Trials / Completed
CompletedNCT04323098
Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A
A Phase 3b, Single Arm, Open-Label Study to Evaluate the Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII, With Prophylactic Corticosteroids in Hemophilia A Patients
- Status
- Completed
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 22 (actual)
- Sponsor
- BioMarin Pharmaceutical · Industry
- Sex
- Male
- Age
- 18 Years
- Healthy volunteers
- Not accepted
Summary
This Phase III clinical study will evaluate the safety and effectiveness of valoctocogene roxaparvovec in combination with prophylactic corticosteroids in patients with severe hemophilia A.
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
- 2020-12-08
- Primary completion
- 2023-01-27
- Completion
- 2025-05-08
- First posted
- 2020-03-26
- Last updated
- 2025-10-08
- Results posted
- 2024-04-04
Locations
12 sites across 4 countries: United States, Australia, Brazil, Taiwan
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT04323098. Inclusion in this directory is not an endorsement.