Clinical trials
Completed Observational Lysosomal Storage Diseases trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
17 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2003 | 1 |
| 2006 | 2 |
| 2008 | 1 |
| 2009 | 1 |
| 2011 | 1 |
| 2012 | 1 |
| 2015 | 1 |
| 2017 | 1 |
| 2018 | 1 |
| 2019 | 1 |
| 2020 | 3 |
| 2022 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 17 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| National Institute of Neurological Disorders and Stroke (NINDS) | 2 |
| Alexion Pharmaceuticals, Inc. | 1 |
| Amicus Therapeutics | 1 |
| Azafaros B.V. | 1 |
| CENTOGENE GmbH Rostock | 1 |
| Denali Therapeutics Inc. | 1 |
| Duke University | 1 |
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 1 |
| Greenwood Genetic Center | 1 |
| LYSOGENE | 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 |
|---|---|
| Lysosomal Storage Diseases | 17 |
| Genetic Diseases, Inborn | 4 |
| Gangliosidosis, GM1 | 3 |
| Gaucher Disease | 3 |
| Metabolic Diseases | 3 |
| Metabolism, Inborn Errors | 3 |
| Sphingolipidoses | 3 |
| Lipid Metabolism Disorders | 2 |
| Lipidoses | 2 |
| Mucopolysaccharidosis II | 2 |
Trials
Showing 1 to 17 of 17. Each trial links to its page on Pharma Observatory.
Recently changed trials
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No changes to these trials in the last 7 days.
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