Clinical trials
Phase 1 Rare Diseases trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
35 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 4 |
| 2001 | 2 |
| 2006 | 1 |
| 2016 | 1 |
| 2018 | 1 |
| 2021 | 1 |
| 2023 | 1 |
| 2025 | 2 |
| 2026 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 24 |
| Unknown status | 5 |
| Terminated | 3 |
| Recruiting | 2 |
| Not yet recruiting | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| National Center for Research Resources (NCRR) | 10 |
| FDA Office of Orphan Products Development | 2 |
| Fred Hutchinson Cancer Center | 2 |
| National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | 2 |
| Northwestern Memorial Hospital | 2 |
| University of Alabama at Birmingham | 2 |
| Connecta Therapeutics, S.L. | 1 |
| Dana-Farber Cancer Institute | 1 |
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 1 |
| Genethon | 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 | 35 |
| Genetic Diseases, Inborn | 8 |
| Respiratory Tract Diseases | 8 |
| Metabolism, Inborn Errors | 7 |
| Cystic Fibrosis | 6 |
| Communicable Diseases | 3 |
| Hematologic Diseases | 3 |
| Immune System Diseases | 3 |
| Mental Disorders | 3 |
| Multiple Sclerosis | 3 |
Trials
Showing 26 to 35 of 35. 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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