Clinical trials
Phase 1/2 Genetic Diseases, Inborn trials in the United Kingdom
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
9 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2015 | 2 |
| 2018 | 1 |
| 2020 | 1 |
| 2021 | 3 |
| 2022 | 1 |
| 2024 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 3 |
| Recruiting | 3 |
| Terminated | 2 |
| Active, not recruiting | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| ModernaTX, Inc. | 4 |
| BioMarin Pharmaceutical | 3 |
| Alexion Pharmaceuticals, Inc. | 1 |
| Vertex Pharmaceuticals Incorporated | 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 | 9 |
| Metabolism, Inborn Errors | 5 |
| Blood Coagulation Disorders | 3 |
| Blood Coagulation Disorders, Inherited | 3 |
| Coagulation Protein Disorders | 3 |
| Hematologic Diseases | 3 |
| Hemophilia A | 3 |
| Hemorrhagic Disorders | 3 |
| Hemostatic Disorders | 3 |
| Metabolic Diseases | 3 |
Trials
Showing 1 to 9 of 9. 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.
Go deeper on Pharma Observatory
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