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

What this Trial Is About

Researchers are evaluating whether personalized, pharmacokinetic (PK)-guided dosing of emicizumab is as effective as conventional dosing in preventing bleeding in patients with congenital haemophilia A. Haemophilia A is a genetic bleeding disorder caused by a deficiency or dysfunction of clotting factor VIII, leading to bleeding episodes mainly in joints. Emicizumab is a bispecific antibody mimicking factor VIII, administered subcutaneously, and has shown promising results in reducing bleeding rates compared to traditional treatments. The study involves multiple groups based on patients' emicizumab blood levels. Patients will be followed retrospectively and prospectively on conventional dosing for 6 months each. Those with emicizumab concentrations of 40 µg/mL or higher will receive individualized PK-guided dose reductions targeting a trough concentration of 30 µg/mL and will be followed for 12 months. Patients with concentrations between 25 and 39 µg/mL continue their current dose for 12 months, while those below 25 µg/mL have their dosing adjusted per local protocols and are monitored for safety data. Participants will undergo regular monitoring to assess bleeding events, joint health through physical exams and ultrasound, biomarker evaluations, quality of life, sports participation, and pain during emicizumab administration. The main outcome measured is the proportion of patients without treated bleeds over 12 months. Secondary outcomes include longer-term bleeding rates, joint health stability, cost-effectiveness, and pharmacodynamic biomarker performance. The study spans up to 24 months of follow-up per participant.

Pharmacokinetic-guided Dosing of Emicizumab