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

Part of paid clinical trials in Los Angeles, California.

Sponsor
Sanofi
Study ID
NCT07285460
Phase
PHASE3
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
MALE
Age
1 Year - 11 Years
Healthy Volunteers
Not accepted

Interventions

  • Fitusiran — DRUG
    Pharmaceutical form: solution for injection in PBS Route of administration: subcutaneous
  • Clotting factor concentrates (CFC) or bypassing agents (BPA) — BIOLOGICAL
    Pharmaceutical form: solution for injection Route of administration: intravenous injection
  • Antithrombin concentrate (ATIIIC) — BIOLOGICAL
    Pharmaceutical form: solution for injection Route of administration: intravenous injection

Study Details

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.

Key Dates

Start date
Dec 18, 2025
Status verified
May 2026
Primary completion
Aug 4, 2029
Completion
Dec 30, 2031

Study Design

Enrollment
85 participants (estimated)
Allocation
NA
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Fitusiran-naïve arm
    Participants will go through a 24-week standard of care (SOC) period before receiving a selected dose of fitusiran at regular interval. If a fitusiran dose adjustment is needed during the study, participants will follow a specific dosing regimen as per study protocol.
  • Experimental: EFC15647 roll-over arm
    Participants will continue receiving their current fitusiran dose from EFC15467. If a fitusiran dose adjustment is needed during the study, participants will follow a specific dosing regimen as per study protocol.

Primary Outcome Measure

Annualized treated bleeding rate (ABR) in the fitusiran primary efficacy period and in the SOC period [ Time Frame: Day 85 to Day 421 (fitusiran primary efficacy period); Day -168 to Day -1 (SOC period) ]

Central Contacts

  • Trial Transparency email recommended (Toll free for US & Canada)
    800-633-1610
    [email protected]

Locations (4)

FacilityCityStateZIPSite coordinators
The Luskin Orthopaedic Institute for Children- Site Number : 8400013Los AngelesCalifornia90007-
The Center for Inherited Blood Disorders- Site Number : 8400009OrangeCalifornia92868-
Cure 4 The Kids Foundation- Site Number : 8400001Las VegasNevada89147-
Hackensack Meridian Health - Hackensack University Medical Center- Site Number : 8400008HackensackNew Jersey07601-

Find similar trials in Los Angeles, CA

By research site
The Luskin Orthopaedic Institute for Children· Los Angeles, CAThe Center for Inherited Blood Disorders· Orange, CACure 4 The Kids Foundation· Las Vegas, NVHackensack Meridian Health - Hackensack University Medical Center· Hackensack, NJ

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