Clinical trials
Expanded access Rare Diseases trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
4 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
No trials to chart.
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| No longer available | 2 |
| Available | 1 |
| Temporarily not available | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Joyce Fox | 1 |
| Rapa Therapeutics LLC | 1 |
| RiboNova Inc. | 1 |
| University of Florida | 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 | 4 |
| Acidosis | 1 |
| Amyotrophic Lateral Sclerosis | 1 |
| Genetic Diseases, Inborn | 1 |
| Lysosomal Storage Diseases | 1 |
| Metabolic Diseases | 1 |
| Mitochondrial Diseases | 1 |
| Mucopolysaccharidosis VII | 1 |
| Muscular Diseases | 1 |
| Renal Insufficiency, Chronic | 1 |
Trials
Showing 1 to 4 of 4. 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.