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

NCT ID: NCT03974113

Last Updated: 2025-08-08

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE3
• Total Enrollment: 32 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-01-28
• Study Completion Date: 2026-12-15

Brief Summary

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

Detailed Description

The estimated total time on study is up to 256 weeks for participants who roll over into the extension study and up to 280 weeks for participants who do not roll over into the extension study (due to the requirement for up to an additional 6 months of follow-up for monitoring of AT levels).

Conditions

Hemophilia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Fitusiran

Participants will receive a selected dose of fitusiran at regular intervals, as per study protocol

Group Type: EXPERIMENTAL

Fitusiran

Intervention Type: DRUG

Pharmaceutical form:Solution for injection-Route of administration:Subcutaneous

Interventions

Fitusiran

Pharmaceutical form:Solution for injection-Route of administration:Subcutaneous

Intervention Type: DRUG

Other Intervention Names

SAR439774

Eligibility Criteria

Inclusion Criteria

• 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

Exclusion Criteria

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.

• Minimum Eligible Age: 1 Year
• Maximum Eligible Age: 11 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Genzyme, a Sanofi Company

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Clinical Sciences & Operations

Role: STUDY_DIRECTOR

Sanofi

Locations

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)

Countries

United States; Canada; India; Italy; Spain; Turkey (Türkiye)

Other Identifiers

U1111-1223-4368

Identifier Type: REGISTRY

Identifier Source: secondary_id

2024-512501-76

Identifier Type: REGISTRY

Identifier Source: secondary_id

2019-000679-18

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

EFC15467

Identifier Type: -

Identifier Source: org_study_id