A Study of Fitusiran in Severe Hemophilia A and B Patients Previously Receiving Factor or Bypassing Agent Prophylaxis

Summary

Primary Objective: To characterize the frequency of bleeding episodes (BE) while receiving fitusiran treatment, relative to the frequency of bleeding episodes while receiving factor concentrate or bypassing agent (BPA) prophylaxis. Secondary Objectives: * To characterize the following while receiving fitusiran treatment, relative to receiving factor or BPA prophylaxis: * the frequency of spontaneous bleeding episodes * the frequency of joint bleeding episodes * health related quality of life (HRQOL) in participants greater than or equal to (\>=) 17 years of age * To characterize the frequency of bleeding episodes during the onset and treatment periods in participants receiving fitusiran. * To characterize the safety and tolerability of fitusiran. * To characterize the annualized weight-adjusted consumption of factor/BPA while receiving fitusiran treatment, relative to receiving factor or BPA prophylaxis.

Timeline

Start

2018-07-25

Primary completion

2022-01-20

Completion

2022-03-25

Publications

• Kenet G, Nolan B, Zulfikar B, Antmen B, Kampmann P, Matsushita T, You CW, Vilchevska K, Bagot CN, Sharif A, Peyvandi F, Young G, Negrier C, Chi J, Kittner B, Sussebach C, Shammas F, Mei B, Andersson S, Kavakli K. Fitusiran prophylaxis in people with hemophilia A or B who switched from prior BPA/CFC prophylaxis: the ATLAS-PPX trial. Blood. 2024 May 30;143(22):2256-2269. doi: 10.1182/blood.2023021864.

Drugs

Indications