mucopolysaccharidosis II
Information
- Disease name
- mucopolysaccharidosis II
- Disease ID
- DOID:12799
- Description
- "A mucopolysaccharidosis characterized by a deficiency of the lysosomal enzyme iduronate sulfatase." [url:http\://en.wikipedia.org/wiki/Hunter_syndrome]
Disease area statistics
[No Data.]
Chromosome band
[No Data.]
Annotation
| Genes | Mutation | Description | Source | Links |
|---|
| NCT ID | Status | Phase | Summary | Start date | Completion date |
|---|---|---|---|---|---|
| NCT04628871 | Active, not recruiting | Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX | November 3, 2020 | January 1, 2030 | |
| NCT04251026 | Active, not recruiting | Phase 1/Phase 2 | A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome | July 16, 2020 | July 2027 |
| NCT03708965 | Active, not recruiting | Phase 2 | An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II | January 1, 2019 | March 31, 2024 |
| NCT04348136 | Active, not recruiting | Phase 2/Phase 3 | An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II | September 1, 2019 | March 31, 2030 |
| NCT03153319 | Active, not recruiting | Phase 1/Phase 2 | Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI | June 5, 2017 | June 2026 |
| NCT02262338 | Completed | Phase 1 | Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Syndrome | April 2015 | March 27, 2017 |
| NCT00004454 | Completed | Phase 1/Phase 2 | Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome) | October 1996 | |
| NCT00069641 | Completed | Phase 2/Phase 3 | Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II) | September 18, 2003 | March 16, 2005 |
| NCT00607386 | Completed | Phase 4 | Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy | December 31, 2007 | July 8, 2011 |
| NCT01301898 | Completed | Phase 1/Phase 2 | To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients | May 2010 | March 2011 |
| NCT01602601 | Completed | A Study to Test the Possibility of Cross Reaction Induced by the Idursulfase Drug to GSK2788723 | April 9, 2012 | June 11, 2012 | |
| NCT03128593 | Completed | Phase 1/Phase 2 | A Study of JR-141 in Patients With Mucopolysaccharidosis Type II | March 30, 2017 | October 4, 2017 |
| NCT03359213 | Completed | Phase 2 | A Study of JR-141 in Patients With Mucopolysaccharidosis II | July 26, 2018 | October 4, 2019 |
| NCT03529786 | Completed | Mucopolysaccharidosis Type II Natural History | September 27, 2017 | March 22, 2022 | |
| NCT03568175 | Completed | Phase 2/Phase 3 | A Study of JR-141 in Patients With Mucopolysaccharidosis II | August 1, 2018 | February 20, 2020 |
| NCT04007536 | Completed | A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome | October 23, 2019 | March 1, 2024 | |
| NCT05368038 | Enrolling by invitation | ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program | May 10, 2021 | July 31, 2026 | |
| NCT06075537 | Enrolling by invitation | Phase 2/Phase 3 | 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 | September 20, 2023 | June 2027 |
| NCT04597385 | Enrolling by invitation | Long-term Follow-Up for RGX-121 | March 14, 2021 | September 2025 | |
| NCT05594992 | Enrolling by invitation | Phase 3 | An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in Mucopolysaccharidosis Type II (Hunter Syndrome) Subjects | February 22, 2023 | February 28, 2028 |
| NCT06036693 | Recruiting | MPS (RaDiCo Cohort) (RaDiCo-MPS) | December 20, 2017 | December 20, 2024 | |
| NCT02254863 | Recruiting | Phase 1 | UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells | September 2014 | October 2024 |
| NCT04573023 | Recruiting | Phase 3 | A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT) | February 14, 2022 | January 31, 2026 |
| NCT04976231 | Recruiting | MPS II Immunophenotyping | April 1, 2022 | April 2025 | |
| NCT05371613 | Recruiting | Phase 2/Phase 3 | A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II) | July 21, 2022 | December 2025 |
| NCT05422482 | Recruiting | Phase 1 | A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients With MPS Ⅱ | September 20, 2022 | June 2026 |
| NCT05619900 | Recruiting | Registry of Patients Diagnosed With Lysosomal Storage Diseases | May 31, 2022 | May 31, 2050 | |
| NCT05665166 | Recruiting | Phase 1/Phase 2 | Autologous Ex-vivo Gene Modified HSCT in MPSII | June 1, 2023 | December 2026 |
| NCT05687474 | Recruiting | Baby Detect : Genomic Newborn Screening | September 1, 2022 | August 31, 2025 | |
| NCT01675674 | Terminated | Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics | September 2011 | March 2014 | |
| NCT00748969 | Terminated | Phase 2/Phase 3 | Clinical Trial of Growth Hormone in MPS I, II, and VI | November 2008 | September 2013 |
| NCT01330277 | Terminated | Biomarkers for Hunter Syndrome | August 20, 2018 | December 31, 2022 | |
| NCT03041324 | Terminated | Phase 1/Phase 2 | Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II | May 11, 2017 | May 7, 2021 |
| NCT02663024 | Unknown status | Phase 2 | Study of Idursulfase-beta (GC1111) in Hunter Syndrome | December 2016 | June 2020 |
| NCT04591834 | Withdrawn | Mucopolysaccharidosis Type II Observational | March 2022 | July 2025 | |
| NCT05238324 | Withdrawn | Phase 1 | Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II | September 8, 2022 | January 2029 |
- Disase is a (Disease Ontology)
- DOID:12798
- Cross Reference ID (Disease Ontology)
- GARD:6675
- Cross Reference ID (Disease Ontology)
- ICD10CM:E76.1
- Cross Reference ID (Disease Ontology)
- MESH:D016532
- Cross Reference ID (Disease Ontology)
- MIM:309900
- Cross Reference ID (Disease Ontology)
- NCI:C61260
- Cross Reference ID (Disease Ontology)
- SNOMEDCT_US_2023_03_01:190936000
- Cross Reference ID (Disease Ontology)
- UMLS_CUI:C0026705
- Exact Synonym (Disease Ontology)
- deficiency of iduronate-2-sulphatase
- Exact Synonym (Disease Ontology)
- Hunter syndrome
- Exact Synonym (Disease Ontology)
- Hunter's syndrome
- Exact Synonym (Disease Ontology)
- MPS II - Hunter syndrome
- Exact Synonym (Disease Ontology)
- Mucopolysaccharidosis, MPS-II
- MeSH unique ID (MeSH (Medical Subject Headings))
- D016532