Clinical trials
Terminated Phase 1 Genetic Diseases, Inborn trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
4 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2016 | 2 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Terminated | 4 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Dicerna Pharmaceuticals, Inc., a Novo Nordisk company | 1 |
| Eloxx Pharmaceuticals, Inc. | 1 |
| New York Blood Center | 1 |
| University of Pennsylvania | 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 | 4 |
| Anemia, Aplastic | 1 |
| Anemia, Refractory, with Excess of Blasts | 1 |
| Hyperoxaluria | 1 |
| Kidney Calculi | 1 |
| Leukemia | 1 |
| Lymphoma | 1 |
| Metabolic Diseases | 1 |
| Metabolism, Inborn Errors | 1 |
| Ornithine Carbamoyltransferase Deficiency Disease | 1 |
Trials
Showing 1 to 4 of 4. 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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