{"disease":{"id":"mucopolysaccharidosis-type-i","name":"mucopolysaccharidosis type i"},"drugs":{"marketed":[{"drug_id":"hunterase","indication_name":"Mucopolysaccharidosis type II","indication_type":"approved","phase":null,"line_of_therapy":null,"is_primary_indication":false,"pivotal_trial_result":null,"pivotal_trial_p_value":null,"patient_population":null,"brand_name":null,"generic_name":"Hunterase","company_name":"Green Cross Corporation","drug_phase":"phase_3","molecular_target":"","drug_class":"","quality_score":null,"revenue":null,"mechanism":null},{"drug_id":"idursulfase-beta","indication_name":"Mucopolysaccharidosis type II","indication_type":"approved","phase":null,"line_of_therapy":null,"is_primary_indication":false,"pivotal_trial_result":null,"pivotal_trial_p_value":null,"patient_population":null,"brand_name":"Hunterase","generic_name":"IDURSULFASE BETA","company_name":"Clinigen K.K.","drug_phase":"marketed","molecular_target":"","drug_class":"","quality_score":25,"revenue":null,"mechanism":""}],"pipeline":[],"offLabel":[],"totalMarketed":2,"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":"NCT02171104","title":"MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis","phase":"PHASE2","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":149,"lead_sponsor_name":"Masonic Cancer Center, University of Minnesota","has_results":false},{"nct_id":"NCT05284006","title":"Non-invasive Functional Assessment and Pathogenesis of Morquio A","phase":"","overall_status":"RECRUITING","enrollment_count":60,"lead_sponsor_name":"Nemours Children's Clinic","has_results":false},{"nct_id":"NCT05073783","title":"A Study to Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care Setting","phase":"","overall_status":"COMPLETED","enrollment_count":57,"lead_sponsor_name":"Sanofi","has_results":false},{"nct_id":"NCT06022380","title":"Core Outcome Set for Head, Neck and Respiratory Disease in Mucopolysaccharidosis II","phase":"","overall_status":"COMPLETED","enrollment_count":46,"lead_sponsor_name":"Manchester University NHS Foundation Trust","has_results":false},{"nct_id":"NCT03161171","title":"Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III","phase":"","overall_status":"COMPLETED","enrollment_count":34,"lead_sponsor_name":"University Hospital Heidelberg","has_results":false},{"nct_id":"NCT03576729","title":"MRS to Determine Neuroinflammation and Oxidative Stress in MPS I","phase":"","overall_status":"COMPLETED","enrollment_count":30,"lead_sponsor_name":"University of Minnesota","has_results":false},{"nct_id":"NCT03580083","title":"RGX-111 Gene Therapy in Patients With MPS I","phase":"PHASE1, PHASE2","overall_status":"SUSPENDED","enrollment_count":21,"lead_sponsor_name":"REGENXBIO Inc.","has_results":false},{"nct_id":"NCT04539340","title":"A Multi-cohort Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers","phase":"PHASE1","overall_status":"COMPLETED","enrollment_count":20,"lead_sponsor_name":"AO GENERIUM","has_results":false},{"nct_id":"NCT01572636","title":"Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome","phase":"","overall_status":"TERMINATED","enrollment_count":20,"lead_sponsor_name":"Masonic Cancer Center, University of Minnesota","has_results":false},{"nct_id":"NCT06475404","title":"A Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers","phase":"PHASE1","overall_status":"COMPLETED","enrollment_count":18,"lead_sponsor_name":"AO GENERIUM","has_results":false},{"nct_id":"NCT05634512","title":"Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.","phase":"","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":13,"lead_sponsor_name":"Masonic Cancer Center, University of Minnesota","has_results":false},{"nct_id":"NCT03173521","title":"Gene Therapy in Patients With Mucopolysaccharidosis Disease","phase":"PHASE1, PHASE2","overall_status":"COMPLETED","enrollment_count":9,"lead_sponsor_name":"Fondazione Telethon","has_results":false},{"nct_id":"NCT04571970","title":"RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)","phase":"PHASE1, PHASE2","overall_status":"COMPLETED","enrollment_count":6,"lead_sponsor_name":"REGENXBIO Inc.","has_results":false},{"nct_id":"NCT06519552","title":"A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of JWK008 in Patients With Mucopolysaccharidosis Type I","phase":"PHASE1","overall_status":"RECRUITING","enrollment_count":6,"lead_sponsor_name":"West China Hospital","has_results":false},{"nct_id":"NCT04201405","title":"Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIA","phase":"PHASE1, PHASE2","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":5,"lead_sponsor_name":"University of Manchester","has_results":false},{"nct_id":"NCT02618512","title":"A Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously","phase":"PHASE1, PHASE2","overall_status":"TERMINATED","enrollment_count":3,"lead_sponsor_name":"Alexion Pharmaceuticals, Inc.","has_results":true},{"nct_id":"NCT02298712","title":"Biomarker for Hurler Disease (BioHurler)","phase":"","overall_status":"WITHDRAWN","enrollment_count":0,"lead_sponsor_name":"CENTOGENE GmbH Rostock","has_results":false}],"total":19},"guidelines":[],"source":"Drug Landscape verified database"}