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: 0
• Participant gender: Male

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

• 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 without inhibitors

• Severity confirmed by a central laboratory where FVIII level is <1% or FIX level is ≤2% at Screening;

• On-demand use of factor concentrate to manage bleeding episodes for at least the last 6 months prior to Screening, and meet each of the following criterion:

• Nijmegen modified Bethesda assay inhibitor titer of <0.6 BU/mL at Screening

• No use of BPAs to treat bleeding episodes for at least the last 6 months prior to Screening

• No history of immune tolerance induction therapy within the last 3 years prior to Screening

• A minimum of 6 bleeding episodes requiring factor concentrate 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, i.e. Von Willebrand's disease, additional factor deficiencies, or platelet disorders

• 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 judgment