At a glance
ClinicalIndex Comparison Record- ✓Body weight at least 40 kg at screening
- ✓Diagnosis of severe congenital hemophilia A or hemophilia A with FVIII inhibitors
- ✓Currently using or willing to switch to rFVIIa as primary bypassing agent for breakthrough bleeds
- ✓FVIII inhibitor test performed during screening with titer results available before first study drug dose
- ✕Inherited or acquired bleeding disorder other than hemophilia A
- ✕Ongoing or planned immune tolerance induction therapy (washout allowed for failed ITI)
- ✕History of illicit drug or alcohol abuse within 48 weeks prior to screening
- ✕High risk for thrombotic microangiopathy (TMA) including previous personal or family history
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
A Multicenter, Open-Label, Phase III Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Emicizumab Given Every 4 Weeks (Q4W) in Patients With Hemophilia A
In Brief
A Phase 3 clinical trial evaluating Emicizumab for Hemophilia A. Completed, enrolled 48 participants across 17 sites in 6 countries.
Detailed Summary
This multicenter, open-label, non-randomized study will assess the efficacy, safety, pharmacokinetics, and pharmacodynamics of emicizumab administered at a dose of 6 milligrams per kilogram (mg/kg) every 4 weeks in participants with hemophilia A with or without inhibitors against factor VIII (FVIII). The study consists of 2 parts: a pharmacokinetic (PK) run-in part followed by an expansion part.
Study Details
Timeline
Interventions
Emicizumab will be administered according to dose and schedule described in respective arms. After at least 24 weeks on prophylactic emicizumab, individuals who experienced suboptimal bleeding control on emicizumab (according to protocol-defined criteria) had the opportunity to increase their dose to 3 mg/kg weekly. Upon implementation of protocol Version 5 (20-Dec-2019), treatment duration was extended. During this study prolongation, participants had the opportunity to switch to a preferred emicizumab dosing regimen (1.5 mg/kg weekly, 3 mg/kg every 2 weeks, or 6 mg/kg every 4 weeks) in order to provide them the same flexibility as with commercial product.