Clinical trials
Phase 1/2 Mucopolysaccharidosis III trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
19 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2010 | 1 |
| 2011 | 3 |
| 2013 | 2 |
| 2015 | 2 |
| 2016 | 1 |
| 2017 | 1 |
| 2018 | 1 |
| 2019 | 2 |
| 2020 | 2 |
| 2023 | 2 |
| 2024 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 9 |
| Terminated | 5 |
| Active, not recruiting | 4 |
| Not yet recruiting | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Alexion Pharmaceuticals, Inc. | 2 |
| JCR Pharmaceuticals Co., Ltd. | 2 |
| LYSOGENE | 2 |
| Shire | 2 |
| Swedish Orphan Biovitrum | 2 |
| Abeona Therapeutics, Inc | 1 |
| Allievex Corporation | 1 |
| Denali Therapeutics Inc. | 1 |
| Lynda E Polgreen | 1 |
| NeuroGT | 1 |
Top conditions
MeSH conditions the trials were registered with, not their broader categories. A condition with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Mucopolysaccharidosis III | 19 |
| Genetic Diseases, Inborn | 4 |
| Lysosomal Storage Diseases | 4 |
| Metabolic Diseases | 4 |
| Mucopolysaccharidoses | 3 |
| Carbohydrate Metabolism, Inborn Errors | 2 |
| Connective Tissue Diseases | 2 |
| Metabolism, Inborn Errors | 2 |
| Mucinoses | 2 |
| Neurodegenerative Diseases | 2 |
Trials
Showing 26 to 19 of 19. Each trial links to its page on Pharma Observatory.
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Recently changed trials
Changes observed in the last 7 days, newest first, site-count changes left out. Source: ClinicalTrials.gov.
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