Clinical trials
Recruiting Genetic Diseases, Inborn trials in the United States
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
44 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2005 | 1 |
| 2007 | 1 |
| 2010 | 1 |
| 2011 | 1 |
| 2012 | 1 |
| 2015 | 2 |
| 2016 | 1 |
| 2018 | 3 |
| 2020 | 3 |
| 2021 | 4 |
| 2022 | 5 |
| 2023 | 4 |
| 2024 | 5 |
| 2025 | 2 |
| 2026 | 8 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Recruiting | 44 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Children's Hospital of Philadelphia | 3 |
| ModernaTX, Inc. | 3 |
| National Human Genome Research Institute (NHGRI) | 3 |
| National Institute of Allergy and Infectious Diseases (NIAID) | 3 |
| Boston Children's Hospital | 2 |
| CSL Behring | 2 |
| Genomic Prediction Inc. | 2 |
| Rady Pediatric Genomics & Systems Medicine Institute | 2 |
| Arrowhead Pharmaceuticals | 1 |
| Baylor College of Medicine | 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 | 44 |
| Metabolism, Inborn Errors | 6 |
| Metabolic Diseases | 5 |
| Congenital, Hereditary, and Neonatal Diseases and Abnormalities | 4 |
| Genetic Diseases, X-Linked | 4 |
| Rare Diseases | 4 |
| Amino Acid Metabolism, Inborn Errors | 3 |
| Genetic Predisposition to Disease | 3 |
| Infertility | 3 |
| Musculoskeletal Diseases | 3 |
Trials
Showing 1 to 25 of 44. 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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