NCT03754790

Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX

An Open-label, Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 281 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: Male
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

Primary Objective: To characterize the long-term safety and tolerability of fitusiran Secondary Objectives: * To characterize the efficacy and long-term efficacy of fitusiran as assessed by the frequency of: * Bleeding episodes * Spontaneous bleeding episodes * Joint bleeding episodes * To characterize the effects of fitusiran on health-related quality of life (HRQOL) measures in participants ≥17 years of age

Detailed description

The estimated total time on the study for a participant is up to 90 months (including screening, treatment period and safety follow up period).

Conditions

• Hemophilia

Interventions

Type	Name	Description
DRUG	Fitusiran	Pharmaceutical form:solution for injection Route of administration: subcutaneous

Timeline

• Start date: 2019-01-09
• Primary completion: 2026-11-05
• Completion: 2026-11-05
• First posted: 2018-11-27
• Last updated: 2025-12-17

Locations

79 sites across 20 countries: United States, Australia, Canada, China, Denmark, France, Germany, Hungary, India, Ireland, Israel, Italy, Japan, Malaysia, South Africa, South Korea, Taiwan, Turkey (Türkiye), Ukraine, United Kingdom

Regulatory

• FDA-regulated drug study