Clinical trials
Not yet recruiting Observational Rare Diseases trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
10 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2025 | 2 |
| 2026 | 8 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Not yet recruiting | 10 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Akcea Therapeutics | 1 |
| Assistance Publique - Hôpitaux de Paris | 1 |
| Istituto Ortopedico Rizzoli | 1 |
| Markus A. Landolt | 1 |
| Novartis Pharmaceuticals | 1 |
| Research in Real-Life Ltd | 1 |
| The Hospital for Sick Children | 1 |
| University Hospital, Bordeaux | 1 |
| University of Exeter | 1 |
| University of Manchester | 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 | 10 |
| Genetic Diseases, Inborn | 2 |
| Albinism | 1 |
| Charcot-Marie-Tooth Disease | 1 |
| Congenital Abnormalities | 1 |
| Friedreich Ataxia | 1 |
| Intellectual Disability | 1 |
| Muscular Atrophy, Spinal | 1 |
| Neuritis | 1 |
| Neuromuscular Diseases | 1 |
Trials
Showing 1 to 10 of 10. 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.
Go deeper on Pharma Observatory
Pharma Observatory connects clinical trials, sponsors, sites and investigators in one searchable platform.