NCT03191799

A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors

A Single-Arm, Multicenter Phase IIIB Clinical Trial to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 195 (actual)
• Sponsor: Hoffmann-La Roche · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Hemophilia A

Interventions

Type	Name	Description
DRUG	Emicizumab	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

Timeline

• Start date: 2017-09-05
• Primary completion: 2020-11-19
• Completion: 2020-11-19
• First posted: 2017-06-19
• Last updated: 2021-06-11
• Results posted: 2021-06-11

Locations

72 sites across 24 countries: Australia, Belgium, Brazil, Canada, Colombia, Finland, Germany, Guatemala, Hungary, India, Israel, Italy, Mexico, Netherlands, Panama, Poland, Portugal, Romania, Russia, Saudi Arabia, Spain, Sweden, Switzerland, United Kingdom