NCT03392974View on ClinicalTrials.gov

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg

PHASE3CompletedINTERVENTIONAL
Enrollment

1

Participants

Timeline

Start Date

March 14, 2018

Primary Completion Date

May 22, 2019

Study Completion Date

June 5, 2023

Conditions
Hemophilia A
Interventions
BIOLOGICAL

Valoctocogene Roxaparvovec

Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A

Trial Locations (1)

15213-4306

Hemophilia Center of Western Pennsylvania, Pittsburgh

Sponsors
All Listed Sponsors
lead

BioMarin Pharmaceutical

INDUSTRY