Clinical trials
Rare Diseases trials in the United States with no applicable phase
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
54 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 12 |
| 2001 | 1 |
| 2017 | 2 |
| 2018 | 1 |
| 2021 | 1 |
| 2024 | 1 |
| 2025 | 2 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 45 |
| Unknown status | 5 |
| Recruiting | 2 |
| Enrolling by invitation | 1 |
| Terminated | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| National Center for Research Resources (NCRR) | 12 |
| Fairview University Medical Center | 6 |
| Icahn School of Medicine at Mount Sinai | 4 |
| Children's National Research Institute | 3 |
| Case Western Reserve University | 2 |
| Children's Hospital Medical Center, Cincinnati | 2 |
| National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | 2 |
| National Institute of Mental Health (NIMH) | 2 |
| University of Chicago | 2 |
| Baylor College of Medicine | 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 | 54 |
| Genetic Diseases, Inborn | 10 |
| Hematologic Diseases | 10 |
| Mental Disorders | 9 |
| Neurologic Manifestations | 9 |
| Communicable Diseases | 8 |
| Immune System Diseases | 8 |
| Endocrine System Diseases | 7 |
| Respiratory Tract Diseases | 6 |
| Graft vs Host Disease | 5 |
Trials
Showing 1 to 25 of 54. 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.