Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A

Summary

Primary Objective: \- To evaluate the safety of BIVV001 in previously treated pediatric participants with hemophilia A. Secondary Objectives: * To evaluate the efficacy of BIVV001 as a prophylaxis treatment. * To evaluate the efficacy of BIVV001 in the treatment of bleeding episodes. * To evaluate BIVV001 consumption for prevention and treatment of bleeding episodes. * To evaluate the effect of BIVV001 prophylaxis on joint health outcomes. * To evaluate the effect of BIVV001 prophylaxis on Quality of Life (QoL) outcomes. * To evaluate the efficacy of BIVV001 for perioperative management. * To evaluate the safety and tolerability of BIVV001 treatment. * To assess the pharmacokinetics (PK) of BIVV001.

Timeline

Start

2021-02-19

Primary completion

2023-01-18

Completion

2023-01-18

Publications

• Malec L, Mathias M, Dunn AL, Nolan B, Wong N, Bystricka L, Khan U, Neill G, Santagostino E, Gunawardena S, Tarango C, Peyvandi F. Post hoc analysis of bleeding episodes and clinically relevant pharmacokinetic parameters among children <12 years old with severe hemophilia A receiving once-weekly efanesoctocog alfa prophylaxis in the XTEND-Kids phase 3 multinational trial. J Thromb Haemost. 2025 Nov;23(11):3481-3491. doi: 10.1016/j.jtha.2025.07.011. Epub 2025 Jul 21.
• Klamroth R, von Drygalski A, Hermans C, Park YS, Chan AKC, Kupesiz A, Alvarez-Roman MT, Malec L, Santagostino E, Neill G, Bystricka L, Dumont J, Abad-Franch L, Fetita LS, Khoo L. Perioperative Management With Efanesoctocog Alfa in Patients With Haemophilia A in the Phase 3 XTEND-1 and XTEND-Kids Studies. Haemophilia. 2025 May;31(3):391-400. doi: 10.1111/hae.70017. Epub 2025 Mar 18.
• Malec L, Peyvandi F, Chan AKC, Konigs C, Zulfikar B, Yuan H, Simpson M, Alvarez Roman MT, Carcao M, Staber JM, Dunn AL, Chou SC, d'Oiron R, Albisetti M, Demissie M, Santagostino E, Yarramaneni A, Wong N, Abad-Franch L, Gunawardena S, Fijnvandraat K; XTEND-Kids Trial Group. Efanesoctocog Alfa Prophylaxis for Children with Severe Hemophilia A. N Engl J Med. 2024 Jul 18;391(3):235-246. doi: 10.1056/NEJMoa2312611.

Drugs

Indications