Clinical trials
Recruiting Congenital, Hereditary, and Neonatal Diseases and Abnormalities trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
14 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2010 | 1 |
| 2020 | 1 |
| 2022 | 2 |
| 2023 | 2 |
| 2025 | 4 |
| 2026 | 4 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Recruiting | 14 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Arrowhead Pharmaceuticals | 1 |
| Biossil Inc. | 1 |
| Children's Hospital of Philadelphia | 1 |
| Dyne Therapeutics | 1 |
| Hadassah Medical Organization | 1 |
| Indiana University | 1 |
| JCR Pharmaceuticals Co., Ltd. | 1 |
| Johns Hopkins University | 1 |
| Nantes University Hospital | 1 |
| National Human Genome Research Institute (NHGRI) | 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 |
|---|---|
| Congenital, Hereditary, and Neonatal Diseases and Abnormalities | 14 |
| Genetic Diseases, Inborn | 5 |
| Congenital Abnormalities | 2 |
| Intellectual Disability | 2 |
| Metabolism, Inborn Errors | 2 |
| Acquired Immunodeficiency Syndrome | 1 |
| Anemia | 1 |
| Anemia, Hemolytic | 1 |
| Anemia, Hemolytic, Congenital | 1 |
| Anemia, Sickle Cell | 1 |
Trials
Showing 26 to 14 of 14. Each trial links to its page on Pharma Observatory.
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Recently changed trials
Changes observed in the last 7 days, newest first, site-count changes left out. Source: ClinicalTrials.gov.
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