Clinical trial
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene…
NCT03392974 · BioMarin Pharmaceutical, Industry · Completed
Key facts
| NCT number | NCT03392974 |
|---|---|
| Title | Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg |
| 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 at a Dose of 4E13vg/kg in Hemophilia A Patients With Residual FVIII Levels ≤1IU/dL Receiving Prophylactic FVIII Infusions |
| Acronym | GENEr8-2 |
| Lead sponsor | BioMarin Pharmaceutical, Industry |
| Status | Completed |
| Phase | Phase 3 |
| Study type | Interventional |
| Conditions | Hemophilia A |
| Intervention type | Biological |
| Interventions | Valoctocogene Roxaparvovec |
| Enrollment | 1 |
| Start date | Mar 14, 2018 |
| Primary completion | May 22, 2019 |
| Completion | Jun 5, 2023 |
| First posted | Jan 8, 2018 |
| Last update posted | Oct 3, 2023 |
| Countries | United States |
At a glance
- 1–9 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: