Clinical trials
Phase 1 Lysosomal Storage Diseases trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
10 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2005 | 1 |
| 2006 | 1 |
| 2008 | 2 |
| 2009 | 1 |
| 2011 | 1 |
| 2014 | 1 |
| 2020 | 1 |
| 2021 | 1 |
| 2023 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 5 |
| Terminated | 4 |
| Withdrawn | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| AO GENERIUM | 2 |
| Amicus Therapeutics | 2 |
| Patricia I. Dickson, M.D. | 2 |
| Duke University | 1 |
| Genzyme, a Sanofi Company | 1 |
| Joanne Kurtzberg, MD | 1 |
| Terence Flotte | 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 | 10 |
| Mucopolysaccharidoses | 4 |
| Mucopolysaccharidosis I | 3 |
| Mucopolysaccharidosis II | 3 |
| Fabry Disease | 2 |
| Gangliosidoses, GM2 | 2 |
| Genetic Diseases, Inborn | 2 |
| Genetic Diseases, X-Linked | 2 |
| Metabolic Diseases | 2 |
| Metabolism, Inborn Errors | 2 |
Trials
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Recently changed trials
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