Implementation of pharmacokinetic-guided dosing of prophylaxis in hemophilia patients

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON27791

Enrollment

Registered

2018-10-04

Start date

2019-01-01

Completion date

Unknown

Last updated

2024-02-28

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

Conditions

Bleeding, hemophilia, pharmacokinetics, prophylaxis

Interventions

Stratum 1(A/B): Hemophilia patients on SHL prophylaxis undergo individual PK-profiling on SHL. During an initial total period of 12 weeks, all patients will be on PK-guided treatment dosing. Periodic

Eligibility

Inclusion criteria

Inclusion criteria: - Hemophilia A and B patients of all ages on prophylaxis; - Prophylaxis with SHL factor concentrates or EHL factor concentrates; - Severe (FVIII/ FIX<0.01 IU/mL) and non-severe (FVIII/FIX¡Ý0.01 IU/mL) hemophilia patients; - Initiation of EHL or other novel products in compliance with national guidelines; - Written (parental) informed consent, according to local law and regulations

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. Due to the intention to treat analyses that will be applied, patients who develop inhibitors during the intervention will still be included in the statistical analyses.

Design outcomes

Primary

Measure	Time frame
1. Observed trough and peak FVIII and FIX levels in comparison to predicted FVIII and FIX levels by population PK-modeling (predictive performance).	—

Secondary

Measure	Time frame
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 (standard half-life versus extended half-life) as reported by patient/ caretakers and physician will be measured using a Visual Analogue Scale (VAS) at the start and end of the study (Appendix VAS). 3. Economic analysis in which costs and benefits of standard prophylactic treatment and PK-guided prophylaxis are compared. 4. Analysis of modifiers effecting PK parameters of FVIII/FIX concentrate in order to further optimize population PK models.	—

Measure

Time frame

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 (standard half-life versus extended half-life) as reported by patient/ caretakers and physician will be measured using a Visual Analogue Scale (VAS) at the start and end of the study (Appendix VAS). 3. Economic analysis in which costs and benefits of standard prophylactic treatment and PK-guided prophylaxis are compared. 4. Analysis of modifiers effecting PK parameters of FVIII/FIX concentrate in order to further optimize population PK models.

—

Contacts

Public ContactM.H. Cnossen

Pediatric hematologist Erasmus Medical Centre - Sophia Children's Hospital Postbus 2060

m.cnossen@erasmusmc.nl+31-107036691

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)