Clinical trials
Lysosomal Storage Diseases trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
123 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2002 | 2 |
| 2003 | 1 |
| 2004 | 2 |
| 2005 | 1 |
| 2006 | 5 |
| 2007 | 1 |
| 2008 | 6 |
| 2009 | 6 |
| 2010 | 4 |
| 2011 | 10 |
| 2012 | 7 |
| 2013 | 3 |
| 2014 | 6 |
| 2015 | 6 |
| 2016 | 2 |
| 2017 | 2 |
| 2018 | 12 |
| 2019 | 7 |
| 2020 | 9 |
| 2021 | 6 |
| 2022 | 5 |
| 2023 | 3 |
| 2024 | 3 |
| 2025 | 2 |
| 2026 | 4 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 56 |
| Terminated | 21 |
| Recruiting | 12 |
| Unknown status | 10 |
| Active, not recruiting | 8 |
| Withdrawn | 8 |
| No longer available | 3 |
| Not yet recruiting | 2 |
| Suspended | 1 |
| Enrolling by invitation | 1 |
| Approved for marketing | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| BioMarin Pharmaceutical | 9 |
| Shire | 9 |
| Alexion Pharmaceuticals, Inc. | 7 |
| CENTOGENE GmbH Rostock | 6 |
| Amicus Therapeutics | 4 |
| Ultragenyx Pharmaceutical Inc | 4 |
| AO GENERIUM | 3 |
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 3 |
| LYSOGENE | 3 |
| National Human Genome Research Institute (NHGRI) | 3 |
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 | 123 |
| Metabolic Diseases | 24 |
| Fabry Disease | 18 |
| Genetic Diseases, Inborn | 17 |
| Metabolism, Inborn Errors | 16 |
| Mucopolysaccharidosis II | 15 |
| Mucopolysaccharidoses | 14 |
| Gaucher Disease | 13 |
| Mucopolysaccharidosis IV | 11 |
| Sudden Infant Death | 11 |
Trials
Showing 26 to 50 of 123. Each trial links to its page on Pharma Observatory.
23 more trials match. See them 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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