NCT02847637

A Clinical Trial to Evaluate Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants Without Inhibitors

A Randomized, Multicenter, Open-Label, Phase III Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Patients Without Inhibitors

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 152 (actual)
• Sponsor: Hoffmann-La Roche · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

This is a randomized, global, multicenter, open-label, Phase 3 clinical study in participants with severe hemophilia A without inhibitors against Factor VIII (FVIII) who are 12 years or older. The study evaluates two prophylactic emicizumab regimens versus no prophylaxis in this population with emphasis on efficacy, safety, and pharmacokinetics.

Conditions

• Hemophilia A

Interventions

Type	Name	Description
DRUG	Emicizumab	Participants received emicizumab prophylaxis subcutaneously at the specified dose for each arm. 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 4 (20-Dec-2019), treatment duration was extended. During this study prolongation, each participant had the option to choose a preferred emicizumab dosing regimen among those permitted (i.e., emicizumab 1.5 mg/kg once every week \[QW\], 3 mg/kg once every 2 weeks \[Q2W\], or 6 mg/kg once every 4 weeks \[Q4W\]) and continue on that dosing regimen until discontinuation from the study.
DRUG	Factor VIII (FVIII)	FVIII was allowed to treat bleeds on an episodic basis, per the local prescribing information. Specific dosages of FVIII were not mandated in the study. Breakthrough bleeds were to be treated with the lowest FVIII dose expected to achieve hemostasis, which may have been lower than the participant's prior FVIII dose. To avoid bleeds before adequate emicizumab level is reached, patients in Arm D continued their regular FVIII prophylaxis until the second emicizumab loading dose. Concomitant routine FVIII prophylaxis was not permissible otherwise during the study.

Timeline

• Start date: 2016-09-27
• Primary completion: 2017-09-15
• Completion: 2022-05-12
• First posted: 2016-07-28
• Last updated: 2022-11-16
• Results posted: 2018-11-14

Locations

39 sites across 14 countries: United States, Australia, Costa Rica, France, Germany, Ireland, Italy, Japan, Poland, South Africa, South Korea, Spain, Taiwan, United Kingdom

Regulatory

• FDA-regulated drug study