Hemophilia MedDRA version: 20.0 Level: LLT Classification code 10060614 Term: Hemophilia B (Factor IX) System Organ Class: 100000004850 MedDRA version: 20.0 Level: LLT Classification code 10060613 Term: Hemophilia A (Factor VIII) System Organ Class: 100000004850
Conditions
Interventions
Trade Name: Advate
Pharmaceutical Form: Powder and solvent for solution for injection
Trade Name: Kogenate Bayer
Pharmaceutical Form: Powder and solven
Sponsors
Erasmus University Medical Center
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: - Hemophilia A and B patients of all ages on prophylaxis; -Prophylaxis with SHL factor concentrate or EHL factor concentrate; -Written (parental) informed consent, according to local law and regulations Are the trial subjects under 18? yes Number of subjects for this age range: 25 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 20 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 5
Exclusion criteria
Exclusion criteria: -Patients with other congenital or acquired hemostatic abnormalities; -General medical conditions which may interfere with participation in the study; -Inability to adhere to prophylaxis and/ or inability to keep detailed logs of infusions and bleeding episodes; -Withdrawal of (parental) informed consent.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 1. Observed FVIII and FIX levels in comparison to predicted FVIII and FIX levels by population PK-modeling (predictive performance).;Primary end point(s): Observed trough and peak FVIII and FIX levels in comparison to predicted FVIII and FIX levels by population PK-modeling (predictive performance).;Timepoint(s) of evaluation of this end point: Whole study period, maximum 6 years (1 January 2019 till 1 January 2025); Secondary Objective: 1. Association of (real life or simulated) FVIII/FIX (trough and peak levels) with bleeding episodes and daily activities. 2. Expectations, feasibility and experience with PK-guided dosing with the different factor concentrates (SHL versus EHL) as reported by patient/ caretakers and physician will be measured using a Visual Analogue Scale (VAS) questionnaire. 3. Economic analysis in which costs and benefits of standard prophylactic treatment and PK-guided prophylaxis will be compared. 4. Analysis of modifiers effecting PK parameters of FVIII/FIX concentrate in order to further optimize population PK models. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Association of (real life or simulated) FVIII/FIX (trough and peak levels) with bleeding episodes and daily activities. 2. Expectations, feasibility and experience with PK-guided dosing with the different factor concentrates (SHL versus EHL) as reported by patient/ caretakers and physician will be measured using VAS questionnaires (VAS (start of the study; end of the study)). 3. Economic analysis in which costs and benefits of standard prophylactic treatment and PK-guided prophylaxis will be compared. 4. Analysis of modifiers effecting PK parameters of FVIII/FIX concentrate in order to further optimize population PK models. This will be addressed by analysis of patient and treatment characteristics with effect on FVIII/FIX levels and PK parameters. ;Timepoint(s) of evaluation of this end point: Whole study period, maximum 6 years (1 January 2019 till 1 January 2025) | — |
Countries
Netherlands
Contacts
Public ContactM.H.Cnossen
Erasmus University Medical Center
Outcome results
None listed