Pharmacokinetic-guided Dosing of Emicizumab in Congenital Haemophilia A Patients - The DosEmi Study
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;
• 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