A multicentre, open-label study of emicizumab given every 2 or 4 weeks in children with severe haemophilia A without inhibitors.

Midori ShimaKeiji NogamiSayaka NagamiSeitaro YoshidaKoichiro Yoneyama Akira IshiguroTakashi SuzukiMasashi Taki

Published in: Haemophilia : the official journal of the World Federation of Hemophilia (2019)

Emicizumab administered Q2W or Q4W was efficacious and safe in paediatric patients with severe haemophilia A without inhibitors. This study was registered at http://www.clinicaltrials.jp (JapicCTI-173710).

Keyphrases

open label
clinical trial
emergency department
early onset
young adults
intensive care unit
study protocol
randomized controlled trial
phase iii
rectal cancer
phase ii
gestational age