A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Participants From Birth to 12 Months of Age With Hemophilia A Without Inhibitors

Summary

This is a Phase IIIb, multicenter, open-label, single-arm study of prophylactic emicizumab in previously untreated and minimally treated patients at study enrollment from birth to ≤12 months of age with severe hemophilia A (intrinsic factor VIII \[FVIII\] level \<1%) without FVIII inhibitors. The study is designed to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of emicizumab administered at 3 milligrams per kilogram of body weight (mg/kg) once every 2 weeks (Q2W) for 52 weeks. After 1 year of treatment, participants will continue to receive emicizumab (1.5 mg/kg once every week \[QW\], 3 mg/kg Q2W or 6 mg/kg once every 4 weeks \[Q4W\]) over a 7-year long-term follow-up period under this study frame.

Timeline

Start

2021-02-04

Primary completion

2023-05-22

Completion

2030-05-18

Publications

• Pipe SW, Collins P, Dhalluin C, Kenet G, Schmitt C, Buri M, Jimenez-Yuste V, Peyvandi F, Young G, Oldenburg J, Mancuso ME, Kavakli K, Kiialainen A, Deb S, Niggli M, Chang T, Lehle M, Fijnvandraat K. Emicizumab prophylaxis in infants with hemophilia A (HAVEN 7): primary analysis of a phase 3b open-label trial. Blood. 2024 Apr 4;143(14):1355-1364. doi: 10.1182/blood.2023021832.
• Lopez-Jaime FJ, Benitez O, Diaz Jordan BL, Montano A, Coll J, Quintana Paris L, Gomez-Del Castillo Solano MDC. Expert opinion paper on the treatment of hemophilia a with emicizumab. Hematology. 2023 Dec;28(1):2166334. doi: 10.1080/16078454.2023.2166334.
• Hart DP. Commentary on "Development of a novel fully functional coagulation factor VIII with reduced immunogenicity utilizing an in silico prediction and deimmunization approach" - Will we ever be able to avoid inhibitor formation in hemophilia A? J Thromb Haemost. 2021 Sep;19(9):2125-2126. doi: 10.1111/jth.15404. No abstract available.

Drugs

Indications