Clinical trials
Phase 1/2 Congenital, Hereditary, and Neonatal Diseases and Abnormalities trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
2 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
No trials to chart.
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Not yet recruiting | 2 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Agnieszka Czechowicz | 1 |
| NeuroGT | 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 | 2 |
| Genetic Diseases, Inborn | 2 |
| Anemia, Hypoplastic, Congenital | 1 |
| Bone Marrow Failure Disorders | 1 |
| Carbohydrate Metabolism, Inborn Errors | 1 |
| Congenital Bone Marrow Failure Syndromes | 1 |
| Connective Tissue Diseases | 1 |
| DNA Repair-Deficiency Disorders | 1 |
| Fanconi Anemia | 1 |
| Lysosomal Storage Diseases | 1 |
Trials
Showing 1 to 2 of 2. 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
Pharma Observatory connects clinical trials, sponsors, sites and investigators in one searchable platform.