Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A

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.

Timeline

Start

2020-12-08

Primary completion

2023-01-27

Completion

2025-05-08

Outcome

Met primary endpoint

registry MEAN BMN 270 (Valoctocogene roxaparvovec): 15.13 (Standard Deviation 22.38) IU /dL NCT04323098 ↗

registry p = 0.0057; t-test, 2 sided; note: P-value was based on two-sided t-test against 0. NCT04323098 ↗

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	valoctocogene roxaparvovec	Gene therapy (AAV / viral vector)	6e+13 vg/kg	—

Indications