Clinical trials
Phase 3 Genetic Diseases, Inborn trials in the United States
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
43 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2005 | 1 |
| 2007 | 1 |
| 2008 | 1 |
| 2009 | 5 |
| 2013 | 1 |
| 2014 | 2 |
| 2016 | 3 |
| 2017 | 5 |
| 2018 | 7 |
| 2019 | 1 |
| 2020 | 1 |
| 2021 | 3 |
| 2022 | 5 |
| 2023 | 2 |
| 2024 | 2 |
| 2026 | 3 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 26 |
| Active, not recruiting | 8 |
| Recruiting | 4 |
| Terminated | 3 |
| Enrolling by invitation | 1 |
| Withdrawn | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| BioMarin Pharmaceutical | 6 |
| Regeneron Pharmaceuticals | 5 |
| Vertex Pharmaceuticals Incorporated | 5 |
| GenSight Biologics | 3 |
| PTC Therapeutics | 3 |
| Genzyme, a Sanofi Company | 2 |
| Kastle Therapeutics, LLC | 2 |
| Pfizer | 2 |
| Quince Therapeutics S.p.A. | 2 |
| Arrowhead Pharmaceuticals | 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 | 43 |
| Genetic Diseases, X-Linked | 11 |
| Musculoskeletal Diseases | 11 |
| Nervous System Diseases | 11 |
| Hematologic Diseases | 10 |
| Metabolic Diseases | 8 |
| Metabolism, Inborn Errors | 7 |
| Blood Coagulation Disorders | 6 |
| Blood Coagulation Disorders, Inherited | 6 |
| Coagulation Protein Disorders | 6 |
Trials
Showing 1 to 25 of 43. 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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