Pharmacokinetic-guided Dosing of Emicizumab
Trial Parameters
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Brief Summary
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.
Eligibility Criteria
Inclusion Criteria: * 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; * Having good bleeding control, defined as: i No spontaneous joint/muscle bleeds in the previous 6 months AND ii A maximum of two treated (traumatic) bleeds in the previous 6 months. * Willing and able to provide written informed consent, either by the subject or its parents/legal guardian * Willing to provide bleeding assessment information * Willing to adhere to the medication regimen Exclusion Criteria: * Acquired haemophilia A