Clinical trials
Rare Diseases trials in the United States
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
244 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 22 |
| 2001 | 7 |
| 2003 | 1 |
| 2004 | 1 |
| 2006 | 2 |
| 2009 | 2 |
| 2010 | 5 |
| 2011 | 1 |
| 2012 | 1 |
| 2013 | 4 |
| 2014 | 2 |
| 2015 | 4 |
| 2016 | 6 |
| 2017 | 4 |
| 2018 | 7 |
| 2019 | 5 |
| 2020 | 4 |
| 2021 | 9 |
| 2022 | 6 |
| 2023 | 3 |
| 2024 | 5 |
| 2025 | 12 |
| 2026 | 3 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 141 |
| Unknown status | 35 |
| Recruiting | 34 |
| Terminated | 15 |
| Enrolling by invitation | 8 |
| Active, not recruiting | 3 |
| Suspended | 2 |
| No longer available | 2 |
| Withdrawn | 2 |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) | 67 |
| National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | 9 |
| Fairview University Medical Center | 7 |
| National Human Genome Research Institute (NHGRI) | 6 |
| Amgen | 5 |
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 5 |
| Icahn School of Medicine at Mount Sinai | 5 |
| Children's National Research Institute | 4 |
| Fred Hutchinson Cancer Center | 4 |
| Ultragenyx Pharmaceutical Inc | 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 | 244 |
| Genetic Diseases, Inborn | 44 |
| Neurologic Manifestations | 27 |
| Mental Disorders | 26 |
| Respiratory Tract Diseases | 23 |
| Hematologic Diseases | 22 |
| Communicable Diseases | 20 |
| Immune System Diseases | 20 |
| Metabolism, Inborn Errors | 19 |
| Connective Tissue Diseases | 17 |
Trials
Showing 51 to 75 of 244. Each trial links to its page on Pharma Observatory.
144 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.