Trials / Completed
CompletedNCT03392974
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
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
- Status
- Completed
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 1 (actual)
- Sponsor
- BioMarin Pharmaceutical · Industry
- Sex
- Male
- Age
- 18 Years
- Healthy volunteers
- Not accepted
Summary
This Phase III clinical study will assess the efficacy of BMN 270 defined as FVIII activity, during weeks 49-52 following intravenous infusion of BMN 270 and assess the impact of BMN 270 on usage of exogenous FVIII replacement therapy and the number of bleeding episodes from week 5 to week 52.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| BIOLOGICAL | Valoctocogene Roxaparvovec | Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A |
Timeline
- Start date
- 2018-03-14
- Primary completion
- 2019-05-22
- Completion
- 2023-06-05
- First posted
- 2018-01-08
- Last updated
- 2023-10-03
- Results posted
- 2021-10-08
Locations
1 site across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT03392974. Inclusion in this directory is not an endorsement.