Clinical trials
Active, not recruiting Phase 3 Genetic Diseases, Inborn trials
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 |
|---|---|
| 2017 | 1 |
| 2019 | 1 |
| 2021 | 1 |
| 2022 | 2 |
| 2023 | 2 |
| 2024 | 2 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Active, not recruiting | 9 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| BioMarin Pharmaceutical | 3 |
| Capricor Inc. | 1 |
| Dicerna Pharmaceuticals, Inc., a Novo Nordisk company | 1 |
| Forma Therapeutics, Inc. | 1 |
| Neurogene Inc. | 1 |
| Vertex Pharmaceuticals Incorporated | 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 | 9 |
| Genetic Diseases, X-Linked | 3 |
| Hematologic Diseases | 3 |
| Nervous System Diseases | 3 |
| Bone Diseases | 2 |
| Bone Diseases, Developmental | 2 |
| Dwarfism | 2 |
| Hemoglobinopathies | 2 |
| Musculoskeletal Diseases | 2 |
| Osteochondrodysplasias | 2 |
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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