Clinical trials
Phase 2 Genetic Diseases, Inborn trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
70 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 1 |
| 2001 | 1 |
| 2003 | 1 |
| 2006 | 2 |
| 2007 | 3 |
| 2009 | 4 |
| 2010 | 2 |
| 2011 | 5 |
| 2012 | 3 |
| 2013 | 1 |
| 2014 | 1 |
| 2016 | 1 |
| 2017 | 1 |
| 2018 | 6 |
| 2019 | 3 |
| 2020 | 1 |
| 2021 | 4 |
| 2022 | 3 |
| 2024 | 1 |
| 2025 | 3 |
| 2026 | 5 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 42 |
| Terminated | 11 |
| Unknown status | 6 |
| Recruiting | 6 |
| Enrolling by invitation | 3 |
| Not yet recruiting | 1 |
| Active, not recruiting | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| National Center for Research Resources (NCRR) | 6 |
| Vertex Pharmaceuticals Incorporated | 4 |
| National Cancer Institute (NCI) | 3 |
| Novartis Pharmaceuticals | 3 |
| QED Therapeutics, a BridgeBio company | 3 |
| Emory University | 2 |
| Kastle Therapeutics, LLC | 2 |
| National Institute of Neurological Disorders and Stroke (NINDS) | 2 |
| National Institutes of Health Clinical Center (CC) | 2 |
| Pharming Technologies B.V. | 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 | 70 |
| Rare Diseases | 14 |
| Metabolic Diseases | 8 |
| Musculoskeletal Diseases | 8 |
| Nervous System Diseases | 8 |
| Anemia, Sickle Cell | 7 |
| Cystic Fibrosis | 7 |
| Hematologic Diseases | 7 |
| Infant, Newborn, Diseases | 6 |
| Metabolism, Inborn Errors | 6 |
Trials
Showing 1 to 25 of 70. 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.
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