Clinical trials
Genetic Diseases, Inborn trials in the United States with no applicable phase
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
45 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 3 |
| 2012 | 2 |
| 2014 | 2 |
| 2015 | 2 |
| 2016 | 2 |
| 2017 | 3 |
| 2018 | 4 |
| 2019 | 1 |
| 2020 | 4 |
| 2021 | 2 |
| 2022 | 3 |
| 2023 | 4 |
| 2024 | 3 |
| 2025 | 4 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 26 |
| Active, not recruiting | 9 |
| Recruiting | 4 |
| Unknown status | 3 |
| Enrolling by invitation | 2 |
| Terminated | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Rady Pediatric Genomics & Systems Medicine Institute | 5 |
| Fairview University Medical Center | 3 |
| Abramson Cancer Center at Penn Medicine | 2 |
| Boston Children's Hospital | 2 |
| Brigham and Women's Hospital | 2 |
| Grand Valley State University | 2 |
| Massachusetts General Hospital | 2 |
| University of North Carolina, Chapel Hill | 2 |
| Vanderbilt University Medical Center | 2 |
| Children's Hospital Medical Center, Cincinnati | 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 | 45 |
| Rare Diseases | 10 |
| Hematologic Diseases | 7 |
| Genetic Predisposition to Disease | 6 |
| Anemia, Sickle Cell | 5 |
| Cardiovascular Diseases | 3 |
| Cystic Fibrosis | 3 |
| Disease | 3 |
| Immune System Diseases | 3 |
| Metabolism, Inborn Errors | 3 |
Trials
Showing 1 to 25 of 45. 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.