Clinical trials
Enrolling by invitation Rare Diseases trials in the United States
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
8 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2011 | 1 |
| 2018 | 1 |
| 2020 | 1 |
| 2021 | 3 |
| 2025 | 1 |
| 2026 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Enrolling by invitation | 8 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Mayo Clinic | 2 |
| TESS Research Foundation | 2 |
| Albert Einstein College of Medicine | 1 |
| Children's National Research Institute | 1 |
| Prof. Valérie Mc Lin | 1 |
| Scripps Translational Science Institute | 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 | 8 |
| Genetic Diseases, Inborn | 3 |
| Epilepsy | 2 |
| Movement Disorders | 2 |
| Undiagnosed Diseases | 2 |
| Anxiety Disorders | 1 |
| CHARGE Syndrome | 1 |
| Cockayne Syndrome | 1 |
| Congenital Portosystemic Shunt | 1 |
| Fabry Disease | 1 |
Trials
Showing 1 to 8 of 8. 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.