Clinical trials
Completed Phase 1 Lysosomal Storage Diseases trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
5 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2008 | 1 |
| 2011 | 1 |
| 2014 | 1 |
| 2020 | 1 |
| 2023 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 5 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| AO GENERIUM | 2 |
| Amicus Therapeutics | 2 |
| Duke University | 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 | 5 |
| Mucopolysaccharidosis II | 3 |
| Fabry Disease | 2 |
| Genetic Diseases, Inborn | 2 |
| Genetic Diseases, X-Linked | 2 |
| Metabolic Diseases | 2 |
| Mucopolysaccharidoses | 2 |
| Neurobehavioral Manifestations | 2 |
| Neurologic Manifestations | 2 |
| Anemia, Diamond-Blackfan | 1 |
Trials
Showing 1 to 5 of 5. 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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