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Fitusiran prophylaxis for boys aged 1 to under 12 with severe hemophilia A or B

An Open-label, Parallel, Phase 3, Two-arm 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 With or Without Inhibitory Antibodies to Factors VIII or IX

Phase 3 Interventional Sanofi · NCT07285460

This study tests whether fitusiran given as prophylaxis can reduce bleeding in boys aged 1 to under 12 with severe hemophilia A or B, with or without inhibitors.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment85 (estimated)
Ages1 Year to 11 Years
SexMale
SponsorSanofi Industry-sponsored
Drugs / interventionsemicizumab
Locations24 sites (Los Angeles, California and 23 other locations)
Trial IDNCT07285460 on ClinicalTrials.gov

What this trial studies

This open-label, Phase 3, parallel two-arm trial enrolls about 85 male pediatric participants aged 1 to <12 with severe hemophilia A or B, including those with inhibitors. Participants are either fitusiran-naïve or rolling over from a prior pediatric dose-confirmation study and will receive fitusiran prophylaxis, with treatment lasting up to 160 weeks for naïve participants and up to 60 weeks for roll-over participants. Concomitant clotting factor concentrates, bypassing agents, and antithrombin concentrate are permitted per protocol to manage bleeding and safety events. The study collects data on bleeding rates, safety, and tolerability to characterize fitusiran's effect in this pediatric population.

Who should consider this trial

Good fit: Boys aged 1 to <12 with severe hemophilia A (FVIII <1%) or B (FIX ≤2%), with or without inhibitors, who meet the study's medical and laboratory eligibility criteria.

Not a fit: Children with mild or moderate hemophilia, those older than 12, or those with medical contraindications to fitusiran or who cannot comply with long-term follow-up are unlikely to benefit from this study.

Why it matters

Potential benefit: If successful, fitusiran could substantially reduce bleeding frequency and the need for frequent factor or bypassing agent infusions in young boys with severe hemophilia.

How similar studies have performed: Fitusiran has reduced bleeding rates in adult trials and was previously studied in pediatrics (EFC15467) for safety and tolerability, though safety monitoring (including for thrombotic events) has been required.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

Participants not previously exposed to fitusiran are eligible to be included in the study only if all of the following criteria apply:

* Participant must be 1 to \<12 years of age at the time of enrollment.
* Participants must have severe hemophilia A or B (FVIII \<1% or FIX ≤2%) as evidenced by a central laboratory measurement at screening or documented medical record evidence.
* Participants must meet inhibitor or non-inhibitor status as defined below:

Inhibitor:

Requiring use of BPA for prophylaxis or BPA as on-demand therapy for any bleeding episodes for at least the last 3 months prior to screening, and 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 (eg, anaphylaxis) or nephrotic syndrome

Non-inhibitor:

Requiring use of clotting factor concentrates (CFCs) for prophylaxis or CFCs as on-demand therapy for any bleeding episodes for at least the last 3 months prior to screening, and meet each of the following criterion:

* Nijmegen-modified Bethesda assay inhibitor titer of \<0.6 BU/mL at screening, AND
* No use of BPA to treat bleeding episodes for at least the last 3 months prior to screening

  * Participants must have adequate peripheral venous access, as determined by the Investigator, to allow the blood draws required by the study protocol.
  * Male: There are no contraceptive requirements for this study except where required by local regulations.
  * Capable of giving signed informed consent/assent. A signed written informed consent must be obtained from parent(s)/legal guardian (hereafter referred to as the "parent"), as well as a written or oral assent obtained from participant, per local and national requirements.

Exclusion Criteria:

Participants not previously exposed to fitusiran are excluded from the study if any of the following criteria apply:

* Known co-existing bleeding disorders other than hemophilia A or B.
* Presence of clinically significant liver disease.
* History of antiphospholipid antibody syndrome.
* History of arterial or venous thromboembolism, unrelated to an indwelling venous access
* Any condition (eg, medical concern), which in the opinion of the Investigator, would make the participant unsuitable for dosing or which could interfere with the study compliance, the participant's safety and/or the participant's participation in the completion of the treatment period of the study.
* History of multiple drug allergies or history of allergic reaction to an oligonucleotide or GalNAc.
* Subjects with a central or peripheral indwelling catheter, with a history of venous access complications (such as infections, thrombosis) leading to hospitalization and/or systemic anticoagulation therapy in the last 12 months.
* At screening, anticipated need of surgery during the study or planned surgery scheduled to occur during the study.
* Completion of a surgical procedure within 14 days prior to screening, or currently receiving additional BPA infusion for postoperative hemostasis.
* History of intolerance to SC injection(s).
* Current participation in ITI therapy.
* The use of emicizumab (Hemlibra®) or any non-factor bleed management treatment within 6 months prior to screening
* Prior gene therapy
* Current or future participation in another clinical study, scheduled to occur during this study, involving an investigational product other than fitusiran or an investigational device.
* AT activity \<60% at screening, as determined by central laboratory analysis.
* Co-existing thrombophilic disorder.
* Presence of an active Hepatitis C virus infection
* Presence of acute hepatitis A or Hepatitis E virus infection.
* Presence of acute or chronic hepatitis B virus infection.
* Platelet count ≤100 000/μL.
* Presence of acute infection at screening.
* Human immunodeficiency virus (HIV) positive with a CD4 count of \<400 cells/μL.
* Estimated glomerular filtration rate ≤45 mL/min/1.73 m2 (using the Schwartz formula).

The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Where this trial is running

Los Angeles, California and 23 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Hemophilia
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.