{"disease":{"id":"glycogen-storage-disease-type-ia","name":"glycogen storage disease type ia"},"drugs":{"marketed":[],"pipeline":[],"offLabel":[],"totalMarketed":0,"totalPipeline":0},"trials":{"data":[{"nct_id":"NCT03655223","title":"Early Check: Expanded Screening in Newborns","phase":"","overall_status":"ENROLLING_BY_INVITATION","enrollment_count":30000,"lead_sponsor_name":"RTI International","has_results":false},{"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":"NCT04909346","title":"Adeno-Associated Virus (AAV) Antibody Study in Subjects OTC Deficiency, GSDIa, and Wilson Disease","phase":"","overall_status":"TERMINATED","enrollment_count":51,"lead_sponsor_name":"Ultragenyx Pharmaceutical Inc","has_results":false},{"nct_id":"NCT05139316","title":"A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)","phase":"PHASE3","overall_status":"COMPLETED","enrollment_count":49,"lead_sponsor_name":"Ultragenyx Pharmaceutical Inc","has_results":false},{"nct_id":"NCT06735755","title":"A Phase 1/2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients With Glycogen Storage Disease Type Ia (GSDIa)","phase":"PHASE1, PHASE2","overall_status":"RECRUITING","enrollment_count":36,"lead_sponsor_name":"Beam Therapeutics Inc.","has_results":false},{"nct_id":"NCT04311307","title":"Endogenous Glucose Production in Patients With Glycogen Storage Disease Type Ia","phase":"PHASE1, PHASE2","overall_status":"COMPLETED","enrollment_count":20,"lead_sponsor_name":"University Medical Center Groningen","has_results":false},{"nct_id":"NCT04708015","title":"Retrospective Study of Glucose Monitoring for Glycemic Control in Patients With GSDIa","phase":"","overall_status":"COMPLETED","enrollment_count":15,"lead_sponsor_name":"Ultragenyx Pharmaceutical Inc","has_results":false},{"nct_id":"NCT03970278","title":"Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia","phase":"","overall_status":"COMPLETED","enrollment_count":12,"lead_sponsor_name":"Ultragenyx Pharmaceutical Inc","has_results":true},{"nct_id":"NCT06843330","title":"Accuracy of Lactate Meter in GSDIa","phase":"","overall_status":"RECRUITING","enrollment_count":10,"lead_sponsor_name":"Connecticut Children's Medical Center","has_results":false}],"total":9},"guidelines":[],"source":"Drug Landscape verified database"}