Emicizumab dosing based on the concentration in blood of patients with haemophilia A – The DosEmi study

Pharmacokinetic-guided dosing of emicizumab in congenital haemophilia A patients – The DosEmi study - DosEmi study

Status

Active, not recruiting

Phases

Phase 4

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2021-004039-10-NL

Enrollment

Registered

2022-01-24

Start date

2022-06-24

Completion date

Unknown

Last updated

2025-02-03

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Congenital hemophilia A MedDRA version: 20.0 Level: LLT Classification code 10060612 Term: Hemophilia A System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Trade Name: Hemlibra Product Name: Hemlibra / Emicizumab Product Code: RO5534262/ACE910 Pharmaceutical Form: Solution for injection

Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: - Confirmed diagnosis of congenital haemophilia A, with a baseline endogenous FVIII of 1 year at inclusion - Receiving conventional dosing of emicizumab (6 mg/kg/4 weeks with varying intervals) for a duration of at least 6 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 Are the trial subjects under 18? yes Number of subjects for this age range: 33 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 57 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 5

Exclusion criteria

Exclusion criteria: Acquired haemophilia A

Design outcomes

Primary

Measure	Time frame
Main Objective: The primary objective 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.;Secondary Objective: -% of patients without treated bleeds: 12M Clinical Phase+Bleeding Assessment Phase vs 12M PK-guided Dosing Phase+Dose Continuation Phase -% of patients with spontaneous joint/muscle bleeds: 6 and 12M Clinical Phase+Bleeding Assessment Phase vs 6 and 12M PK-guided Dosing Phase+Dose Continuation Phase -Annualized bleeding rate of treated bleeds, including joint/sports-induced bleeds: 6M Bleeding Assessment Phase vs6M PK-Guided Dosing Phase -Annualized bleeding rate of treated bleeds, including joint/sports-induced bleeds: 6M retrospective + 6M prospective data (Clinical Phase+Bleeding Assessment Phase) vs12M prospective data of PK guided dosing (PK-guided Dosing Phase+Dose Continuation Phase) -Cost-effectiveness between 6M of conventional dosing (Bleeding Assessment Phase) vs 6M of individualized PK-guided dosing (PK-Guided Dosing Phase) -Performance of MAP Bayesian procedure -Joint status -QoL -Pain at emicizumab administration -Sports participation -Coagulation potential;Primary end point(s): The primary outcome parameter is bleeding, defined as: Proportion of patients without treated bleeds (6 months Bleeding Assessment Phase versus 6 months PK-Guided Dosing Phase). ;Timepoint(s) of evaluation of this end point: All endpoints will be assessed at the end of follow-up time (6 months after intervention).	—

Measure

Time frame

Main Objective: The primary objective 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.;Secondary Objective: -% of patients without treated bleeds: 12M Clinical Phase+Bleeding Assessment Phase vs 12M PK-guided Dosing Phase+Dose Continuation Phase -% of patients with spontaneous joint/muscle bleeds: 6 and 12M Clinical Phase+Bleeding Assessment Phase vs 6 and 12M PK-guided Dosing Phase+Dose Continuation Phase -Annualized bleeding rate of treated bleeds, including joint/sports-induced bleeds: 6M Bleeding Assessment Phase vs6M PK-Guided Dosing Phase -Annualized bleeding rate of treated bleeds, including joint/sports-induced bleeds: 6M retrospective + 6M prospective data (Clinical Phase+Bleeding Assessment Phase) vs12M prospective data of PK guided dosing (PK-guided Dosing Phase+Dose Continuation Phase) -Cost-effectiveness between 6M of conventional dosing (Bleeding Assessment Phase) vs 6M of individualized PK-guided dosing (PK-Guided Dosing Phase) -Performance of MAP Bayesian procedure -Joint status -QoL -Pain at emicizumab administration -Sports participation -Coagulation potential;Primary end point(s): The primary outcome parameter is bleeding, defined as: Proportion of patients without treated bleeds (6 months Bleeding Assessment Phase versus 6 months PK-Guided Dosing Phase). ;Timepoint(s) of evaluation of this end point: All endpoints will be assessed at the end of follow-up time (6 months after intervention).

