Pharmacokinetic-guided Dosing of Emicizumab

Launched by KATHELIJN FISCHER · Mar 12, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a medication called emicizumab, which helps prevent bleeding in patients with a condition known as congenital hemophilia A. The researchers want to find out if adjusting the dose of emicizumab based on how each person's body processes the medication (this is called pharmacokinetic-guided dosing) is just as effective as the standard dosing method that many patients currently use. The trial will involve participants from different centers and is aimed at both children and adults who have been receiving the standard treatment for at least a year and have had good control over their bleeding.

To be eligible for this trial, participants need to be over one year old and have a confirmed diagnosis of congenital hemophilia A, showing very low levels of a certain protein important for blood clotting. They should also have had minimal bleeding episodes in the past six months. Participants will be asked to follow specific medication guidelines and will be monitored closely throughout the study. This could be an important opportunity for patients to help researchers better understand how to tailor treatments for hemophilia A.

Gender

MALE

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