Clinical trials
Completed Genetic Diseases, Inborn trials in Italy
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
29 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2006 | 1 |
| 2010 | 1 |
| 2011 | 2 |
| 2013 | 2 |
| 2014 | 1 |
| 2015 | 1 |
| 2016 | 3 |
| 2017 | 3 |
| 2018 | 7 |
| 2019 | 4 |
| 2022 | 2 |
| 2023 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 29 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| GenSight Biologics | 4 |
| Genzyme, a Sanofi Company | 2 |
| Novartis Pharmaceuticals | 2 |
| Novo Nordisk A/S | 2 |
| Pfizer | 2 |
| QED Therapeutics, a BridgeBio company | 2 |
| Quince Therapeutics S.p.A. | 2 |
| Swedish Orphan Biovitrum | 2 |
| Alexion Pharmaceuticals, Inc. | 1 |
| Azafaros B.V. | 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 | 29 |
| Nervous System Diseases | 8 |
| Hematologic Diseases | 7 |
| Blood Coagulation Disorders | 5 |
| Coagulation Protein Disorders | 5 |
| Hemorrhagic Disorders | 5 |
| Eye Diseases | 4 |
| Eye Diseases, Hereditary | 4 |
| Hemophilia A | 4 |
| Mitochondrial Diseases | 4 |
Trials
Showing 1 to 25 of 29. 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.
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