Medical School","has_results":false},{"nct_id":"NCT06666413","title":"China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD","phase":"PHASE4","overall_status":"RECRUITING","enrollment_count":13,"lead_sponsor_name":"Genzyme, a Sanofi Company","has_results":false},{"nct_id":"NCT00486889","title":"Growth and Development Study of Alglucosidase Alfa","phase":"PHASE4","overall_status":"COMPLETED","enrollment_count":12,"lead_sponsor_name":"Genzyme, a Sanofi Company","has_results":true},{"nct_id":"NCT04942912","title":"Effect of Enzyme Replacement Therapy in Patients With Juvenile-onset Pompe Disease","phase":"","overall_status":"UNKNOWN","enrollment_count":10,"lead_sponsor_name":"Central Hospital, Nancy, France","has_results":false},{"nct_id":"NCT06575829","title":"Treatment Frequency Reduction in Pompe Disease","phase":"PHASE4","overall_status":"NOT_YET_RECRUITING","enrollment_count":10,"lead_sponsor_name":"Erasmus Medical Center","has_results":false},{"nct_id":"NCT04532047","title":"PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)","phase":"PHASE1","overall_status":"RECRUITING","enrollment_count":10,"lead_sponsor_name":"University of California, San Francisco","has_results":false},{"nct_id":"NCT00701701","title":"Immune Tolerance Induction Study","phase":"PHASE4","overall_status":"TERMINATED","enrollment_count":4,"lead_sponsor_name":"Genzyme, a Sanofi Company","has_results":true},{"nct_id":"NCT02363153","title":"Diet and Exercise in Pompe Disease","phase":"NA","overall_status":"COMPLETED","enrollment_count":2,"lead_sponsor_name":"University of Florida","has_results":false}],"total":22},"guidelines":[],"source":"Drug Landscape verified database"}