Clinical trials
Active, not recruiting Genetic Diseases, Inborn trials in the United Kingdom
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
8 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2017 | 1 |
| 2019 | 1 |
| 2021 | 1 |
| 2022 | 1 |
| 2023 | 1 |
| 2024 | 3 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Active, not recruiting | 8 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| BioMarin Pharmaceutical | 2 |
| Vertex Pharmaceuticals Incorporated | 2 |
| Dicerna Pharmaceuticals, Inc., a Novo Nordisk company | 1 |
| Forma Therapeutics, Inc. | 1 |
| Neurogene Inc. | 1 |
| iECURE, Inc. | 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 | 8 |
| Musculoskeletal Diseases | 3 |
| Nervous System Diseases | 3 |
| Bone Diseases | 2 |
| Bone Diseases, Developmental | 2 |
| Dwarfism | 2 |
| Genetic Diseases, X-Linked | 2 |
| Hematologic Diseases | 2 |
| Hemoglobinopathies | 2 |
| Osteochondrodysplasias | 2 |
Trials
Showing 1 to 8 of 8. 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
Pharma Observatory connects clinical trials, sponsors, sites and investigators in one searchable platform.