Clinical trials
Completed Phase 2 Lysosomal Storage Diseases trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
7 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2004 | 1 |
| 2011 | 2 |
| 2014 | 2 |
| 2015 | 1 |
| 2018 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 7 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Alexion Pharmaceuticals, Inc. | 2 |
| BioMarin Pharmaceutical | 1 |
| Orchard Therapeutics | 1 |
| Shire | 1 |
| The Hospital for Sick Children | 1 |
| Ultragenyx Pharmaceutical Inc | 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 | 7 |
| Cholesterol Ester Storage Disease | 2 |
| Metabolic Diseases | 2 |
| Wolman Disease | 2 |
| Gangliosidoses, GM2 | 1 |
| Genetic Diseases, Inborn | 1 |
| Infant, Newborn, Diseases | 1 |
| Leukodystrophy, Metachromatic | 1 |
| Lipid Metabolism Disorders | 1 |
| Lipid Metabolism, Inborn Errors | 1 |
Trials
Showing 1 to 7 of 7. 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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