Clinical trials
Phase 2/3 Genetic Diseases, Inborn trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
10 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2004 | 1 |
| 2009 | 1 |
| 2011 | 1 |
| 2015 | 1 |
| 2018 | 1 |
| 2021 | 2 |
| 2023 | 1 |
| 2025 | 1 |
| 2026 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 4 |
| Withdrawn | 2 |
| Active, not recruiting | 1 |
| Enrolling by invitation | 1 |
| Recruiting | 1 |
| Terminated | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| AO GENERIUM | 2 |
| BioMarin Pharmaceutical | 1 |
| Children's Hospital of Fudan University | 1 |
| Novartis Pharmaceuticals | 1 |
| Pharming Technologies B.V. | 1 |
| QED Therapeutics, a BridgeBio company | 1 |
| Radboud University Medical Center | 1 |
| University of Washington, the Collaborative Health Studies Coordinating Center | 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 | 10 |
| Bone Diseases | 2 |
| Digestive System Diseases | 2 |
| Musculoskeletal Diseases | 2 |
| Osteochondrodysplasias | 2 |
| Achondroplasia | 1 |
| Adenoma | 1 |
| Adenomatous Polyposis Coli | 1 |
| Adenomatous Polyps | 1 |
| Angioedema | 1 |
Trials
Showing 1 to 10 of 10. 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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