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Secondary

Measure	Time frame
Secondary end point(s): The secondary outcomes are defined as: - Proportion of patients without treated bleeds (12 months Clinical Phase+Bleeding Assessment Phase versus 12 months PK-guided Dosing Phase+Dose Continuation Phase) - Proportion of patients with spontaneous joint- or muscle bleeds (6 and 12 months Clinical Phase+Bleeding Assessment Phase versus 6 and 12 months PK-guided Dosing Phase+Dose Continuation Phase). - Annualized bleeding rate (ABR) of treated bleeds, including joint bleeds and sports induced bleeds (6 months Bleeding Assessment Phase versus 6 months PK-Guided Dosing Phase). - Annualized bleeding rate (ABR) of treated bleeds, including joint bleeds and sports induced bleeds (6 months retrospective + 6 months prospective data (Clinical Phase+Bleeding Assessment Phase) versus 12 months prospective data of PK guided dosing (PK-guided Dosing Phase+Dose Continuation Phase). - Cost-effectiveness between 6 months of conventional dosing (Bleeding Assessment Phase) and 6 months of individualized PK-guided dosing of emicizumab (PK-Guided Dosing Phase): o Direct and indirect medical costs: Direct medical costs are predominantly determined by consumption of emicizumab, additional FVIII, and/or bypassing agents, extracted from the hospital’s pharmacy records. These data are highly reliable, as this medication is exclusively distributed by haemophilia treatment centers. o Indirect medical costs: are number of (emergency) hospital visits, bleeding related hospital admissions and/or unscheduled surgeries, and days lost from work/school (for patients and/or caregivers). - Predictive performance of the MAP Bayesian procedure used for the dose adaptation procedure, defined as % of patients within ±20% of target level/within the target level of 25-39 µg/mL of emicizumab. All emicizumab plasma levels will be determined in the laboratory of the University Medical Center Utrecht using a specifically developed LC-MS/MS. [24] - Joint status as measured by physical e	—

Measure

Time frame

Secondary end point(s): The secondary outcomes are defined as: - Proportion of patients without treated bleeds (12 months Clinical Phase+Bleeding Assessment Phase versus 12 months PK-guided Dosing Phase+Dose Continuation Phase) - Proportion of patients with spontaneous joint- or muscle bleeds (6 and 12 months Clinical Phase+Bleeding Assessment Phase versus 6 and 12 months PK-guided Dosing Phase+Dose Continuation Phase). - Annualized bleeding rate (ABR) of treated bleeds, including joint bleeds and sports induced bleeds (6 months Bleeding Assessment Phase versus 6 months PK-Guided Dosing Phase). - Annualized bleeding rate (ABR) of treated bleeds, including joint bleeds and sports induced bleeds (6 months retrospective + 6 months prospective data (Clinical Phase+Bleeding Assessment Phase) versus 12 months prospective data of PK guided dosing (PK-guided Dosing Phase+Dose Continuation Phase). - Cost-effectiveness between 6 months of conventional dosing (Bleeding Assessment Phase) and 6 months of individualized PK-guided dosing of emicizumab (PK-Guided Dosing Phase): o Direct and indirect medical costs: Direct medical costs are predominantly determined by consumption of emicizumab, additional FVIII, and/or bypassing agents, extracted from the hospital’s pharmacy records. These data are highly reliable, as this medication is exclusively distributed by haemophilia treatment centers. o Indirect medical costs: are number of (emergency) hospital visits, bleeding related hospital admissions and/or unscheduled surgeries, and days lost from work/school (for patients and/or caregivers). - Predictive performance of the MAP Bayesian procedure used for the dose adaptation procedure, defined as % of patients within ±20% of target level/within the target level of 25-39 µg/mL of emicizumab. All emicizumab plasma levels will be determined in the laboratory of the University Medical Center Utrecht using a specifically developed LC-MS/MS. [24] - Joint status as measured by physical e

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Countries

Netherlands

Contacts

Public ContactVan Creveldkliniek Research

University Medical Center Utrecht

00310887558450

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 9, 2026