Natural History Observational Study of MPS IIIa in SMC

A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

NCT02493998 ·Status: COMPLETED

A Natural History Study of Biomarkers and Clinical Outcomes in Mucopolysaccharidosis Type IIIA (MPS IIIA; Sanfilippo Syndrome)

NCT05523206 ·Status: COMPLETED

Natural History Study of Participants With Sanfilippo Syndrome Type IIIC

NCT05825131 ·Status: RECRUITING

Natural History Study to Characterise the Course of Disease Progression in Participants With Mucopolysaccharidosis Type IIIB

NCT02293408 ·Status: TERMINATED

A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB

NCT02293382 ·Status: COMPLETED

Natural History Studies of Mucopolysaccharidosis III

NCT02037880 ·Status: COMPLETED

Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease

NCT02060526 ·Status: COMPLETED ·Phase: PHASE2

Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)

NCT06181136 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2

Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients

NCT01155778 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

Mucopolysaccharidosis Type II Observational

NCT04591834 ·Status: WITHDRAWN

A Natural History Study of Sanfilippo Syndrome Type D

NCT05648851 ·Status: COMPLETED

Mucopolysaccharidosis Type II Natural History

NCT03529786 ·Status: COMPLETED

A Treatment Study of Mucopolysaccharidosis Type IIIB

NCT02754076 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)

NCT00787995 ·Status: TERMINATED

Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIA

NCT04201405 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2

A Study to Improve the Awareness of Mucopolysaccharidosis Type II in Brazil

NCT05155488 ·Status: COMPLETED

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

NCT02618512 ·Status: TERMINATED ·Phase: PHASE1/PHASE2

Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)

NCT02324049 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)

NCT06567769 ·Status: RECRUITING ·Phase: PHASE1

Safety and Efficacy of Encapsulated Allogeneic MPS-1 Therapy

NCT05665036 ·Status: WITHDRAWN ·Phase: PHASE1/PHASE2

Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease

NCT04088734 ·Status: TERMINATED ·Phase: PHASE1/PHASE2

A Treatment Extension Study of Mucopolysaccharidosis Type IIIB

NCT03784287 ·Status: UNKNOWN ·Phase: PHASE2

A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients

NCT03423186 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

Natural History of Oculo-Pharyngeal Muscular Dystrophy (OPMD) - Israel National OPMD Registry

NCT07146256 ·Status: RECRUITING

Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA

NCT04360265 ·Status: ENROLLING_BY_INVITATION ·Phase: PHASE3