Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)

Completed

Plain-English translation of NCT03370913 on ClinicalTrials.gov ↗ · Source last updated March 2025 · How we translate trials

Phase 3 — Testing in thousands of people, comparing the treatment against what doctors currently use. This is the last big step before approval.

Trial locations(49 sites)

United States

Los Angeles Orthopedic Hospital, Orthopedic Hemophilia Treatment Center, Los Angeles, California

UC Davis Hemophilia Treatment Center, Sacramento, California

University of California San Diego, Hematology and Oncology, Hemophilia &Thrombosis Treatment Center, San Diego, California

UCSF Medical Center, San Francisco, California

University of Colorado, Aurora, Colorado