Clinical trials
Completed Phase 1 Rare Diseases trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
24 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 2 |
| 2001 | 1 |
| 2006 | 1 |
| 2016 | 1 |
| 2021 | 1 |
| 2023 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 24 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| National Center for Research Resources (NCRR) | 8 |
| FDA Office of Orphan Products Development | 2 |
| Fred Hutchinson Cancer Center | 2 |
| National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | 2 |
| University of Alabama at Birmingham | 2 |
| Connecta Therapeutics, S.L. | 1 |
| Genethon | 1 |
| Gradalis, Inc. | 1 |
| Northwestern Memorial Hospital | 1 |
| Stony Brook University | 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 | 24 |
| Respiratory Tract Diseases | 8 |
| Genetic Diseases, Inborn | 7 |
| Cystic Fibrosis | 6 |
| Metabolism, Inborn Errors | 4 |
| Mental Disorders | 3 |
| Multiple Sclerosis | 3 |
| Neurologic Manifestations | 3 |
| Communicable Diseases | 2 |
| Immune System Diseases | 2 |
Trials
Showing 1 to 24 of 24. 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
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