Clinical trials
Congenital, Hereditary, and Neonatal Diseases and Abnormalities trials in the United States
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
74 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 2 |
| 2001 | 5 |
| 2002 | 7 |
| 2003 | 1 |
| 2004 | 7 |
| 2005 | 6 |
| 2006 | 5 |
| 2007 | 3 |
| 2008 | 2 |
| 2010 | 1 |
| 2011 | 1 |
| 2016 | 1 |
| 2019 | 1 |
| 2022 | 1 |
| 2023 | 1 |
| 2025 | 3 |
| 2026 | 4 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Terminated | 38 |
| Completed | 22 |
| Recruiting | 6 |
| Active, not recruiting | 4 |
| Not yet recruiting | 2 |
| Withdrawn | 2 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Children's Healthcare of Atlanta | 47 |
| Emory University | 11 |
| Taysha Gene Therapies, Inc. | 2 |
| Agnieszka Czechowicz | 1 |
| Arrowhead Pharmaceuticals | 1 |
| Children's Hospital Medical Center, Cincinnati | 1 |
| Children's Hospital of Philadelphia | 1 |
| Dyne Therapeutics | 1 |
| JCR Pharmaceuticals Co., Ltd. | 1 |
| Johns Hopkins University | 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 | 74 |
| Heart Defects, Congenital | 9 |
| Genetic Diseases, Inborn | 8 |
| Metabolism, Inborn Errors | 4 |
| Congenital Abnormalities | 3 |
| Genetic Diseases, X-Linked | 3 |
| Intellectual Disability | 3 |
| Metabolic Diseases | 3 |
| Anemia, Hypoplastic, Congenital | 2 |
| Carbohydrate Metabolism, Inborn Errors | 2 |
Trials
Showing 26 to 50 of 74. 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.
Go deeper on Pharma Observatory
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