Fitusiran prophylaxis in male pediatric subjects aged 1 to less than 12 years with hemophilia A or B

Phase 1

• Conditions: Hemophilia; MedDRA version: 20.0Level: LLTClassification code: 10066439Term: Hemophilia Class: 10010331; Therapeutic area: Diseases [C] - Hemic and Lymphatic Diseases [C15]

• Registration Number: CTIS2024-512501-76-00
• Lead Sponsor: Genzyme Corp.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-05-13
• Last Update Posted: 21 August 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 32

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

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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main 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 Objective: To characterize the safety and tolerability, To determine fitusiran plasma concentrations at selected time points;Primary end point(s): Plasma antithrombin (AT) activity levels

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s):Number of participants reported with adverse events;Secondary end point(s):Fitusiran plasma concentrations