At a glance
ClinicalIndex Comparison Record- ✓Age 12 years or older at informed consent
- ✓Confirmed hemophilia A diagnosis with persistent inhibitors against FVIII
- ✓Documented prior treatment with bypassing agents or FVIII concentrates within last 6 months (on-demand or prophylaxis)
- ✓Adequate hematologic, hepatic, and renal function
- ✕Bleeding disorder other than hemophilia A
- ✕Current or planned immune tolerance induction (ITI) therapy; must discontinue bypassing agents and FVIII prophylaxis prior to enrollment, except patients completing ITI may enroll after 72-hour washout
- ✕High risk for thrombotic microangiopathy (TMA) or history of TMA
- ✕Previous or current treatment for thromboembolic disease in past 12 months (except resolved catheter-associated thrombosis) or current signs of thromboembolism
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
A Single-Arm, Multicenter Phase IIIB Clinical Trial to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors
In Brief
A Phase 3 clinical trial evaluating Emicizumab for Hemophilia A. Completed, enrolled 195 participants across 72 sites in 24 countries.
Detailed Summary
This is a phase IIIb, single arm, open-label, multi-center study to evaluate the safety and tolerability of emicizumab in participants with congenital hemophilia A who have documented inhibitors against Factor VIII (FVIII) at enrollment. Approximately 200 participants, aged 12 or older, will be enrolled in this study and are expected to be enrolled at approximately 85 sites globally. Participants will receive an initial weekly dose of prophylactic emicizumab subcutaneously for 4 weeks, followed by a weekly maintenance dose subcutaneously for the remainder of the 2-year treatment period.
Study Details
Timeline
Interventions
Initial dosing will be 3 mg/kg/week subcutaneously for 4 weeks; Maintenance dosing will follow at 1.5 mg/kg/week subcutaneously for the remainder of the 2-year treatment period