Clinical trials
Niemann-Pick Disease, Type A trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
10 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2013 | 1 |
| 2014 | 2 |
| 2015 | 2 |
| 2018 | 1 |
| 2019 | 1 |
| 2023 | 2 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 6 |
| Active, not recruiting | 2 |
| Approved for marketing | 1 |
| Recruiting | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Genzyme, a Sanofi Company | 3 |
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 2 |
| Sanofi | 2 |
| Hubei Cancer Hospital | 1 |
| Joanne Kurtzberg, MD | 1 |
| RTI International | 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 |
|---|---|
| Niemann-Pick Disease, Type A | 10 |
| Leukodystrophy, Globoid Cell | 2 |
| Leukodystrophy, Metachromatic | 2 |
| Mucopolysaccharidosis II | 2 |
| Neuronal Ceroid-Lipofuscinoses | 2 |
| Niemann-Pick Disease, Type C | 2 |
| Smith-Lemli-Opitz Syndrome | 2 |
| Adrenoleukodystrophy | 1 |
| Anemia, Sickle Cell | 1 |
| Angelman Syndrome | 1 |
Trials
Showing 1 to 10 of 10. 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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