Clinical trials
Active, not recruiting Lysosomal Storage Diseases trials
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 |
|---|---|
| 2018 | 1 |
| 2020 | 2 |
| 2021 | 2 |
| 2022 | 1 |
| 2023 | 1 |
| 2024 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Active, not recruiting | 8 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| 4D Molecular Therapeutics | 1 |
| AO GENERIUM | 1 |
| Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico | 1 |
| Forge Biologics, Inc | 1 |
| Gemma Biotherapeutics | 1 |
| Orchard Therapeutics | 1 |
| REGENXBIO Inc. | 1 |
| Spark Therapeutics, 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 | 8 |
| Genetic Diseases, Inborn | 3 |
| Metabolic Diseases | 3 |
| Nervous System Diseases | 3 |
| Brain Diseases, Metabolic | 2 |
| Central Nervous System Diseases | 2 |
| Lipid Metabolism Disorders | 2 |
| Lipidoses | 2 |
| Mucopolysaccharidosis II | 2 |
| Sphingolipidoses | 2 |
Trials
Showing 51 to 8 of 8. Each trial links to its page on Pharma Observatory.
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Recently changed trials
Changes observed in the last 7 days, newest first, site-count changes left out. Source: ClinicalTrials.gov.
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