Clinical trials
Genetic Diseases, Inborn trials in the United Kingdom
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
84 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2006 | 1 |
| 2007 | 1 |
| 2008 | 1 |
| 2009 | 4 |
| 2010 | 1 |
| 2011 | 3 |
| 2012 | 1 |
| 2013 | 2 |
| 2014 | 3 |
| 2015 | 2 |
| 2016 | 4 |
| 2017 | 3 |
| 2018 | 11 |
| 2019 | 6 |
| 2020 | 2 |
| 2021 | 8 |
| 2022 | 7 |
| 2023 | 5 |
| 2024 | 5 |
| 2025 | 4 |
| 2026 | 8 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 42 |
| Recruiting | 14 |
| Active, not recruiting | 8 |
| Terminated | 8 |
| Enrolling by invitation | 7 |
| Unknown status | 2 |
| Not yet recruiting | 2 |
| Withdrawn | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| BioMarin Pharmaceutical | 10 |
| QED Therapeutics, a BridgeBio company | 7 |
| Vertex Pharmaceuticals Incorporated | 7 |
| GenSight Biologics | 4 |
| ModernaTX, Inc. | 4 |
| Novartis Pharmaceuticals | 4 |
| Alexion Pharmaceuticals, Inc. | 3 |
| Novo Nordisk A/S | 3 |
| CSL Behring | 2 |
| Forma Therapeutics, Inc. | 2 |
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 | 84 |
| Musculoskeletal Diseases | 19 |
| Nervous System Diseases | 19 |
| Hematologic Diseases | 16 |
| Metabolism, Inborn Errors | 15 |
| Metabolic Diseases | 13 |
| Bone Diseases | 12 |
| Osteochondrodysplasias | 12 |
| Blood Coagulation Disorders | 10 |
| Coagulation Protein Disorders | 10 |
Trials
Showing 1 to 25 of 84. 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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