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

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.

Timeline

Start

2016-09-27

Primary completion

2017-09-15

Completion

2022-05-12

Outcome

Met primary endpoint

registry analysis (superiority test); Arm C (Control): No Prophylaxis vs Arm A: Emicizumab 1.5 mg/kg QW; p <0.0001; ABR Ratio 0.04 (95% CI 0.020 to 0.075); NCT02847637 ↗

registry analysis (superiority test); Arm C (Control): No Prophylaxis vs Arm B: Emicizumab 3 mg/kg Q2W; p <0.0001; ABR Ratio 0.03 (95% CI 0.017 to 0.066); NCT02847637 ↗

release “HAVEN 3 study met primary endpoint and key secondary endpoints” gene.com ↗

release “Designation based on Phase III HAVEN 3 study demonstrating HEMLIBRA prophylaxis significantly reduced bleeds compared to no prophylaxis” gene.com ↗

release “Reduced Treated Bleeds by 96 Percent Compared to No Prophylaxis in Phase III Haven 3 Study in Hemophilia A Without Factor VIII Inhibitors” gene.com ↗

release “The Phase III HAVEN 3 study in people with hemophilia A without factor VIII inhibitors met its primary endpoint and key secondary endpoints.” gene.com ↗

release “HAVEN 3 study in people with haemophilia A without factor VIII inhibitors, Hemlibra prophylaxis led to statistically significant and clinically meaningful reductions in treated bleeds” roche.com ↗

Publications

• Kruse-Jarres R, Peyvandi F, Oldenburg J, Chang T, Chebon S, Doral MY, Croteau SE, Lambert T, Kempton CL, Pipe SW, Ko RH, Trzaskoma B, Dhalluin C, Bienz NS, Niggli M, Lehle M, Paz-Priel I, Young G, Jimenez-Yuste V. Surgical outcomes in people with hemophilia A taking emicizumab prophylaxis: experience from the HAVEN 1-4 studies. Blood Adv. 2022 Dec 27;6(24):6140-6150. doi: 10.1182/bloodadvances.2022007458.
• Kiialainen A, Niggli M, Kempton CL, Castaman G, Chang T, Paz-Priel I, Adamkewicz JI, Levy GG. Effect of emicizumab prophylaxis on bone and joint health markers in people with haemophilia A without factor VIII inhibitors in the HAVEN 3 study. Haemophilia. 2022 Nov;28(6):1033-1043. doi: 10.1111/hae.14642. Epub 2022 Jul 29.
• Mahlangu J, Oldenburg J, Paz-Priel I, Negrier C, Niggli M, Mancuso ME, Schmitt C, Jimenez-Yuste V, Kempton C, Dhalluin C, Callaghan MU, Bujan W, Shima M, Adamkewicz JI, Asikanius E, Levy GG, Kruse-Jarres R. Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors. N Engl J Med. 2018 Aug 30;379(9):811-822. doi: 10.1056/NEJMoa1803550.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Emicizumab	Monoclonal antibody	1.5 mg/kg	Subcutaneous
Subject	Emicizumab	Monoclonal antibody	3 mg/kg	Subcutaneous
Subject	Emicizumab	Monoclonal antibody	6 mg/kg	Subcutaneous
Background	Factor VIII	Protein / enzyme biologic	—	—

Indications