Clinical trials
Active, not recruiting Genetic Diseases, Inborn trials in the United States
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
32 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2014 | 1 |
| 2017 | 1 |
| 2018 | 1 |
| 2019 | 1 |
| 2020 | 4 |
| 2021 | 3 |
| 2022 | 8 |
| 2023 | 4 |
| 2024 | 5 |
| 2025 | 4 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Active, not recruiting | 32 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| BioMarin Pharmaceutical | 2 |
| Grand Valley State University | 2 |
| University of California, San Diego | 2 |
| Vanderbilt University Medical Center | 2 |
| Vertex Pharmaceuticals Incorporated | 2 |
| 4D Molecular Therapeutics | 1 |
| Abramson Cancer Center at Penn Medicine | 1 |
| Boston Children's Hospital | 1 |
| Brigham and Women's Hospital | 1 |
| Capricor 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 |
|---|---|
| Genetic Diseases, Inborn | 32 |
| Nervous System Diseases | 6 |
| Metabolic Diseases | 4 |
| Brain Diseases, Metabolic | 3 |
| Central Nervous System Diseases | 3 |
| Genetic Diseases, X-Linked | 3 |
| Hematologic Diseases | 3 |
| Hemoglobinopathies | 3 |
| Metabolism, Inborn Errors | 3 |
| Musculoskeletal Diseases | 3 |
Trials
Showing 1 to 25 of 32. 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.