Overview

An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B

Status:
Active, not recruiting

Trial end date:
2023-10-20

Target enrollment:

Participant gender:

Summary

Primary Objective: To evaluate the long-term safety and tolerability of fitusiran in male patients with moderate or severe hemophilia A or B Secondary Objectives: - To investigate the long-term efficacy of fitusiran - To characterize the safety and efficacy of concomitantly administered Factor VIII (FVIII), Factor IX (FIX) or bypassing agents (BPA) and fitusiran for treatment of bleeding episodes - To assess changes in health-related quality of life (QOL) over time - To characterize antithrombin (AT) reduction and thrombin generation (TG) increase - To characterize the pharmacokinetics (PK) of fitusiran

Phase:

Phase 1/Phase 2

Details

Lead Sponsor:

Alnylam Pharmaceuticals
Genzyme, a Sanofi Company

Treatments:

Factor VIII