alpha-mannosidosis
Information
- Disease name
- alpha-mannosidosis
- Disease ID
- DOID:3413
- Description
- "A lysosomal storage disease that has_material_basis_in deficiency of the alpha-D-manosidase enzyme resulting in the impairment of cell function from a build up of complex sugars derived from glycoproteins in the lysosome." [url:http\://en.wikipedia.org/wiki/Alpha-mannosidosis]
Disease area statistics
[No Data.]
Chromosome band
[No Data.]
Annotation
| Genes | Mutation | Description | Source | Links |
|---|
| NCT ID | Status | Phase | Summary | Start date | Completion date |
|---|---|---|---|---|---|
| NCT04959240 | Available | Expanded Access to Velmanase Alfa | |||
| NCT01681953 | Completed | Phase 3 | A Placebo-Controlled Phase 3 Trial of Repeated Lamazym Treatment of Subjects With Alpha-Mannosidosis | August 2012 | May 2014 |
| NCT01908712 | Completed | Phase 3 | Lamazym Aftercare Study FR Designed to Provide Treatment for French Patients | August 2013 | September 30, 2022 |
| NCT01908725 | Completed | Phase 3 | Lamazym Aftercare Study | June 2013 | September 30, 2022 |
| NCT02141503 | Completed | Clinical Biomarkers in Alpha-mannosidosis | July 24, 2014 | November 29, 2019 | |
| NCT01681940 | Completed | Phase 2 | Long-term Efficacy and Safety of Lamazym for the Treatment of Patients With Alpha-Mannosidosis | January 2012 | September 2013 |
| NCT02478840 | Completed | Phase 3 | Evaluation of Long-term Efficacy of Treatment With Lamazym | February 2015 | June 2015 |
| NCT02998879 | Completed | Phase 2 | Trial on Safety and Efficacy of Velmanase Alfa Treatment in Pediatric Patients With Alpha-Mannosidosis | December 2016 | July 2020 |
| NCT06184503 | Not yet recruiting | Analysis of Velmanase Alfa (Lamzede®)'s Effects in the Body of Children With Alpha-Mannosidosis Under the Age 3 | May 2024 | September 2029 | |
| NCT02254863 | Recruiting | Phase 1 | UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells | September 2014 | October 2024 |
| NCT02171104 | Recruiting | Phase 2 | MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis | July 10, 2014 | July 14, 2028 |
| NCT03333200 | Recruiting | Longitudinal Study of Neurodegenerative Disorders | January 11, 2012 | January 2035 | |
| NCT01372228 | Terminated | Phase 1/Phase 2 | Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders | April 2011 | April 2016 |
| NCT04031066 | Withdrawn | Phase 3 | Interventional Study to Assess Efficacy and Safety of Velmanase Alfa in Patients With Alpha Mannosidosis | January 11, 2021 | December 29, 2021 |
- Disase is a (Disease Ontology)
- DOID:3211
- Cross Reference ID (Disease Ontology)
- GARD:6968
- Cross Reference ID (Disease Ontology)
- MESH:D008363
- Cross Reference ID (Disease Ontology)
- MIM:248500
- Cross Reference ID (Disease Ontology)
- NCI:C84548
- Cross Reference ID (Disease Ontology)
- SNOMEDCT_US_2023_03_01:65524005
- Cross Reference ID (Disease Ontology)
- UMLS_CUI:C0024748
- Exact Synonym (Disease Ontology)
- Alpha-D-mannosidosis
- Exact Synonym (Disease Ontology)
- alpha-mannosidase deficiency
- Exact Synonym (Disease Ontology)
- deficiency of alpha-mannosidase
- OrphaNumber from OrphaNet (Orphanet)
- 61
- MeSH unique ID (MeSH (Medical Subject Headings))
- D008363