Safety, pharmacokinetics and efficacy of a subcutaneous recombinant FVIII (OCTA101) in adult patients with severe haemophilia A.

Toshko LissitchkovMartina JansenJohann BichlerSigurd Knaub

Published in: Haemophilia : the official journal of the World Federation of Hemophilia (2023)

Despite promising PK and efficacy results, the trial was terminated due to the incidence of FVIII inhibitors. The occurrence of inhibitors at two dose levels suggests that their development may be related to the subcutaneous route of administration.

Keyphrases

risk assessment
clinical trial
risk factors
study protocol
phase iii
early onset
phase ii
drug induced
double blind