Clinical trials
Phase 1/2 Lysosomal Storage Diseases trials in the United States
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 |
|---|---|
| 2009 | 1 |
| 2010 | 1 |
| 2011 | 1 |
| 2015 | 1 |
| 2018 | 1 |
| 2019 | 3 |
| 2020 | 2 |
| 2021 | 2 |
| 2022 | 1 |
| 2023 | 1 |
| 2025 | 1 |
| 2026 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 6 |
| Active, not recruiting | 4 |
| Terminated | 3 |
| Recruiting | 2 |
| Not yet recruiting | 1 |
| Suspended | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Alexion Pharmaceuticals, Inc. | 2 |
| Gemma Biotherapeutics | 2 |
| 4D Molecular Therapeutics | 1 |
| AVROBIO | 1 |
| AskBio Inc | 1 |
| Forge Biologics, Inc | 1 |
| LYSOGENE | 1 |
| NeuroGT | 1 |
| Novartis Pharmaceuticals | 1 |
| Sangamo Therapeutics | 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 |
| Fabry Disease | 4 |
| Genetic Diseases, Inborn | 4 |
| Metabolic Diseases | 4 |
| Metabolism, Inborn Errors | 3 |
| Mucopolysaccharidoses | 3 |
| Brain Diseases, Metabolic | 2 |
| Carbohydrate Metabolism, Inborn Errors | 2 |
| Central Nervous System Diseases | 2 |
| Connective Tissue Diseases | 2 |
Trials
Showing 1 to 17 of 17. 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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