A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors

• Status: Completed
• Trial end date: 2021-06-23
• Target enrollment: 0
• Participant gender: Male

Summary

The purpose of this study is to determine the frequency of bleeding episodes in patients receiving fitusiran as prophylactic treatment of hemophilia compared with patients who are assigned to continue with their regular medication. In addition, the study will assess safety, quality of life, pharmacodynamics (PD), and pharmacokinetics (PK).

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Genzyme, a Sanofi Company
• Treatments: Factor VIII

Criteria

Inclusion Criteria:

• Males, ≥12 years of age

• Severe hemophilia A or B with inhibitors

• (Severity confirmed by a central laboratory where FVIII level is <1% or FIX level is ≤2% at Screening; Inhibitors defined as inhibitor titer of ≥0.6 BU/mL or as evidenced by medical records)

• A minimum of 6 bleeding episodes requiring bypassing agent treatment within the last 6 months prior to Screening

• Willing and able to comply with the study requirements and to provide written informed consent and assent

Exclusion Criteria:

• Known co-existing bleeding disorders other than hemophilia A or B

• Antithrombin (AT) activity <60% at Screening

• Co-existing thrombophilic disorder

• Clinically significant liver disease

• Active HCV infection

• HIV positive with a CD4 count of <200 cells/μL

• History of arterial or venous thromboembolism

• Inadequate renal function

• History of multiple drug allergies or history of allergic reaction to an oligonucleotide or N-Acetylgalactosamine (GalNAc)

• History of intolerance to SC injection(s)

• Any other conditions or comorbidities that would make the patient unsuitable for enrollment or could interfere with participation in or completion of the study, per Investigator judgement