Clinical trials
Genetic Diseases, Inborn trials with no applicable phase
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
97 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 4 |
| 2006 | 2 |
| 2009 | 1 |
| 2012 | 2 |
| 2014 | 2 |
| 2015 | 2 |
| 2016 | 7 |
| 2017 | 4 |
| 2018 | 6 |
| 2019 | 4 |
| 2020 | 5 |
| 2021 | 5 |
| 2022 | 5 |
| 2023 | 7 |
| 2024 | 13 |
| 2025 | 10 |
| 2026 | 4 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 46 |
| Recruiting | 21 |
| Unknown status | 10 |
| Active, not recruiting | 9 |
| Not yet recruiting | 5 |
| Terminated | 3 |
| Enrolling by invitation | 2 |
| Withdrawn | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| National Center for Research Resources (NCRR) | 5 |
| Rady Pediatric Genomics & Systems Medicine Institute | 5 |
| Assistance Publique - Hôpitaux de Paris | 3 |
| Brigham and Women's Hospital | 3 |
| Fairview University Medical Center | 3 |
| Hospices Civils de Lyon | 3 |
| IRCCS Eugenio Medea | 3 |
| Abramson Cancer Center at Penn Medicine | 2 |
| Boston Children's Hospital | 2 |
| FDA Office of Orphan Products Development | 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 | 97 |
| Rare Diseases | 22 |
| Hematologic Diseases | 9 |
| Cystic Fibrosis | 7 |
| Respiratory Tract Diseases | 7 |
| Anemia, Sickle Cell | 6 |
| Genetic Predisposition to Disease | 6 |
| Metabolism, Inborn Errors | 6 |
| Cardiovascular Diseases | 4 |
| Cerebral Palsy | 4 |
Trials
Showing 51 to 75 of 97. 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.