Personalized dosing of emicizumab for hemophilia A patients
Pharmacokinetic-guided Dosing of Emicizumab in Congenital Haemophilia A Patients - The DosEmi Study
This study is testing if personalized dosing of the hemophilia drug emicizumab can prevent bleeding just as well as the usual dosing for patients with hemophilia A.
Quick facts
| Phase | Phase 4 |
|---|---|
| Study type | Interventional |
| Enrollment | 95 (estimated) |
| Ages | 1 Year and up |
| Sex | Male |
| Sponsor | UMC Utrecht Academic / other |
| Drugs / interventions | emicizumab |
| Locations | 8 sites (Nijmegen, Gelderland and 7 other locations) |
| Trial ID | NCT06320626 on ClinicalTrials.gov |
What this trial studies
This multicenter, prospective, open-label, cross-over clinical study aims to evaluate whether individualized pharmacokinetic (PK)-guided dosing of emicizumab is as effective as conventional dosing in preventing bleeding in patients with congenital hemophilia A. The study will include patients who have been on standard emicizumab treatment for at least 12 months and have demonstrated good bleeding control. Participants will be assigned to different dosing groups to assess the efficacy and safety of the PK-guided approach compared to standard dosing. The study seeks to improve treatment outcomes for hemophilia A patients, particularly those with inhibitors.
Who should consider this trial
Good fit: Ideal candidates include individuals aged over 1 year with a confirmed diagnosis of congenital hemophilia A and good bleeding control on conventional emicizumab dosing.
Not a fit: Patients with acquired hemophilia A will not benefit from this study.
Why it matters
Potential benefit: If successful, this approach could lead to more effective and personalized treatment for hemophilia A patients, reducing the frequency of bleeding episodes.
How similar studies have performed: Other studies have shown promising results with emicizumab in hemophilia A treatment, indicating that this PK-guided approach may build on existing successful therapies.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Nijmegen, Gelderland and 7 other locations
- Radboud University Medical Center — Nijmegen, Gelderland, Netherlands (Recruiting)
- Maastricht University Medical Center — Maastricht, Limburg, Netherlands (Recruiting)
- Amsterdam University Medical Center — Amsterdam, North Holland, Netherlands (Recruiting)
- Leids Universitair Medisch Centrum — Leiden, South Holland, Netherlands (Recruiting)
- Erasmus University Medical Center — Rotterdam, South Holland, Netherlands (Recruiting)
- HagaZiekenhuis — The Hague, South Holland, Netherlands (Recruiting)
- University Medical Center Groningen — Groningen, Netherlands (Recruiting)
- University Medical Center Utrecht — Utrecht, Netherlands (Recruiting)
Study contacts
- Principal investigator: Study Officials Fischer, Dr, MD — UMC Utrecht
- Study coordinator: Kathelijn Fischer, Dr, MD.
- Email: k.fischer@umcutrecht.nl
- Phone: +318 875 584 50
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.