Clinical trials
Genetic Diseases, Inborn trials with unknown status
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
58 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 1 |
| 2010 | 1 |
| 2011 | 2 |
| 2012 | 1 |
| 2013 | 2 |
| 2014 | 1 |
| 2016 | 4 |
| 2017 | 5 |
| 2018 | 3 |
| 2019 | 8 |
| 2020 | 4 |
| 2021 | 7 |
| 2022 | 5 |
| 2023 | 4 |
| 2024 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Unknown status | 58 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| National Center for Research Resources (NCRR) | 5 |
| Fairview University Medical Center | 2 |
| Igentify Ltd | 2 |
| Maastricht University Medical Center | 2 |
| Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University | 2 |
| Abbott Molecular | 1 |
| Arcensus GmbH | 1 |
| Assistance Publique - Hôpitaux de Paris | 1 |
| Boston Children's Hospital | 1 |
| CENTOGENE GmbH Rostock | 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 | 58 |
| Rare Diseases | 12 |
| Cardiovascular Diseases | 4 |
| Cystic Fibrosis | 4 |
| Respiratory Tract Diseases | 4 |
| Immune System Diseases | 3 |
| Primary Immunodeficiency Diseases | 3 |
| Arrhythmias, Cardiac | 2 |
| Cerebellar Ataxia | 2 |
| Communicable Diseases | 2 |
Trials
Showing 76 to 58 of 58. Each trial links to its page on Pharma Observatory.
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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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