Clinical trials
Lysosomal Storage Diseases trials in the United Kingdom
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
42 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2004 | 1 |
| 2006 | 1 |
| 2008 | 3 |
| 2009 | 2 |
| 2010 | 3 |
| 2011 | 7 |
| 2012 | 2 |
| 2013 | 2 |
| 2014 | 3 |
| 2015 | 2 |
| 2016 | 1 |
| 2018 | 2 |
| 2019 | 4 |
| 2020 | 3 |
| 2021 | 2 |
| 2022 | 2 |
| 2023 | 1 |
| 2026 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 27 |
| Terminated | 9 |
| Active, not recruiting | 2 |
| Recruiting | 2 |
| Suspended | 1 |
| Unknown status | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| BioMarin Pharmaceutical | 8 |
| Shire | 7 |
| Alexion Pharmaceuticals, Inc. | 6 |
| Gemma Biotherapeutics | 2 |
| LYSOGENE | 2 |
| Spark Therapeutics, Inc. | 2 |
| Spur Therapeutics | 2 |
| ZevraDenmark | 2 |
| Amicus Therapeutics | 1 |
| Azafaros B.V. | 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 | 42 |
| Mucopolysaccharidosis IV | 8 |
| Mucopolysaccharidosis II | 7 |
| Sudden Infant Death | 6 |
| Cholesterol Ester Storage Disease | 5 |
| Fabry Disease | 5 |
| Mucopolysaccharidosis III | 5 |
| Wolman Disease | 5 |
| Gangliosidosis, GM1 | 4 |
| Genetic Diseases, Inborn | 4 |
Trials
Showing 1 to 25 of 42. 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.
Go deeper on Pharma Observatory
Pharma Observatory connects clinical trials, sponsors, sites and investigators in one searchable platform.