RECRUITINGPhase 4INTERVENTIONAL
Pharmacokinetic-guided Dosing of Emicizumab
Pharmacokinetic-guided Dosing of Emicizumab in Congenital Haemophilia A Patients - The DosEmi Study
About This Trial
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 May Be Eligible (Plain English)
Who May Qualify:
- 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 willing to sign a consent form, either by the subject or its parents/legal guardian
- Willing to provide bleeding assessment information
- Willing to adhere to the medication regimen
Who Should NOT Join This Trial:
- Acquired haemophilia A
Always talk to your doctor about whether this trial is right for you.
Original Eligibility Criteria
View original clinical language
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
Treatments Being Tested
OTHER
Emicizumab - PK-guided dose reduction
PK-guided dose reduction emicizumab targeted at a Ctrough of 30μg/mL.
OTHER
Emicizumab - Dosis continuation group
Continue on their current dose regimen
OTHER
Emicizumab - Dose adjustment group
Adjusted in dosing regimen according to local protocol
Locations (8)
Radboud University Medical Center
Nijmegen, Gelderland, Netherlands
Maastricht University Medical Center
Maastricht, Limburg, Netherlands
Amsterdam University Medical Center
Amsterdam, North Holland, Netherlands
Leids Universitair Medisch Centrum
Leiden, South Holland, Netherlands
Erasmus University Medical Center
Rotterdam, South Holland, Netherlands
HagaZiekenhuis
The Hague, South Holland, Netherlands
University Medical Center Groningen
Groningen, Netherlands
University Medical Center Utrecht
Utrecht, Netherlands