Clinical trials
Completed Rare Diseases trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
352 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 21 |
| 2001 | 4 |
| 2003 | 2 |
| 2004 | 1 |
| 2006 | 4 |
| 2007 | 1 |
| 2009 | 3 |
| 2010 | 2 |
| 2011 | 1 |
| 2012 | 2 |
| 2013 | 6 |
| 2014 | 3 |
| 2015 | 3 |
| 2016 | 4 |
| 2017 | 3 |
| 2018 | 5 |
| 2019 | 7 |
| 2020 | 7 |
| 2021 | 7 |
| 2022 | 3 |
| 2023 | 5 |
| 2024 | 8 |
| 2025 | 2 |
| 2026 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 352 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| National Center for Research Resources (NCRR) | 133 |
| FDA Office of Orphan Products Development | 19 |
| National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | 10 |
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 9 |
| National Institute of Neurological Disorders and Stroke (NINDS) | 8 |
| Icahn School of Medicine at Mount Sinai | 6 |
| National Institute of Allergy and Infectious Diseases (NIAID) | 6 |
| Amgen | 5 |
| Ultragenyx Pharmaceutical Inc | 5 |
| Children's Hospital Medical Center, Cincinnati | 4 |
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 | 352 |
| Genetic Diseases, Inborn | 58 |
| Mental Disorders | 56 |
| Neurologic Manifestations | 56 |
| Respiratory Tract Diseases | 40 |
| Communicable Diseases | 39 |
| Immune System Diseases | 39 |
| Metabolism, Inborn Errors | 31 |
| Endocrine System Diseases | 28 |
| Hematologic Diseases | 27 |
Trials
Showing 76 to 100 of 352. Each trial links to its page on Pharma Observatory.
252 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.
Go deeper on Pharma Observatory
Pharma Observatory connects clinical trials, sponsors, sites and investigators in one searchable platform.