Clinical trials
Active, not 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.
4 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2023 | 1 |
| 2025 | 2 |
| 2026 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Active, not recruiting | 4 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Taysha Gene Therapies, Inc. | 2 |
| Napo Pharmaceuticals, Inc. | 1 |
| Prime Medicine, 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 | 4 |
| Genetic Diseases, X-Linked | 2 |
| Intellectual Disability | 2 |
| Nervous System Diseases | 2 |
| Neurodevelopmental Disorders | 2 |
| Neurologic Manifestations | 2 |
| Pathologic Processes | 2 |
| Rett Syndrome | 2 |
| X-Linked Intellectual Disability | 2 |
| Genetic Diseases, Inborn | 1 |
Trials
Showing 51 to 4 of 4. 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.
No changes to these trials in the last 7 days.
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