Clinical trials
Phase 2 Rare Diseases trials in the United States
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
44 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 3 |
| 2001 | 2 |
| 2006 | 1 |
| 2013 | 1 |
| 2015 | 1 |
| 2016 | 2 |
| 2017 | 1 |
| 2022 | 2 |
| 2025 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 31 |
| Unknown status | 6 |
| Terminated | 3 |
| Recruiting | 2 |
| Active, not recruiting | 1 |
| Suspended | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| National Center for Research Resources (NCRR) | 11 |
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 2 |
| Johns Hopkins University | 2 |
| National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | 2 |
| University of Utah | 2 |
| Boston University | 1 |
| Charles Drew University of Medicine and Science | 1 |
| Children's Hospital Medical Center, Cincinnati | 1 |
| Fred Hutchinson Cancer Center | 1 |
| Genervon Biopharmaceuticals, LLC | 1 |
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 | 44 |
| Connective Tissue Diseases | 7 |
| Genetic Diseases, Inborn | 7 |
| Hematologic Diseases | 7 |
| Arthritis | 6 |
| Metabolism, Inborn Errors | 6 |
| Respiratory Tract Diseases | 5 |
| Mental Disorders | 4 |
| Neurologic Manifestations | 4 |
| Communicable Diseases | 3 |
Trials
Showing 1 to 25 of 44. Each trial links to its page 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.