Clinical trials
Recruiting Lysosomal Storage Diseases trials in the United States
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
8 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2002 | 1 |
| 2006 | 1 |
| 2012 | 1 |
| 2022 | 1 |
| 2023 | 1 |
| 2025 | 1 |
| 2026 | 2 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Recruiting | 8 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| AskBio Inc | 1 |
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 1 |
| JCR Pharmaceuticals Co., Ltd. | 1 |
| National Human Genome Research Institute (NHGRI) | 1 |
| Novartis Pharmaceuticals | 1 |
| Rocket Pharmaceuticals Inc. | 1 |
| University of California, San Francisco | 1 |
| University of Pittsburgh | 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 | 8 |
| Gaucher Disease | 2 |
| Metabolism, Inborn Errors | 2 |
| Mucopolysaccharidosis IV | 2 |
| Brain Diseases, Metabolic, Inborn | 1 |
| Carbohydrate Metabolism, Inborn Errors | 1 |
| Cardiomyopathies | 1 |
| Cardiomyopathy, Hypertrophic | 1 |
| Congenital, Hereditary, and Neonatal Diseases and Abnormalities | 1 |
| Cystinosis | 1 |
Trials
Showing 1 to 8 of 8. 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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