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Monthly shot could free kids with hemophilia from frequent infusions

NCT ID NCT03974113

Disease control Sponsor: Genzyme, a Sanofi Company Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Sep 09, 2026 · Updated 2 times

Summary

This study tests fitusiran, a monthly injection, in boys aged 1 to under 12 with severe hemophilia A or B who have developed inhibitors (antibodies that block standard treatment). The goal is to find the right dose to safely lower a protein called antithrombin, which helps prevent bleeding episodes. Researchers will monitor side effects and drug levels in the blood.

What this could mean

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

Active substance
fitusiran
What this could lead to
If successful, fitusiran could offer a convenient, monthly injection to prevent bleeding episodes in young children with hemophilia who have developed inhibitors to standard treatments.
What could go wrong
This is an early-to-mid-stage trial with only 32 participants, so results may not apply to all children. Risks include potential side effects from lowering antithrombin levels, such as blood clots.

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

32 people

The number who actually took part.

Started

Jan 2020

Finished

Aug 2026

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: * 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: Participants are excluded from the study if any of the following criteria apply: * Known co-existing bleeding disorders other than hemophilia A or B * 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 * 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 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 hemophilia B severe hemophilia A severe hemophilia B

As listed by the trial registrant

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

Hemophilia

Contacts and locations

Locations

  • Children's Hospital Los Angeles Site Number : 8400002

    Los Angeles, California, 90027, United States

  • Hackensack University Medical Center- Site Number : 8400008

    Hackensack, New Jersey, 07601, United States

  • Investigational Site Number : 1240001

    Hamilton, Ontario, L8N 3Z5, Canada

  • Investigational Site Number : 1240002

    Ottawa, Ontario, K1H 8L1, Canada

  • Investigational Site Number : 3560001

    Pune-411011, 411 011, India

  • Investigational Site Number : 3560002

    Mumbai, 400 022, India

  • Investigational Site Number : 3560004

    Vellore, 632004, India

  • Investigational Site Number : 3560006

    Bangalore, 560034, India

  • Investigational Site Number : 3800001

    Milan, 20122, Italy

  • Investigational Site Number : 3800002

    Florence, 50134, Italy

  • Investigational Site Number : 7240002

    Madrid, Madrid, Comunidad de, 28046, Spain

  • Investigational Site Number : 7920001

    Adana, 01130, Turkey (Türkiye)

  • Investigational Site Number : 7920002

    Istanbul, 34093, Turkey (Türkiye)

  • Investigational Site Number : 7920003

    Izmir, TR-35100, Turkey (Türkiye)

  • Penn State Milton S. Hershey Medical Center- Site Number : 8400006

    Hershey, Pennsylvania, 17033, United States

  • University Hospitals of Cleveland- Site Number : 8400007

    Cleveland, Ohio, 44106, United States

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