Clinical trial
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene…
NCT03370913 · BioMarin Pharmaceutical, Industry · Completed
Key facts
| NCT number | NCT03370913 |
|---|---|
| Title | Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301) |
| Official title | A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients With Residual FVIII Levels ≤ 1 IU/dL Receiving Prophylactic FVIII Infusions |
| Acronym | BMN 270-301 |
| Lead sponsor | BioMarin Pharmaceutical, Industry |
| Status | Completed |
| Phase | Phase 3 |
| Study type | Interventional |
| Conditions | Hemophilia A |
| Intervention type | Biological |
| Interventions | valoctocogene roxaparvovec |
| Enrollment | 144 |
| Start date | Dec 19, 2017 |
| Primary completion | Nov 16, 2020 |
| Completion | Nov 20, 2024 |
| First posted | Dec 13, 2017 |
| Last update posted | Mar 25, 2025 |
| Countries | Australia, Belgium, Brazil, France, Germany, Israel, Italy, South Africa, South Korea, Spain, Taiwan, United Kingdom and 1 more |
At a glance
- 10–99 Study sites
- Yes Results posted
- None Linked FDA submissions
Counts are shown as ranges. Exact figures, trends and every underlying record are available in Orca1.
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Source: U.S. National Library of Medicine (NLM), ClinicalTrials.gov · Source date: