Clinical trials
Rare Diseases trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
590 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 29 |
| 2001 | 8 |
| 2003 | 2 |
| 2004 | 1 |
| 2006 | 4 |
| 2007 | 1 |
| 2009 | 3 |
| 2010 | 6 |
| 2011 | 2 |
| 2012 | 2 |
| 2013 | 7 |
| 2014 | 4 |
| 2015 | 9 |
| 2016 | 9 |
| 2017 | 7 |
| 2018 | 15 |
| 2019 | 17 |
| 2020 | 12 |
| 2021 | 28 |
| 2022 | 14 |
| 2023 | 24 |
| 2024 | 28 |
| 2025 | 32 |
| 2026 | 22 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 352 |
| Recruiting | 87 |
| Unknown status | 74 |
| Terminated | 26 |
| Not yet recruiting | 15 |
| Enrolling by invitation | 12 |
| Active, not recruiting | 10 |
| Withdrawn | 8 |
| Suspended | 2 |
| No longer available | 2 |
| Temporarily not available | 1 |
| Available | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| National Center for Research Resources (NCRR) | 164 |
| FDA Office of Orphan Products Development | 20 |
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 13 |
| National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | 13 |
| National Institute of Neurological Disorders and Stroke (NINDS) | 13 |
| University Hospital Tuebingen | 13 |
| Assistance Publique - Hôpitaux de Paris | 10 |
| IRCCS Policlinico S. Donato | 10 |
| Fairview University Medical Center | 7 |
| Institut National de la Santé Et de la Recherche Médicale, France | 7 |
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 | 590 |
| Genetic Diseases, Inborn | 94 |
| Neurologic Manifestations | 73 |
| Mental Disorders | 71 |
| Respiratory Tract Diseases | 50 |
| Communicable Diseases | 48 |
| Immune System Diseases | 48 |
| Metabolism, Inborn Errors | 42 |
| Endocrine System Diseases | 34 |
| Hematologic Diseases | 34 |
Trials
Showing 51 to 75 of 590. Each trial links to its page on Pharma Observatory.
490 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.