Clinical trials
Active, not recruiting Observational Genetic Diseases, Inborn trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
11 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2014 | 1 |
| 2018 | 1 |
| 2020 | 2 |
| 2022 | 2 |
| 2023 | 3 |
| 2024 | 1 |
| 2025 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Active, not recruiting | 11 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Boston Children's Hospital | 1 |
| Columbia University | 1 |
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 1 |
| Karolinska Institutet | 1 |
| M.D. Anderson Cancer Center | 1 |
| Nicklaus Children's Hospital f/k/a Miami Children's Hospital | 1 |
| Northwestern University | 1 |
| Prime Medicine, Inc. | 1 |
| Rocket Pharmaceuticals Inc. | 1 |
| Sanofi | 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 |
|---|---|
| Genetic Diseases, Inborn | 11 |
| Congenital Abnormalities | 2 |
| Anemia | 1 |
| Anxiety Disorders | 1 |
| Atherosclerosis | 1 |
| Blood Coagulation Disorders, Inherited | 1 |
| Bone Marrow Failure Disorders | 1 |
| Carcinoma, Squamous Cell | 1 |
| Cardiovascular Diseases | 1 |
| Coagulation Protein Disorders | 1 |
Trials
Showing 1 to 11 of 11. 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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