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

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

Natural History Study of Participants With Sanfilippo Syndrome Type IIIC

NCT05825131 ·Status: RECRUITING

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

NCT05523206 ·Status: COMPLETED

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

NCT04088734 ·Status: TERMINATED ·Phase: PHASE1/PHASE2

Natural History Study of Patients With MPS IIIA

NCT02746341 ·Status: COMPLETED

A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)

NCT01047306 ·Status: COMPLETED

The Natural History Study of Patients With Sanfilippo Disease(s) (MPS3)

NCT05705674 ·Status: RECRUITING

Natural History Observational Study of MPS IIIa in SMC

NCT04918641 ·Status: UNKNOWN

Natural History Studies of Mucopolysaccharidosis III

NCT02037880 ·Status: COMPLETED

Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics

NCT01675674 ·Status: TERMINATED

A Treatment Study of Mucopolysaccharidosis Type IIIB

NCT02754076 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101

NCT04655911 ·Status: TERMINATED

Mucopolysaccharidosis Type II Observational

NCT04591834 ·Status: WITHDRAWN

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

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

NCT02716246 ·Status: RECRUITING ·Phase: PHASE2/PHASE3

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

NCT04201405 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2

Mucopolysaccharidosis Type II Natural History

NCT03529786 ·Status: COMPLETED

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I

NCT02702115 ·Status: TERMINATED ·Phase: PHASE1/PHASE2

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

NCT00787995 ·Status: TERMINATED

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

A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients

NCT03811028 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA

NCT01733615 ·Status: TERMINATED

A Treatment Extension Study of Mucopolysaccharidosis Type IIIB

NCT03784287 ·Status: UNKNOWN ·Phase: PHASE2

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

NCT05155488 ·Status: COMPLETED

A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C

NCT02435030 ·Status: COMPLETED