Clinical trials
Congenital, Hereditary, and Neonatal Diseases and Abnormalities trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
107 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 2 |
| 2001 | 5 |
| 2002 | 8 |
| 2003 | 1 |
| 2004 | 7 |
| 2005 | 8 |
| 2006 | 5 |
| 2007 | 3 |
| 2008 | 3 |
| 2010 | 1 |
| 2011 | 1 |
| 2012 | 1 |
| 2016 | 1 |
| 2017 | 4 |
| 2018 | 3 |
| 2019 | 2 |
| 2020 | 2 |
| 2021 | 3 |
| 2022 | 5 |
| 2023 | 5 |
| 2024 | 2 |
| 2025 | 6 |
| 2026 | 5 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 40 |
| Terminated | 39 |
| Recruiting | 14 |
| Unknown status | 5 |
| Active, not recruiting | 4 |
| Withdrawn | 3 |
| Not yet recruiting | 2 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Children's Healthcare of Atlanta | 49 |
| Emory University | 12 |
| Ain Shams University | 2 |
| G. d'Annunzio University | 2 |
| Taysha Gene Therapies, Inc. | 2 |
| Agnieszka Czechowicz | 1 |
| Ahmed Hamdy Rateb | 1 |
| Arrowhead Pharmaceuticals | 1 |
| Assiut University | 1 |
| Biossil Inc. | 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 | 107 |
| Genetic Diseases, Inborn | 10 |
| Heart Defects, Congenital | 9 |
| Congenital Abnormalities | 4 |
| Intellectual Disability | 4 |
| Metabolic Diseases | 4 |
| Metabolism, Inborn Errors | 4 |
| Genetic Diseases, X-Linked | 3 |
| Nervous System Diseases | 3 |
| Anemia, Hypoplastic, Congenital | 2 |
Trials
Showing 1 to 25 of 107. Each trial links to its page on Pharma Observatory.
7 more trials match. See them 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
Pharma Observatory connects clinical trials, sponsors, sites and investigators in one searchable platform.