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Clinical Trials

Emicizumab’s effectiveness, safety, and movement through the body (pharmacokinetics) were evaluated in phase 3 studies in people with hemophilia A. Learn more below about the data from these studies. 


Medical Information Letters

The Genentech resources below are patient-friendly summaries of data from clinical trials. These patient-friendly summaries include studies with the strongest and most relevant data and are provided only for educational purposes. You are encouraged to discuss any questions you have about your condition and any current or potential treatments with your heathcare provider.

HEMLIBRA Use in Hemophilia A Without Inhibitors

HEMLIBRA Use in Hemophilia A With Inhibitors

HEMLIBRA Use in Children Less Than 12 Years Old With Hemophilia A Without Inhibitors


Published Phase 3 clinical trials of emicizumab are provided below. Click to view abstracts and full publications, where available.

With Factor VIII Inhibitors (Adults and Young Adults): HAVEN 1 Study

Emicizumab Prophylaxis in Hemophilia A With Inhibitors.
Oldenburg J, Mahlangu JN, Kim B, et al. New Engl J Med.2017;377:809–818.

With Factor VIII Inhibitors (Children): HAVEN 2 Study

A Multicenter, Open-Label Phase 3 Study of Emicizumab Prophylaxis in Children With Hemophilia A With Inhibitors
Young G, Liesner R, Chang T, et al. Blood. 2019;134:2127–2138.

Without Factor VIII Inhibitors (Adults and Young Adults): HAVEN 3 Study

Emicizumab Prophylaxis in Patients Who Have Hemophilia A Without Inhibitors.
Mahlangu J, Oldenburg J, Pza-Priel I, et al. New Engl J Med. 2018;379:811-822.

With or Without Factor VIII Inhibitors (Adults and Young Adults): HAVEN 4 Study

Efficacy, Safety, and Pharmacokinetics of Emicizumab Prophylaxis Given Every 4 Weeks in People With Haemophilia A (HAVEN 4): A Multicentre, Open-Label, Non-Randomised Phase 3 Study.
Pipe SW, Shima M, Lehle M, et al. Lancet Haematol. 2019;6:e295–e305.

Without Factor VIII Inhibitors (Children): HOHOEMI Study

A Multicentre, Open-Label Study of Emicizumab Given Every 2 or 4 Weeks in Children With Severe Haemophilia A Without Inhibitors.
Shima M, Nogami K, Nagami S, et al. Haemophilia. 2019;25:979–987.