Clinical trials
Completed Rare Diseases trials in the United States
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
141 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 15 |
| 2001 | 4 |
| 2003 | 1 |
| 2004 | 1 |
| 2006 | 2 |
| 2009 | 2 |
| 2010 | 2 |
| 2012 | 1 |
| 2013 | 3 |
| 2014 | 2 |
| 2015 | 2 |
| 2016 | 3 |
| 2017 | 3 |
| 2018 | 2 |
| 2019 | 2 |
| 2020 | 3 |
| 2021 | 2 |
| 2022 | 1 |
| 2024 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 141 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| National Center for Research Resources (NCRR) | 38 |
| National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | 7 |
| Amgen | 5 |
| Icahn School of Medicine at Mount Sinai | 5 |
| Fred Hutchinson Cancer Center | 4 |
| Ultragenyx Pharmaceutical Inc | 4 |
| Children's Hospital Medical Center, Cincinnati | 3 |
| Fairview University Medical Center | 3 |
| Johns Hopkins University | 3 |
| National Institute of Neurological Disorders and Stroke (NINDS) | 3 |
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 | 141 |
| Genetic Diseases, Inborn | 23 |
| Mental Disorders | 19 |
| Neurologic Manifestations | 19 |
| Hematologic Diseases | 16 |
| Respiratory Tract Diseases | 14 |
| Metabolism, Inborn Errors | 13 |
| Connective Tissue Diseases | 12 |
| Endocrine System Diseases | 12 |
| Arthritis | 11 |
Trials
Showing 1 to 25 of 141. Each trial links to its page on Pharma Observatory.
41 more trials match. See them 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.