Clinical trials
Niemann-Pick Disease, Type C trials in the United States
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
23 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2006 | 1 |
| 2008 | 1 |
| 2009 | 1 |
| 2011 | 1 |
| 2013 | 1 |
| 2014 | 1 |
| 2015 | 2 |
| 2016 | 1 |
| 2017 | 1 |
| 2018 | 2 |
| 2019 | 3 |
| 2020 | 1 |
| 2021 | 2 |
| 2022 | 2 |
| 2025 | 2 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 9 |
| Recruiting | 5 |
| Terminated | 4 |
| Unknown status | 2 |
| Enrolling by invitation | 1 |
| Available | 1 |
| Active, not recruiting | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 5 |
| Cyclo Therapeutics, Inc. | 3 |
| Mandos LLC | 3 |
| Azafaros B.V. | 2 |
| IntraBio Inc | 2 |
| ZevraDenmark | 2 |
| Albert Einstein College of Medicine | 1 |
| Masonic Cancer Center, University of Minnesota | 1 |
| Mayo Clinic | 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 C | 23 |
| Disease | 3 |
| Leukodystrophy, Metachromatic | 3 |
| Mucopolysaccharidosis VI | 3 |
| Mucopolysaccharidosis VII | 3 |
| Wolman Disease | 3 |
| Cognitive Dysfunction | 2 |
| Deglutition Disorders | 2 |
| Gangliosidosis, GM1 | 2 |
| Leukodystrophy, Globoid Cell | 2 |
Trials
Showing 1 to 23 of 23. 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.
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