NCT03154437

An Expanded Access Program of Emicizumab in Participants With Hemophilia A With Inhibitors

An Open-Label, Multicenter, Expanded Access Program for Emicizumab in Patients With Hemophilia A With Inhibitors

• Status: Approved For Marketing
• Phase: —
• Study type: Expanded Access
• Enrollment: —
• Sponsor: Genentech, Inc. · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: —

Summary

This open-label, multicenter expanded access program (EAP) is designed to provide emicizumab to eligible participants with hemophilia A with factor VIII (FVIII) inhibitors before it is commercially available in the United States for the indication of hemophilia A with FVIII inhibitors. Discontinuation may occur earlier if participant or physician decides to discontinue treatment or the sponsor discontinues emicizumab clinical development.

Conditions

• Hemophilia A

Interventions

Type	Name	Description
BIOLOGICAL	Emicizumab	Participants will receive emicizumab at a loading dose of 3 milligrams per kilogram (mg/kg) per week subcutaneously (SC) for 4 weeks, followed by a maintenance dose of 1.5 mg/kg per week SC thereafter. Treatment with emicizumab will continue until unacceptable toxicity, withdrawal of consent, participant or physician decision to discontinue treatment, death, the participant is able to obtain commercial drug after emicizumab becomes commercially available, or the sponsor decides to discontinue emicizumab clinical development, whichever occurs first.

Timeline

• First posted: 2017-05-16
• Last updated: 2018-03-29

Locations

14 sites across 1 country: United States