Clinical trials
Phase 2 Lysosomal Storage Diseases trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
13 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2004 | 1 |
| 2011 | 3 |
| 2012 | 2 |
| 2014 | 2 |
| 2015 | 1 |
| 2018 | 1 |
| 2023 | 1 |
| 2024 | 1 |
| 2026 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 7 |
| Terminated | 2 |
| Not yet recruiting | 1 |
| Recruiting | 1 |
| Unknown status | 1 |
| Withdrawn | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| BioMarin Pharmaceutical | 3 |
| Alexion Pharmaceuticals, Inc. | 2 |
| McMaster University | 1 |
| National Cancer Institute (NCI) | 1 |
| Orchard Therapeutics | 1 |
| Polaryx Therapeutics, Inc. | 1 |
| Rocket Pharmaceuticals Inc. | 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 | 13 |
| Mucopolysaccharidosis IV | 3 |
| Cholesterol Ester Storage Disease | 2 |
| Metabolic Diseases | 2 |
| Sandhoff Disease | 2 |
| Wolman Disease | 2 |
| Cardiomyopathies | 1 |
| Cardiomyopathy, Hypertrophic | 1 |
| Gangliosidoses, GM2 | 1 |
| Genetic Diseases, Inborn | 1 |
Trials
Showing 76 to 13 of 13. Each trial links to its page on Pharma Observatory.
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Changes observed in the last 7 days, newest first, site-count changes left out. Source: ClinicalTrials.gov.
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