NCT02554773

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

An Open-label Extension Study of Subcutaneously Administered Fitusiran in Patients With Moderate or Severe Hemophilia A or B Who Have Participated in a Previous Clinical Study With Fitusiran

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 34 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: Male
• Age: 18 Years
• Healthy volunteers: Not accepted

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

Detailed description

It is anticipated that patients in this study will receive treatment with open label fitusiran for approximately 7 years or until fitusiran becomes commercially available, whichever occurs first.

Conditions

• Hemophilia A
• Hemophilia B

Interventions

Type	Name	Description
DRUG	Fitusiran (SAR439774)	Pharmaceutical form: solution for injection Route of administration : subcutaneous (sc)

Timeline

• Start date: 2015-09-18
• Primary completion: 2023-03-21
• Completion: 2023-03-21
• First posted: 2015-09-18
• Last updated: 2024-06-05
• Results posted: 2024-06-05

Locations

12 sites across 5 countries: United States, Bulgaria, Russia, Switzerland, United Kingdom

Regulatory

• FDA-regulated drug study