Clinical trials
Not yet recruiting Rare Diseases trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
15 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2023 | 1 |
| 2025 | 2 |
| 2026 | 11 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Not yet recruiting | 15 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Shionogi | 2 |
| Akcea Therapeutics | 1 |
| Assistance Publique - Hôpitaux de Paris | 1 |
| Assistance Publique Hopitaux De Marseille | 1 |
| Istituto Ortopedico Rizzoli | 1 |
| Markus A. Landolt | 1 |
| Novartis Pharmaceuticals | 1 |
| Research in Real-Life Ltd | 1 |
| Sheffield Hallam University | 1 |
| The Hospital for Sick Children | 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 | 15 |
| Genetic Diseases, Inborn | 2 |
| Glycogen Storage Disease | 2 |
| Glycogen Storage Disease Type II | 2 |
| Albinism | 1 |
| Charcot-Marie-Tooth Disease | 1 |
| Congenital Abnormalities | 1 |
| Connective Tissue Diseases | 1 |
| Ehlers-Danlos Syndrome, Type IV | 1 |
| Friedreich Ataxia | 1 |
Trials
Showing 1 to 15 of 15. 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.