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New shot could cut bleeds in kids with hemophilia

NCT ID NCT07285460

Disease control Sponsor: Sanofi Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Aug 18, 2026 · Updated 2 times

Summary

This phase 3 trial is testing a drug called fitusiran in about 85 boys aged 1 to under 12 with severe hemophilia A or B, including those with inhibitors that make standard treatments less effective. The drug is given as a regular shot under the skin to help prevent bleeding episodes. The study compares how many bleeds occur during fitusiran treatment versus standard care.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
fitusiran (a drug given as a shot under the skin to help prevent bleeding)
What this could lead to
If it works, this could give young boys with severe hemophilia a new option to prevent bleeds with fewer injections than current treatments.
What could go wrong
This is still being tested in a relatively small group, and fitusiran may not work for everyone. There are also risks like blood clots or liver problems that need careful monitoring.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 85 people

The number the study aims to enrol. It can still change while the study runs.

Started

Dec 2025

Expected to finish

Dec 2031

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

1 year to 11 years

Sex

Male participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

hemophilia hemophilia A severe hemophilia A severe hemophilia B

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Hemophilia

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    30 sites in 13 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Cure 4 The Kids Foundation- Site Number : 8400001

    RECRUITING

    Las Vegas, Nevada, 89147, United States

  • Department of pediatrics, Chung Shan University Hospital-Site Number : 1580002

    RECRUITING

    Taichung, 40201, Taiwan

  • Hackensack Meridian Health - Hackensack University Medical Center- Site Number : 8400008

    RECRUITING

    Hackensack, New Jersey, 07601, United States

  • Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo- Site Number : 0760004

    RECRUITING

    São Paulo, 05403-000, Brazil

  • Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto- Site Number : 0760003

    RECRUITING

    Ribeirão Preto, São Paulo, 14051140, Brazil

  • Investigational Site Number : 0560001

    RECRUITING

    Brussels, 1200, Belgium

  • Investigational Site Number : 0560002

    RECRUITING

    Brussels, 1020, Belgium

  • Investigational Site Number : 1240001

    RECRUITING

    Hamilton, Ontario, L8N 3Z5, Canada

  • Investigational Site Number : 1240003

    RECRUITING

    Montreal, Quebec, H3T 1C5, Canada

  • Investigational Site Number : 1560001

    RECRUITING

    Beijing, 100045, China

  • Investigational Site Number : 3560001

    RECRUITING

    Pune, 411004, India

  • Investigational Site Number : 3560002

    RECRUITING

    Mumbai, 400022, India

  • Investigational Site Number : 3560003

    RECRUITING

    Vellore, 632004, India

  • Investigational Site Number : 3560004

    RECRUITING

    Bengaluru, 560034, India

  • Investigational Site Number : 3800001

    RECRUITING

    Milan, Milano, 20122, Italy

  • Investigational Site Number : 3800002

    RECRUITING

    Florence, Firenze, 50134, Italy

  • Investigational Site Number : 3800003

    RECRUITING

    Rozzano, Milano, 20089, Italy

  • Investigational Site Number : 6820001

    RECRUITING

    Riyadh, 12746, Saudi Arabia

  • Investigational Site Number : 7240001

    RECRUITING

    Esplugues de Llobregat, Barcelona [Barcelona], 08950, Spain

  • Investigational Site Number : 7240002

    RECRUITING

    Madrid, 28046, Spain

  • Investigational Site Number : 7240003

    RECRUITING

    Zaragoza, 50009, Spain

  • Investigational Site Number : 7920001

    RECRUITING

    Istanbul, 34093, Turkey (Türkiye)

  • Investigational Site Number : 7920002

    RECRUITING

    Izmir, 35100, Turkey (Türkiye)

  • Klinika Transplantacji Szpiku, Onkologii i Hematologii Dziecięcej - Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wrocławiu-Site Number: 6160002

    RECRUITING

    Wroclaw, Lower Silesian Voivodeship, 50-556, Poland

  • Spitalul Clinic De Urgenta Pentru Copii Sfanta Maria Iasi, Department: St. Mary Children's Emergency Clinical Hospital-Site Number : 6420001

    RECRUITING

    Iași, 700309, Romania

  • Spitalul Clinic De Urgenta pentru Copii "Louis Turcanu"-Site Number : 6420003

    RECRUITING

    Timișoara, 300011, Romania

  • The Center for Inherited Blood Disorders- Site Number : 8400009

    RECRUITING

    Orange, California, 92868, United States

  • The Luskin Orthopaedic Institute for Children- Site Number : 8400013

    RECRUITING

    Los Angeles, California, 90007, United States

  • The division of Hematology, National Taiwan University Hospital-Site Number : 1580001

    RECRUITING

    Taipei City, Taipei, 100, Taiwan

  • University of Texas - Southwestern Medical Center- Site Number : 8400015

    RECRUITING

    Dallas, Texas, 75390, United States

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