A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B
Start Date
12/18/2025
Completion Date
12/30/2031
Summary
This is a parallel, Phase 3, two-arm, open-label study to evaluate the efficacy and safety of treatment with fitusiran prophylaxis administered to male pediatric participants (aged 1 to \<12 years) who have severe hemophilia A or B, with or without inhibitory antibodies to FVIII or FIX. Number of participants: Approximately 85 participants will be enrolled into the study: * Approximately 60 fitusiran-naïve participants with severe hemophilia A or B, with or without inhibitors (fitusiran-naïve arm), and * Approximately 25 participants with severe hemophilia A or B with inhibitors rolling over from the EFC15467\* dose confirmation study (roll-over arm). * Fitusiran has been investigated in the pediatric population in study EFC15467, which enrolled male participants aged 1 to \<12 years with hemophilia A or B with inhibitors to examine the safety and tolerability of fitusiran in the pediatric population. Participants will be enrolled into 1 of 2 arms: * Fitusiran-naïve: these participants have not previously received fitusiran, and they will undergo screening and study eligibility assessments. Once enrolled, they will go through a 24-week standard of care (SOC) period before starting fitusiran prophylaxis. * Roll-over participants from the EFC15467 study: only participants who are still on active treatment in study EFC15467 and consenting to study EFC17905 will be eligible to roll over. They will not need to undergo screening or further eligibility assessments. They will directly enroll into the fitusiran treatment period and continue treatment on their current fitusiran dose. The duration of fitusiran treatment will be up to 160 weeks for the fitusiran-naïve arm and up to 60 weeks for the roll-over arm.
Detailed Description
The duration of fitusiran treatment will be up to 160 weeks for the fitusiran-naïve arm and up to 60 weeks for the roll-over arm.
Eligibility Criteria
Age Range: 1 years to 11 years
Interventions
Fitusiran
Clotting factor concentrates (CFC) or bypassing agents (BPA)
Antithrombin concentrate (ATIIIC)
Conditions
Locations
The Luskin Orthopaedic Institute for Children- Site Number : 8400013
Los Angeles, California 90007
United States
The Center for Inherited Blood Disorders- Site Number : 8400009
Orange, California 92868
United States
Cure 4 The Kids Foundation- Site Number : 8400001
Las Vegas, Nevada 89147
United States
Hackensack Meridian Health - Hackensack University Medical Center- Site Number : 8400008
Hackensack, New Jersey 07601
United States
University of Texas - Southwestern Medical Center- Site Number : 8400015
Dallas, Texas 75390
United States
Investigational Site Number : 0560002
Brussels, 1020
Belgium
Investigational Site Number : 0560001
Brussels, 1200
Belgium
Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto- Site Number : 0760003
Ribeirão Preto, São Paulo 14051140
Brazil
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo- Site Number : 0760004
São Paulo, 05403-000
Brazil
Investigational Site Number : 1240001
Hamilton, Ontario L8N 3Z5
Canada
Investigational Site Number : 1240003
Montreal, Quebec H3T 1C5
Canada
Investigational Site Number : 1560001
Beijing, 100045
China
Investigational Site Number : 3560004
Bengaluru, 560034
India
Investigational Site Number : 3560002
Mumbai, 400022
India
Investigational Site Number : 3560001
Pune, 411004
India
Investigational Site Number : 3560003
Vellore, 632004
India
Investigational Site Number : 3800002
Florence, Firenze 50134
Italy
Investigational Site Number : 3800001
Milan, Milano 20122
Italy
Investigational Site Number : 3800003
Rozzano, Milano 20089
Italy
Investigational Site Number : 6160002
Wroclaw, Lower Silesian Voivodeship 50-556
Poland
Investigational Site Number : 6420001
Iași, 700309
Romania
Investigational Site Number : 6420003
Timișoara, 300011
Romania
Investigational Site Number : 6820001
Riyadh, 12746
Saudi Arabia
Investigational Site Number : 7240001
Esplugues de Llobregat, Barcelona [Barcelona] 08950
Spain
Investigational Site Number : 7240002
Madrid, 28046
Spain
Investigational Site Number : 7240003
Zaragoza, 50009
Spain
Investigational Site Number : 1580002
Taichung, 402
Taiwan
Investigational Site Number : 1580001
Taipei, 100
Taiwan
Investigational Site Number : 7920001
Istanbul, 34093
Turkey (Türkiye)
Investigational Site Number : 7920002
Izmir, 35100
Turkey (Türkiye)