Clinical trials
Lysosomal Storage Diseases trials in the United States
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
87 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2002 | 2 |
| 2003 | 1 |
| 2004 | 1 |
| 2005 | 1 |
| 2006 | 5 |
| 2008 | 6 |
| 2009 | 5 |
| 2010 | 1 |
| 2011 | 8 |
| 2012 | 7 |
| 2013 | 2 |
| 2014 | 5 |
| 2015 | 4 |
| 2016 | 2 |
| 2017 | 2 |
| 2018 | 5 |
| 2019 | 6 |
| 2020 | 5 |
| 2021 | 3 |
| 2022 | 3 |
| 2023 | 2 |
| 2024 | 1 |
| 2025 | 1 |
| 2026 | 2 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 43 |
| Terminated | 17 |
| Recruiting | 8 |
| Unknown status | 7 |
| Active, not recruiting | 5 |
| No longer available | 2 |
| Not yet recruiting | 1 |
| Suspended | 1 |
| Enrolling by invitation | 1 |
| Approved for marketing | 1 |
| Withdrawn | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| BioMarin Pharmaceutical | 7 |
| Alexion Pharmaceuticals, Inc. | 6 |
| Shire | 6 |
| Amicus Therapeutics | 3 |
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 3 |
| LYSOGENE | 3 |
| National Human Genome Research Institute (NHGRI) | 3 |
| Patricia I. Dickson, M.D. | 3 |
| Ultragenyx Pharmaceutical Inc | 3 |
| Duke University | 2 |
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 | 87 |
| Metabolic Diseases | 16 |
| Metabolism, Inborn Errors | 15 |
| Genetic Diseases, Inborn | 11 |
| Mucopolysaccharidoses | 11 |
| Mucopolysaccharidosis II | 11 |
| Fabry Disease | 10 |
| Gaucher Disease | 10 |
| Mucopolysaccharidosis IV | 9 |
| Sudden Infant Death | 9 |
Trials
Showing 1 to 25 of 87. 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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