Clinical trials
Mucopolysaccharidosis III trials in the United States
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
43 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2006 | 1 |
| 2008 | 1 |
| 2009 | 1 |
| 2010 | 2 |
| 2011 | 1 |
| 2012 | 4 |
| 2013 | 1 |
| 2014 | 3 |
| 2015 | 3 |
| 2016 | 2 |
| 2017 | 2 |
| 2018 | 4 |
| 2019 | 2 |
| 2020 | 3 |
| 2021 | 1 |
| 2022 | 2 |
| 2023 | 3 |
| 2024 | 3 |
| 2026 | 2 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 18 |
| Terminated | 7 |
| Recruiting | 5 |
| Unknown status | 4 |
| Active, not recruiting | 3 |
| Enrolling by invitation | 3 |
| Not yet recruiting | 2 |
| Available | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Allievex Corporation | 5 |
| Shire | 4 |
| Alexion Pharmaceuticals, Inc. | 3 |
| Phoenix Nest | 3 |
| Ultragenyx Pharmaceutical Inc | 3 |
| Abeona Therapeutics, Inc | 2 |
| Masonic Cancer Center, University of Minnesota | 2 |
| Swedish Orphan Biovitrum | 2 |
| University of Minnesota | 2 |
| Albert Einstein College of Medicine | 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 | 43 |
| Mucopolysaccharidoses | 10 |
| Leukodystrophy, Globoid Cell | 7 |
| Lysosomal Storage Diseases | 7 |
| Leukodystrophy, Metachromatic | 6 |
| Mucopolysaccharidosis II | 6 |
| Mucopolysaccharidosis I | 5 |
| Mucopolysaccharidosis VI | 5 |
| Sandhoff Disease | 5 |
| Sudden Infant Death | 5 |
Trials
Showing 26 to 43 of 43. Each trial links to its page on Pharma Observatory.
Recently changed trials
Changes observed in the last 7 days, newest first, site-count changes left out. Source: ClinicalTrials.gov.
No changes to these trials in the last 7 days.
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