Clinical trials
Rare Diseases trials in the United Kingdom
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
25 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2003 | 1 |
| 2006 | 1 |
| 2007 | 1 |
| 2013 | 3 |
| 2015 | 2 |
| 2016 | 1 |
| 2017 | 1 |
| 2018 | 1 |
| 2020 | 1 |
| 2021 | 1 |
| 2022 | 2 |
| 2023 | 3 |
| 2025 | 1 |
| 2026 | 3 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 12 |
| Not yet recruiting | 3 |
| Recruiting | 3 |
| Active, not recruiting | 2 |
| Enrolling by invitation | 1 |
| Suspended | 1 |
| Terminated | 1 |
| Unknown status | 1 |
| Withdrawn | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Associazione Angela Serra per la ricerca sul cancro | 2 |
| Gemma Biotherapeutics | 2 |
| Laboratoire français de Fractionnement et de Biotechnologies | 2 |
| Novartis Pharmaceuticals | 2 |
| Ultragenyx Pharmaceutical Inc | 2 |
| Amgen | 1 |
| Boston University | 1 |
| Friedreich's Ataxia Research Alliance | 1 |
| National Center for Research Resources (NCRR) | 1 |
| NightstaRx Ltd, a Biogen Company | 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 | 25 |
| Friedreich Ataxia | 3 |
| Genetic Diseases, Inborn | 3 |
| Lysosomal Storage Diseases | 3 |
| Neurodegenerative Diseases | 3 |
| Ataxia | 2 |
| Hematologic Diseases | 2 |
| Huntington Disease | 2 |
| Metabolic Diseases | 2 |
| Peripheral Nervous System Diseases | 2 |
Trials
Showing 1 to 25 of 25. 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.
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