Clinical trials
Completed Genetic Diseases, Inborn trials in the United States
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
156 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 5 |
| 2001 | 3 |
| 2002 | 1 |
| 2003 | 1 |
| 2004 | 3 |
| 2005 | 1 |
| 2006 | 2 |
| 2007 | 3 |
| 2008 | 3 |
| 2009 | 7 |
| 2010 | 3 |
| 2011 | 6 |
| 2012 | 10 |
| 2013 | 2 |
| 2014 | 7 |
| 2015 | 4 |
| 2016 | 7 |
| 2017 | 9 |
| 2018 | 16 |
| 2019 | 12 |
| 2020 | 8 |
| 2021 | 2 |
| 2022 | 7 |
| 2023 | 6 |
| 2025 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 156 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| National Human Genome Research Institute (NHGRI) | 8 |
| BioMarin Pharmaceutical | 6 |
| Regeneron Pharmaceuticals | 6 |
| National Cancer Institute (NCI) | 5 |
| National Center for Research Resources (NCRR) | 5 |
| National Heart, Lung, and Blood Institute (NHLBI) | 5 |
| Vertex Pharmaceuticals Incorporated | 5 |
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 4 |
| GenSight Biologics | 4 |
| Columbia University | 3 |
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 | 156 |
| Rare Diseases | 23 |
| Hematologic Diseases | 22 |
| Metabolism, Inborn Errors | 17 |
| Nervous System Diseases | 17 |
| Musculoskeletal Diseases | 15 |
| Anemia, Sickle Cell | 14 |
| Metabolic Diseases | 14 |
| Respiratory Tract Diseases | 11 |
| Cystic Fibrosis | 10 |
Trials
Showing 1 to 25 of 156. Each trial links to its page on Pharma Observatory.
56 more trials match. See them 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.