Clinical trials
Recruiting Rare Diseases trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
87 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 1 |
| 2001 | 1 |
| 2010 | 2 |
| 2015 | 2 |
| 2016 | 1 |
| 2017 | 2 |
| 2018 | 4 |
| 2019 | 5 |
| 2020 | 1 |
| 2021 | 6 |
| 2022 | 6 |
| 2023 | 11 |
| 2024 | 17 |
| 2025 | 19 |
| 2026 | 8 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Recruiting | 87 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| National Human Genome Research Institute (NHGRI) | 5 |
| IRCCS Policlinico S. Donato | 4 |
| Institut National de la Santé Et de la Recherche Médicale, France | 4 |
| University Hospital Tuebingen | 4 |
| Assistance Publique - Hôpitaux de Paris | 3 |
| Imagine Institute | 3 |
| Boston Children's Hospital | 2 |
| Ipsen | 2 |
| University Hospital, Lille | 2 |
| Ann & Robert H Lurie Children's Hospital of Chicago | 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 | 87 |
| Genetic Diseases, Inborn | 11 |
| Undiagnosed Diseases | 6 |
| Neoplasms | 5 |
| Intellectual Disability | 4 |
| Nervous System Diseases | 4 |
| Ataxia | 3 |
| Cardiovascular Diseases | 3 |
| Friedreich Ataxia | 3 |
| Genetic Predisposition to Disease | 3 |
Trials
Showing 76 to 87 of 87. 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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