Clinical trials
Completed Lysosomal Storage Diseases trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
56 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2003 | 1 |
| 2004 | 2 |
| 2006 | 2 |
| 2007 | 1 |
| 2008 | 3 |
| 2009 | 4 |
| 2010 | 3 |
| 2011 | 8 |
| 2012 | 2 |
| 2013 | 2 |
| 2014 | 5 |
| 2015 | 4 |
| 2016 | 1 |
| 2017 | 2 |
| 2018 | 3 |
| 2019 | 4 |
| 2020 | 4 |
| 2022 | 1 |
| 2023 | 1 |
| 2025 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 56 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Shire | 7 |
| Alexion Pharmaceuticals, Inc. | 6 |
| Amicus Therapeutics | 4 |
| BioMarin Pharmaceutical | 4 |
| Ultragenyx Pharmaceutical Inc | 4 |
| AO GENERIUM | 2 |
| Duke University | 2 |
| National Institute of Neurological Disorders and Stroke (NINDS) | 2 |
| Orchard Therapeutics | 2 |
| University of California, San Diego | 2 |
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 |
|---|---|
| Lysosomal Storage Diseases | 56 |
| Metabolic Diseases | 12 |
| Mucopolysaccharidosis II | 11 |
| Mucopolysaccharidoses | 9 |
| Genetic Diseases, Inborn | 8 |
| Metabolism, Inborn Errors | 7 |
| Sudden Infant Death | 7 |
| Wolman Disease | 6 |
| Cholesterol Ester Storage Disease | 5 |
| Gaucher Disease | 5 |
Trials
Showing 51 to 56 of 56. 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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