Clinical trials
Phase 2 Genetic Diseases, Inborn trials in the United Kingdom
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 |
|---|---|
| 2010 | 1 |
| 2011 | 2 |
| 2012 | 1 |
| 2014 | 1 |
| 2018 | 2 |
| 2019 | 1 |
| 2020 | 1 |
| 2021 | 1 |
| 2022 | 1 |
| 2025 | 1 |
| 2026 | 2 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 9 |
| Enrolling by invitation | 3 |
| Recruiting | 1 |
| Terminated | 1 |
| Unknown status | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Novartis Pharmaceuticals | 3 |
| QED Therapeutics, a BridgeBio company | 3 |
| Alexion Pharmaceuticals, Inc. | 1 |
| Alnylam Pharmaceuticals | 1 |
| BioMarin Pharmaceutical | 1 |
| Boston University | 1 |
| CSL Behring | 1 |
| Forma Therapeutics, Inc. | 1 |
| Imperial College London | 1 |
| Novo Nordisk A/S | 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 |
|---|---|
| Genetic Diseases, Inborn | 15 |
| Musculoskeletal Diseases | 5 |
| Bone Diseases | 4 |
| Osteochondrodysplasias | 4 |
| Achondroplasia | 3 |
| Dwarfism | 3 |
| Fragile X Syndrome | 3 |
| Mucopolysaccharidosis IV | 3 |
| Anemia | 2 |
| Anemia, Sickle Cell | 2 |
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.
No changes to these trials in the last 7 days.
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