Clinical trials
Observational Genetic Diseases, Inborn trials in the United Kingdom
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
20 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2006 | 1 |
| 2013 | 1 |
| 2016 | 1 |
| 2018 | 2 |
| 2019 | 3 |
| 2021 | 1 |
| 2022 | 3 |
| 2023 | 2 |
| 2024 | 2 |
| 2025 | 2 |
| 2026 | 2 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 9 |
| Recruiting | 7 |
| Enrolling by invitation | 2 |
| Not yet recruiting | 1 |
| Terminated | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| QED Therapeutics, a BridgeBio company | 2 |
| Swedish Orphan Biovitrum | 2 |
| Alexion Pharmaceuticals, Inc. | 1 |
| Azafaros B.V. | 1 |
| BioMarin Pharmaceutical | 1 |
| CSL Behring | 1 |
| Friedreich's Ataxia Research Alliance | 1 |
| GenSight Biologics | 1 |
| HemoShear Therapeutics | 1 |
| Imagine Institute | 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 | 20 |
| Metabolism, Inborn Errors | 4 |
| Nervous System Diseases | 4 |
| Blood Coagulation Disorders | 3 |
| Coagulation Protein Disorders | 3 |
| Hematologic Diseases | 3 |
| Hemorrhagic Disorders | 3 |
| Metabolic Diseases | 3 |
| Bone Diseases | 2 |
| Brain Diseases, Metabolic, Inborn | 2 |
Trials
Showing 1 to 20 of 20. 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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