{"disease":{"id":"mucopolysaccharidosis-iii","name":"mucopolysaccharidosis iii"},"drugs":{"marketed":[],"pipeline":[],"offLabel":[],"totalMarketed":0,"totalPipeline":0},"trials":{"data":[{"nct_id":"NCT05368038","title":"ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program","phase":"","overall_status":"ENROLLING_BY_INVITATION","enrollment_count":100000,"lead_sponsor_name":"Albert Einstein College of Medicine","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":"NCT04018755","title":"Open-label Study of Anakinra in MPS III","phase":"PHASE1, PHASE2","overall_status":"COMPLETED","enrollment_count":24,"lead_sponsor_name":"Lynda E Polgreen","has_results":false},{"nct_id":"NCT06095388","title":"Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA","phase":"PHASE1, PHASE2","overall_status":"ACTIVE_NOT_RECRUITING","enrollment_count":15,"lead_sponsor_name":"JCR Pharmaceuticals Co., Ltd.","has_results":false},{"nct_id":"NCT05523206","title":"A Natural History Study of Biomarkers and Clinical Outcomes in Mucopolysaccharidosis Type IIIA (MPS IIIA; Sanfilippo Syndrome)","phase":"","overall_status":"COMPLETED","enrollment_count":15,"lead_sponsor_name":"Sanguine Biosciences","has_results":false},{"nct_id":"NCT02324049","title":"Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)","phase":"PHASE1, PHASE2","overall_status":"COMPLETED","enrollment_count":11,"lead_sponsor_name":"Alexion Pharmaceuticals, Inc.","has_results":true},{"nct_id":"NCT06488924","title":"An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB","phase":"PHASE1, PHASE2","overall_status":"RECRUITING","enrollment_count":10,"lead_sponsor_name":"JCR Pharmaceuticals Co., Ltd.","has_results":false},{"nct_id":"NCT04088734","title":"Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease","phase":"PHASE1, PHASE2","overall_status":"TERMINATED","enrollment_count":5,"lead_sponsor_name":"Ultragenyx Pharmaceutical Inc","has_results":true},{"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":"NCT04655911","title":"A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101","phase":"","overall_status":"TERMINATED","enrollment_count":1,"lead_sponsor_name":"Abeona Therapeutics, Inc","has_results":false}],"total":11},"guidelines":[],"source":"Drug Landscape verified database"}