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 1 to 25 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.