Clinical trials
Recruiting Rare Diseases trials in the United States
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
34 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 1 |
| 2010 | 2 |
| 2015 | 2 |
| 2018 | 2 |
| 2019 | 2 |
| 2021 | 2 |
| 2022 | 4 |
| 2023 | 3 |
| 2024 | 4 |
| 2025 | 9 |
| 2026 | 2 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Recruiting | 34 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| National Human Genome Research Institute (NHGRI) | 4 |
| Boston Children's Hospital | 2 |
| Ipsen | 2 |
| Ann & Robert H Lurie Children's Hospital of Chicago | 1 |
| Aspa Therapeutics | 1 |
| Associazione Angela Serra per la ricerca sul cancro | 1 |
| Baylor College of Medicine | 1 |
| Case Western Reserve University | 1 |
| Children's National Research Institute | 1 |
| Epilepsy Foundation of America | 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 | 34 |
| Genetic Diseases, Inborn | 4 |
| Undiagnosed Diseases | 4 |
| Ataxia | 3 |
| Friedreich Ataxia | 3 |
| Nervous System Diseases | 3 |
| Amyotrophic Lateral Sclerosis | 2 |
| Glycogen Storage Disease | 2 |
| Glycogen Storage Disease Type II | 2 |
| Hematologic Neoplasms | 2 |
Trials
Showing 1 to 25 of 34. 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.