Clinical trials
Phase 3 Rare Diseases trials in the United States
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
18 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 1 |
| 2010 | 2 |
| 2012 | 1 |
| 2013 | 2 |
| 2014 | 1 |
| 2015 | 1 |
| 2018 | 1 |
| 2021 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 12 |
| Terminated | 5 |
| Unknown status | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Amgen | 4 |
| National Center for Research Resources (NCRR) | 3 |
| National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | 3 |
| Ultragenyx Pharmaceutical Inc | 2 |
| AstraZeneca | 1 |
| Gradalis, Inc. | 1 |
| Novartis Pharmaceuticals | 1 |
| Roswell Park Cancer Institute | 1 |
| United Therapeutics | 1 |
| Windtree Therapeutics | 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 |
|---|---|
| Rare Diseases | 18 |
| Cystinosis | 4 |
| Metabolic Diseases | 4 |
| Communicable Diseases | 3 |
| Immune System Diseases | 3 |
| Herpes Simplex | 2 |
| Herpesviridae Infections | 2 |
| Lysosomal Storage Diseases | 2 |
| Mucopolysaccharidoses | 2 |
| Mucopolysaccharidosis VII | 2 |
Trials
Showing 1 to 18 of 18. 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.