Clinical trials
Genetic Diseases, Inborn trials in Italy
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
62 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2006 | 1 |
| 2010 | 1 |
| 2011 | 5 |
| 2013 | 2 |
| 2014 | 2 |
| 2015 | 2 |
| 2016 | 4 |
| 2017 | 4 |
| 2018 | 8 |
| 2019 | 6 |
| 2021 | 7 |
| 2022 | 4 |
| 2023 | 5 |
| 2024 | 3 |
| 2025 | 3 |
| 2026 | 4 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 29 |
| Recruiting | 14 |
| Terminated | 7 |
| Active, not recruiting | 5 |
| Unknown status | 4 |
| Enrolling by invitation | 3 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Vertex Pharmaceuticals Incorporated | 5 |
| BioMarin Pharmaceutical | 4 |
| GenSight Biologics | 4 |
| Novartis Pharmaceuticals | 4 |
| IRCCS Eugenio Medea | 3 |
| Pfizer | 3 |
| QED Therapeutics, a BridgeBio company | 3 |
| Quince Therapeutics S.p.A. | 3 |
| Forma Therapeutics, Inc. | 2 |
| Genzyme, a Sanofi Company | 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 | 62 |
| Nervous System Diseases | 14 |
| Musculoskeletal Diseases | 13 |
| Hematologic Diseases | 12 |
| Bone Diseases | 7 |
| Blood Coagulation Disorders | 6 |
| Coagulation Protein Disorders | 6 |
| Hemoglobinopathies | 6 |
| Hemorrhagic Disorders | 6 |
| Neurodegenerative Diseases | 6 |
Trials
Showing 76 to 62 of 62. 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.
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