Clinical trials
Active, not recruiting Genetic Diseases, Inborn trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
40 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2014 | 1 |
| 2017 | 1 |
| 2018 | 1 |
| 2019 | 1 |
| 2020 | 5 |
| 2021 | 4 |
| 2022 | 10 |
| 2023 | 7 |
| 2024 | 6 |
| 2025 | 4 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Active, not recruiting | 40 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| BioMarin Pharmaceutical | 3 |
| Grand Valley State University | 2 |
| University of California, San Diego | 2 |
| Vanderbilt University Medical Center | 2 |
| Vertex Pharmaceuticals Incorporated | 2 |
| 4D Molecular Therapeutics | 1 |
| AO GENERIUM | 1 |
| Abramson Cancer Center at Penn Medicine | 1 |
| AskBio France, SAS, a subsidiary of AskBio Inc. | 1 |
| Borstkanker Onderzoek Groep | 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 | 40 |
| Nervous System Diseases | 8 |
| Hematologic Diseases | 5 |
| Metabolic Diseases | 5 |
| Central Nervous System Diseases | 4 |
| Genetic Diseases, X-Linked | 4 |
| Anemia | 3 |
| Brain Diseases | 3 |
| Brain Diseases, Metabolic | 3 |
| Cardiovascular Diseases | 3 |
Trials
Showing 26 to 40 of 40. 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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