Clinical trials
Completed Genetic Diseases, Inborn trials with no applicable phase
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
46 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 2 |
| 2006 | 1 |
| 2009 | 1 |
| 2012 | 2 |
| 2014 | 2 |
| 2015 | 2 |
| 2016 | 3 |
| 2017 | 1 |
| 2018 | 6 |
| 2019 | 2 |
| 2020 | 2 |
| 2021 | 3 |
| 2023 | 4 |
| 2024 | 1 |
| 2025 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 46 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| National Center for Research Resources (NCRR) | 5 |
| FDA Office of Orphan Products Development | 2 |
| University of North Carolina, Chapel Hill | 2 |
| Aalborg University Hospital | 1 |
| Abramson Cancer Center at Penn Medicine | 1 |
| Assistance Publique - Hôpitaux de Paris | 1 |
| Boston Children's Hospital | 1 |
| Brigham and Women's Hospital | 1 |
| Centre Hospitalier Universitaire de la Réunion | 1 |
| 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 | 46 |
| Rare Diseases | 17 |
| Cystic Fibrosis | 7 |
| Hematologic Diseases | 7 |
| Respiratory Tract Diseases | 7 |
| Anemia, Sickle Cell | 6 |
| Genetic Predisposition to Disease | 4 |
| Metabolism, Inborn Errors | 4 |
| Anemia, Diamond-Blackfan | 2 |
| Autism Spectrum Disorder | 2 |
Trials
Showing 1 to 25 of 46. 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
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