Pharmacokinetic-guided Dosing of Emicizumab
Pharmacokinetic-guided Dosing of Emicizumab in Congenital Haemophilia A Patients - The DosEmi Study
Kathelijn Fischer
95 participants
Sep 8, 2022
INTERVENTIONAL
Conditions
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
Inclusion Criteria8
- 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 Criteria1
- Acquired haemophilia A
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Interventions
PK-guided dose reduction emicizumab targeted at a Ctrough of 30μg/mL.
Continue on their current dose regimen
Adjusted in dosing regimen according to local protocol
Locations(8)
View Full Details on ClinicalTrials.gov
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NCT06320626