A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors
Status:
Completed
Trial end date:
2021-07-14
Target enrollment:
Participant gender:
Summary
Primary Objective:
-To evaluate the efficacy of fitusiran compared to on-demand treatment with factor
concentrates, as determined by the frequency of bleeding episodes
Secondary Objectives:
- To evaluate the efficacy of fitusiran compared to on-demand treatment with factor
concentrates, as determined by:
- The frequency of spontaneous bleeding episodes
- The frequency of joint bleeding episodes
- Health-related quality of life (HRQOL) in patients ≥17 years of age
- To determine the frequency of bleeding episodes during the onset period
- To determine the safety and tolerability of fitusiran