RecruitingPhase 4

Pharmacokinetic-guided Dosing of Emicizumab

Netherlands95 participantsStarted 2022-09-08

Plain-language 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.

Who can participate

Age range1 Year

SexMALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

Proportion of patients without treated bleeds

Timeframe: 12 months

Trial details

NCT IDNCT06320626

SponsorKathelijn Fischer

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2026-03

Contact for this trial

Kathelijn Fischer, Dr, MD.

+318 875 584 50 k.fischer@umcutrecht.nl

View on ClinicalTrials.gov More Hemophilia A With Inhibitor trials