Clinical trials
Enrolling by invitation Observational Genetic Diseases, Inborn trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
9 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2019 | 2 |
| 2021 | 2 |
| 2023 | 1 |
| 2024 | 1 |
| 2025 | 1 |
| 2026 | 2 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Enrolling by invitation | 9 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| TESS Research Foundation | 2 |
| BioMarin Pharmaceutical | 1 |
| Editas Medicine, Inc. | 1 |
| Karolinska Institutet | 1 |
| Mayo Clinic | 1 |
| The Wellcome Sanger Institute | 1 |
| University of California, San Francisco | 1 |
| University of Washington | 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 | 9 |
| Rare Diseases | 4 |
| Epilepsy | 2 |
| Hematologic Diseases | 2 |
| Movement Disorders | 2 |
| Undiagnosed Diseases | 2 |
| Anemia | 1 |
| Anemia, Hemolytic | 1 |
| Anemia, Hemolytic, Congenital | 1 |
| Anemia, Sickle Cell | 1 |
Trials
Showing 1 to 9 of 9. 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.