Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B

Running, not enrolling · Phase 2/Phase 3

Conditions studied: Hemophilia

In brief

Primary Objective: \- To confirm appropriate dose levels of fitusiran when administered to male pediatric participants (ages 1 to \<12 years of age) with severe hemophilia A or B Secondary Objectives: * To characterize the safety and tolerability * To determine fitusiran plasma concentrations at selected time points

Key facts

Study ID

NCT03974113

Run by

Genzyme, a Sanofi Company

People needed

32

Starts

2020-01-28

Expected to finish

2026-12-15

Last updated by the study team

2026-06-22

Who can join

Age: 1 and older, up to 11. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Male, aged 1 to <12 years at the time of enrollment.
• Severe hemophilia A or B (Factor VIII (FVIII) <1% or Factor IX (FIX) ≤2%)
• Participants must have inhibitory antibodies to FVIII or FIX and must meet one of the following Nijmegen-modified Bethesda assay results criteria:
• Inhibitor titer of ≥0.6 BU/mL at screening, OR
• Inhibitor titer of <0.6 BU/mL at screening with medical record evidence of 2 consecutive titers ≥0.6 BU/mL, OR
• Inhibitor titer of <0.6 BU/mL at screening with medical record evidence of 1 inhibitor titer ≥0.6 BU/mL and a history of anamnestic response or severe allergic reaction (anaphylaxis or nephrotic syndrome)
• Adequate peripheral venous access, as determined by the Investigator, to allow the blood draws required by the study protocol
• Weight requirements at the time of enrollment: 8 to <45 kg
• Willing and able to comply with the study requirements and to provide signed written informed consent obtained from parent(s)/legal guardian (hereinafter the "parent") and written or oral assent obtained from participant, per local and national requirements

You may not qualify if…

• Participants are excluded from the study if any of the following criteria apply:
• 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 Hepatitis C virus infection
• Acute or chronic Hepatitis B virus infection
• Acute Hepatitis A or hepatitis E infection
• HIV positive with a CD4 count of <400 cells/μL
• History of arterial or venous thromboembolism, unrelated to an indwelling venous access
• Inadequate renal function
• History of multiple drug allergies or history of allergic reaction to an oligonucleotide or N-Acetylgalactosamine (GalNAc)
• Subjects with central or peripheral indwelling catheters, with history of venous access complications leading to hospitalization and/or systemic anticoagulation therapy.
• History of intolerance to subcutaneous (SC) injection(s)
• Use of emicizumab (Hemlibra®) within 6 months prior to screening
• 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
• The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Where it is running

• Children's Hospital Los Angeles Site Number : 8400002 — Los Angeles, California, United States
• Hackensack University Medical Center- Site Number : 8400008 — Hackensack, New Jersey, United States
• University Hospitals of Cleveland- Site Number : 8400007 — Cleveland, Ohio, United States
• Penn State Milton S. Hershey Medical Center- Site Number : 8400006 — Hershey, Pennsylvania, United States
• Investigational Site Number : 1240001 — Hamilton, Ontario, Canada
• Investigational Site Number : 1240002 — Ottawa, Ontario, Canada
• Investigational Site Number : 3560006 — Bangalore, India
• Investigational Site Number : 3560002 — Mumbai, India
• Investigational Site Number : 3560001 — Pune-411011, India
• Investigational Site Number : 3560004 — Vellore, India
• Investigational Site Number : 3800002 — Florence, Italy
• Investigational Site Number : 3800001 — Milan, Italy
• Investigational Site Number : 7240002 — Madrid, Madrid, Comunidad de, Spain
• Investigational Site Number : 7920001 — Adana, Turkey (Türkiye)
• Investigational Site Number : 7920002 — Istanbul, Turkey (Türkiye)
• Investigational Site Number : 7920003 — Izmir, Turkey (Türkiye)

Full record on ClinicalTrials.gov

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