A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors

Completed · Phase 3

Conditions studied: Hemophilia A, Hemophilia B

In brief

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 participants \>=17 years of age. * To determine the frequency of bleeding episodes during the onset period. * To determine the safety and tolerability of fitusiran.

Key facts

Study ID

NCT03417245

Run by

Genzyme, a Sanofi Company

People needed

120

Starts

2018-03-01

Expected to finish

2021-07-14

Last updated by the study team

2022-03-28

Who can join

Age: 12 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Males, >=12 years of age.
• Severe hemophilia A or B without inhibitors.
• Severity confirmed by a central laboratory where FVIII level was less than (<) 1 percent (%) or Factor IX (FIX) level was less than or equal to (<=) 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 Bethesda units per milliliter (BU/mL) at Screening.
• No use of Bypassing agents 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 complied with the study requirements and to provide written informed consent and assent.

You may not qualify if…

• 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 hepatitis C virus infection.
• HIV positive with a cluster of differentiation-4 count of <200 cells/microliter.
• 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 participant unsuitable for enrollment or could interfere with participation in or completion of the study, per Investigator judgment.

Where it is running

• Investigational Site Number 128 — Gainesville, Florida, United States
• Investigational Site Number 103 — Tampa, Florida, United States
• Investigational Site Number 102 — Chicago, Illinois, United States
• Investigational Site Number 119 — New Orleans, Louisiana, United States
• Investigational Site Number 125 — Ann Arbor, Michigan, United States
• Investigational Site Number 111 — Las Vegas, Nevada, United States
• Investigational Site Number 110 — Akron, Ohio, United States
• Investigational Site Number 6101 — Camperdown, Australia
• Investigational Site Number 6103 — Murdoch, Australia
• Investigational Site Number 6104 — Prahran, Australia
• Investigational Site Number 8604 — Beijing, China
• Investigational Site Number 8602 — Guangzhou, China
• Investigational Site Number 8605 — Hangzhou, China
• Investigational Site Number 8603 — Shanghai, China
• Investigational Site Number 8601 — Tianjin, China
• Investigational Site Number 4501 — Copenhagen, Denmark
• Investigational Site Number 3303 — Lyon, France
• Investigational Site Number 3305 — Paris, France
• Investigational Site Number 3301 — Rouen, France
• Investigational Site Number 4904 — Berlin, Germany
• Investigational Site Number 4905 — Frankfurt am Main, Germany
• Investigational Site Number 4906 — Leipzig, Germany
• Investigational Site Number 3602 — Budapest, Hungary
• Investigational Site Number 9102 — Bangalore, India
• Investigational Site Number 0140 — Little Rock, Arkansas, United States

Full record on ClinicalTrials.gov

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