Clinical trials
Completed Phase 3 Rare Diseases trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
53 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2010 | 2 |
| 2012 | 1 |
| 2013 | 1 |
| 2014 | 1 |
| 2015 | 2 |
| 2016 | 1 |
| 2018 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 53 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| National Center for Research Resources (NCRR) | 24 |
| Amgen | 4 |
| National Institute of Neurological Disorders and Stroke (NINDS) | 4 |
| FDA Office of Orphan Products Development | 3 |
| National Institute of Allergy and Infectious Diseases (NIAID) | 2 |
| National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | 2 |
| Ultragenyx Pharmaceutical Inc | 2 |
| AO GENERIUM | 1 |
| Assistance Publique - Hôpitaux de Paris | 1 |
| Brigham and Women's Hospital | 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 | 53 |
| Mental Disorders | 13 |
| Neurologic Manifestations | 13 |
| Communicable Diseases | 10 |
| Immune System Diseases | 10 |
| Genetic Diseases, Inborn | 5 |
| Respiratory Tract Diseases | 5 |
| Virus Diseases | 5 |
| Bacterial Infections | 4 |
| Cystinosis | 4 |
Trials
Showing 1 to 25 of 53. 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.