The goal of this multicentre, prospective, open-label, cross-over clinical study is to determine whether individualized PK-guided dosing of emicizumab is non-inferior to conventional dosing of emicizumab in the prevention of bleeding in congenital haemophilia A patients.
Confirmed diagnosis of congenital haemophilia A, with a baseline endogenous FVIII of <6 IU/ml
Aged > 1 year at inclusion (inclusion of children 1-16 years after favourable interim-analysis see protocol)
Receiving conventional dosing of emicizumab (6 mg/kg/4 weeks with varying intervals) for a duration of at least 12 months prior to inclusion;
Willing and able to provide written informed consent, either by the subject or its parents/legal guardian
Acquired haemophilia A
Kathelijn Fischer
Lead sponsor
UMC Utrecht
Sponsor institution
Erasmus Medical Center
Collaborator
Amsterdam University Medical Center
Collaborator
Leiden University Medical Center
Collaborator
Radboud University Medical Center
Collaborator
Maastricht University Medical Center
Collaborator
University Medical Center Groningen
Collaborator
HagaZiekenhuis
Collaborator
Dutch Society of Haemophilia Patients
Collaborator