Clinical trials
Observational Genetic Diseases, Inborn trials in the United States
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
114 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 2 |
| 2001 | 3 |
| 2002 | 1 |
| 2003 | 1 |
| 2004 | 3 |
| 2005 | 1 |
| 2006 | 2 |
| 2007 | 2 |
| 2008 | 1 |
| 2009 | 1 |
| 2010 | 2 |
| 2011 | 5 |
| 2012 | 6 |
| 2013 | 2 |
| 2014 | 4 |
| 2015 | 3 |
| 2016 | 2 |
| 2017 | 3 |
| 2018 | 5 |
| 2019 | 11 |
| 2020 | 6 |
| 2021 | 6 |
| 2022 | 7 |
| 2023 | 8 |
| 2024 | 4 |
| 2025 | 3 |
| 2026 | 5 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 53 |
| Recruiting | 30 |
| Unknown status | 10 |
| Enrolling by invitation | 7 |
| Active, not recruiting | 7 |
| Terminated | 6 |
| Suspended | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| National Human Genome Research Institute (NHGRI) | 12 |
| National Center for Research Resources (NCRR) | 9 |
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 6 |
| National Institute of Allergy and Infectious Diseases (NIAID) | 5 |
| Boston Children's Hospital | 4 |
| Columbia University | 4 |
| National Heart, Lung, and Blood Institute (NHLBI) | 4 |
| Children's Hospital of Philadelphia | 2 |
| Editas Medicine, Inc. | 2 |
| Genomic Prediction Inc. | 2 |
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 | 114 |
| Rare Diseases | 23 |
| Metabolic Diseases | 11 |
| Metabolism, Inborn Errors | 11 |
| Congenital Abnormalities | 7 |
| Nervous System Diseases | 7 |
| Genetic Predisposition to Disease | 6 |
| Lysosomal Storage Diseases | 6 |
| Musculoskeletal Diseases | 5 |
| Neurodevelopmental Disorders | 5 |
Trials
Showing 1 to 25 of 114. Each trial links to its page on Pharma Observatory.
14 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
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