| Enrolling By Invitation | An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Partic NCT06075537 | Denali Therapeutics Inc. | Phase 2 / Phase 3 |
| Active Not Recruiting | Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type II NCT05665166 | University of Manchester | Phase 1 / Phase 2 |
| Enrolling By Invitation | An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subject NCT05594992 | JCR Pharmaceuticals Co., Ltd. | Phase 3 |
| Active Not Recruiting | A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS NCT05422482 | GC Biopharma Corp | Phase 1 |
| Withdrawn | Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II NCT05238324 | Homology Medicines, Inc | Phase 1 |
| Completed | Baby Detect : Genomic Newborn Screening NCT05687474 | Centre Hospitalier Universitaire de Liege | — |
| Recruiting | A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and You NCT05371613 | Denali Therapeutics Inc. | Phase 2 / Phase 3 |
| Recruiting | Registry of Patients Diagnosed With Lysosomal Storage Diseases NCT05619900 | University of California, San Francisco | — |
| Terminated | MPS II Immunophenotyping NCT04976231 | Duke University | — |
| Withdrawn | Mucopolysaccharidosis Type II Observational NCT04591834 | REGENXBIO Inc. | — |
| Active Not Recruiting | A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT) NCT04573023 | JCR Pharmaceuticals Co., Ltd. | Phase 3 |
| Enrolling By Invitation | ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program NCT05368038 | Albert Einstein College of Medicine | — |
| Unknown | Long-term Follow-Up for RGX-121 NCT04597385 | REGENXBIO Inc. | — |
| Active Not Recruiting | Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX NCT04628871 | Sangamo Therapeutics | — |
| Active Not Recruiting | A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome NCT04251026 | Denali Therapeutics Inc. | Phase 1 / Phase 2 |
| Completed | A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome NCT04007536 | Denali Therapeutics Inc. | — |
| Active Not Recruiting | An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II NCT04348136 | JCR Pharmaceuticals Co., Ltd. | Phase 2 / Phase 3 |
| Active Not Recruiting | An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II NCT03708965 | JCR Pharmaceuticals Co., Ltd. | Phase 2 |
| Terminated | Biomarkers for Hunter Syndrome NCT01330277 | CENTOGENE GmbH Rostock | — |
| Completed | A Study of JR-141 in Patients With Mucopolysaccharidosis II NCT03568175 | JCR Pharmaceuticals Co., Ltd. | Phase 2 / Phase 3 |
| Completed | A Study of JR-141 in Patients With Mucopolysaccharidosis II (BR21) NCT03359213 | JCR Pharmaceuticals Co., Ltd. | Phase 2 |
| Recruiting | MPS (RaDiCo Cohort) (RaDiCo-MPS) NCT06036693 | Institut National de la Santé Et de la Recherche Médicale, France | — |
| Completed | Mucopolysaccharidosis Type II Natural History NCT03529786 | REGENXBIO Inc. | — |
| Active Not Recruiting | Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI NCT03153319 | Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center | Phase 1 / Phase 2 |
| Terminated | Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With M NCT03041324 | Sangamo Therapeutics | Phase 1 / Phase 2 |
| Completed | A Study of JR-141 in Patients With Mucopolysaccharidosis Type II NCT03128593 | JCR Pharmaceuticals Co., Ltd. | Phase 1 / Phase 2 |
| Unknown | Study of Idursulfase-beta (GC1111) in Hunter Syndrome NCT02663024 | Green Cross Corporation | Phase 2 |
| Completed | Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Syndrome NCT02262338 | ArmaGen, Inc | Phase 1 |
| Recruiting | UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte- NCT02254863 | Joanne Kurtzberg, MD | Phase 1 |
| Completed | A Study to Test the Possibility of Cross Reaction Induced by the Idursulfase Drug to GSK2788723 NCT01602601 | GlaxoSmithKline | — |
| Terminated | Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplas NCT01675674 | National MPS Society | — |
| Completed | To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Pat NCT01301898 | Green Cross Corporation | Phase 1 / Phase 2 |
| Terminated | Clinical Trial of Growth Hormone in MPS I, II, and VI NCT00748969 | Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center | Phase 2 / Phase 3 |
| Completed | Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Ther NCT00607386 | Shire | Phase 4 |
| Completed | Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II) NCT00069641 | Shire | Phase 2 / Phase 3 |
| Completed | Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients Wi NCT00004454 | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Phase 1 / Phase 2 |