Clinical trials
Terminated Genetic Diseases, Inborn trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
32 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2000 | 1 |
| 2001 | 1 |
| 2006 | 2 |
| 2007 | 1 |
| 2009 | 2 |
| 2011 | 3 |
| 2014 | 1 |
| 2015 | 1 |
| 2016 | 4 |
| 2018 | 2 |
| 2019 | 4 |
| 2021 | 6 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Terminated | 32 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Eloxx Pharmaceuticals, Inc. | 2 |
| HemoShear Therapeutics | 2 |
| National Center for Research Resources (NCRR) | 2 |
| Novartis Pharmaceuticals | 2 |
| BioMarin Pharmaceutical | 1 |
| Centre Hospitalier Universitaire, Amiens | 1 |
| Children's Hospital Los Angeles | 1 |
| Dicerna Pharmaceuticals, Inc., a Novo Nordisk company | 1 |
| Fairview University Medical Center | 1 |
| Fred Hutchinson Cancer Center | 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 | 32 |
| Metabolic Diseases | 7 |
| Metabolism, Inborn Errors | 5 |
| Rare Diseases | 5 |
| Hematologic Diseases | 3 |
| Muscular Dystrophy, Duchenne | 3 |
| Musculoskeletal Diseases | 3 |
| Nervous System Diseases | 3 |
| Anemia, Aplastic | 2 |
| Blood Coagulation Disorders | 2 |
Trials
Showing 76 to 32 of 32. 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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