Hunter Outcome Survey (HOS)

Mucopolysaccharidosis Type II Observational

NCT04591834 ·Status: WITHDRAWN

The Long-term Safety Study of Idursulfase-beta in Hunter Syndrome(Mucopolysaccharidosis II) Patients

NCT02044692 ·Status: UNKNOWN

An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007

NCT06075537 ·Status: ENROLLING_BY_INVITATION ·Phase: PHASE2/PHASE3

Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase®

NCT00937794 ·Status: COMPLETED

Gaucher Disease Outcome Survey (GOS)

NCT03291223 ·Status: RECRUITING

Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Syndrome

NCT02262338 ·Status: COMPLETED ·Phase: PHASE1

A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome

NCT04251026 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2

Study of Idursulfase-beta (GC1111) in Hunter Syndrome

NCT02663024 ·Status: UNKNOWN ·Phase: PHASE2

Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase

NCT00630747 ·Status: COMPLETED ·Phase: PHASE2/PHASE3

RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

NCT04571970 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

Natural History Study of Patients With MPS IIIA

NCT02746341 ·Status: COMPLETED

An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects

NCT05594992 ·Status: ENROLLING_BY_INVITATION ·Phase: PHASE3

A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II

NCT05208281 ·Status: RECRUITING ·Phase: PHASE2/PHASE3

Fabry Outcome Survey (FOS)

NCT03289065 ·Status: COMPLETED

Biomarkers for Hunter Syndrome

NCT01330277 ·Status: TERMINATED

Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II

NCT05238324 ·Status: WITHDRAWN ·Phase: PHASE1

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

NCT01675674 ·Status: TERMINATED

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

NCT05155488 ·Status: COMPLETED

Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)

NCT01509768 ·Status: COMPLETED

A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ

NCT05422482 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1

Safety and Dose Ranging Study of Human Insulin Receptor MAb-IDUA Fusion Protein in Adults and Children With MPS I

NCT03053089 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy

NCT00607386 ·Status: COMPLETED ·Phase: PHASE4

Study of Metabolic Modifications in Children With Noonan Syndrome

NCT02383316 ·Status: COMPLETED ·Phase: NA

A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

NCT05371613 ·Status: RECRUITING ·Phase: PHASE2/PHASE3

Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I

NCT02371226 ·Status: COMPLETED ·Phase: PHASE1