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Pharmacokinetic-guided dosing of emicizumab in congenital haemophilia A patients – The DosEmi study
EU CTIS ID: 2024-515528-35-00
What this study is testing
To determine whether individualized pharmacokinetic (PK)-guided dosing of emicizumab is non-inferior to conventional dosing of emicizumab in the prevention of treated bleeds, spontaneous joint- or muscle bleeds, and overall bleeding in patients with congenital haemophilia A.
- Phase III and phase IV (Integrated)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Confirmed diagnosis of congenital haemophilia A, with a baseline endogenous FVIII of <6 IU/mL;
- Aged > 1 year at inclusion
- 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
You likely can't join if
- Acquired haemophilia A
See the full eligibility criteria
- Confirmed diagnosis of congenital haemophilia A, with a baseline endogenous FVIII of <6 IU/mL;
- Aged > 1 year at inclusion
- 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
- Acquired haemophilia A
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 0-17 years, 65+ years, 18-64 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.