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New shot could cut bleeding episodes in severe hemophilia

NCT ID NCT05662319

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 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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 3 trial tests fitusiran, an injectable drug, in 91 males aged 12 and older with severe hemophilia A or B, with or without inhibitors. Participants first receive standard care for 6 months, then switch to fitusiran for about 3 years. The goal is to see if fitusiran reduces the number of bleeding episodes that require treatment.

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 new preventive treatment that reduces bleeding episodes in people with severe hemophilia, potentially reducing the need for frequent clotting factor infusions.
What could go wrong
This is a single-arm, open-label study with no placebo group, so results may be less definitive. Fitusiran carries risks like blood clotting issues, and long-term safety is still being evaluated.

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

91 people

The number who actually took part.

Started

Feb 2023

Expected to finish

Jan 2029

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

12 years and older

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: * Diagnosis of severe congenital hemophilia A or B (FVIII \<1% or FIX level ≤2%) as evidenced by a central laboratory measurement at screening or documented medical record evidence. * For participants currently not on prophylaxis (CFC or BPA on-demand): A minimum of 4 bleeding episodes requiring BPA (inhibitor participants) or CFC (non-inhibitor participants) treatment within the last 6 months prior to screening. * Willing and able to comply with the study requirements and to provide written informed consent and assent in the case of participants under the age of legal consent, per local and national requirements Exclusion Criteria: * Known co-existing bleeding disorders other than congenital hemophilia A or B * History of arterial or venous thromboembolism, not associated with an indwelling venous access * History of intolerance to SC injection(s). * Current participation in immune tolerance induction therapy (ITI) * Prior gene therapy * Current or prior participation in a fitusiran trial * Current or prior participation in a gene therapy trial * Received an investigational drug or device within 30 days prior to the screening visit or within 5 half-lives of the investigational drug (or device) prior to the screening visit, whichever is longer * Presence of clinically significant liver disease AT activity \<60% at Screening * Co-existing thrombophilic disorder * Hepatitis C virus antibody positive, except participants who have negative Hepatitis C viral load and no evidence of cirrhosis * Presence of acute hepatitis, ie, hepatitis A, hepatitis E. * Presence of acute or chronic hepatitis B infection * Known to be HIV positive with CD4 count \<200 cells/μL. * Reduced renal function 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

Contacts and locations

Locations

  • Center for Inherited Blood Disorders (CIBD) Site Number : 8400012

    Orange, California, 92868-4306, United States

  • Children's Medical Center Dallas- Site Number : 8400018

    Dallas, Texas, 75235, United States

  • Gulf States Hemophilia and Thrombophilia Center- Site Number : 8400002

    Houston, Texas, 77030, United States

  • Hackensack University Site Number : 8400009

    Hackensack, New Jersey, 07601, United States

  • Investigational Site Number : 1240001

    Hamilton, Ontario, L8N 3Z5, Canada

  • Investigational Site Number : 1240002

    Hamilton, Ontario, L8N 3Z5, Canada

  • Investigational Site Number : 1240004

    Toronto, Ontario, M5G 1X8, Canada

  • Investigational Site Number : 1560001

    Guangzhou, 510515, China

  • Investigational Site Number : 1560002

    Jinan, 250013, China

  • Investigational Site Number : 1560003

    Beijing, 100045, China

  • Investigational Site Number : 1580001

    Taipei, 100, Taiwan

  • Investigational Site Number : 1580003

    Taipei, 11031, Taiwan

  • Investigational Site Number : 2500001

    Paris, 75015, France

  • Investigational Site Number : 2500002

    Lille, 59037, France

  • Investigational Site Number : 2500003

    Le Kremlin-Bicêtre, 94275, France

  • Investigational Site Number : 2760001

    Berlin, 10249, Germany

  • Investigational Site Number : 2760002

    Hamburg, 20246, Germany

  • Investigational Site Number : 3000001

    Athens, 115 27, Greece

  • Investigational Site Number : 3000002

    Athens, 11527, Greece

  • Investigational Site Number : 3560001

    Pune-411011, 411 011, India

  • Investigational Site Number : 3560003

    Vellore, 632004, India

  • Investigational Site Number : 3560004

    Bangalore, 560 034, India

  • Investigational Site Number : 3560006

    Punjab, 141008, India

  • Investigational Site Number : 3560007

    Bhubaneswar, 751019, India

  • Investigational Site Number : 3800001

    Milan, 20122, Italy

  • Investigational Site Number : 3800003

    Rozzano, Milano, 20089, Italy

  • Investigational Site Number : 3920001

    Kashihara-shi, Nara, 634-8522, Japan

  • Investigational Site Number : 3920002

    Saitama-shi, Saitama, 330-8777, Japan

  • Investigational Site Number : 3920003

    Nagoya, Aichi-ken, 4668560, Japan

  • Investigational Site Number : 4100001

    Seoul, Seoul-teukbyeolsi, 03722, South Korea

  • Investigational Site Number : 4100002

    Seoul, Seoul-teukbyeolsi, 05278, South Korea

  • Investigational Site Number : 4840001

    Veracruz, 91900, Mexico

  • Investigational Site Number : 4840002

    Monterrey, Nuevo León, 64460, Mexico

  • Investigational Site Number : 4840004

    Chihuahua City, 31000, Mexico

  • Investigational Site Number : 6160001

    Warsaw, Masovian Voivodeship, 02-776, Poland

  • Investigational Site Number : 6160002

    Krakow, Lesser Poland Voivodeship, 30-688, Poland

  • Investigational Site Number : 6160004

    Lodz, Lódzkie, 93-510, Poland

  • Investigational Site Number : 6820002

    Jeddah, 21423, Saudi Arabia

  • Investigational Site Number : 7100001

    Parktown, 2193, South Africa

  • Investigational Site Number : 7100003

    Johannesburg, 1501, South Africa

  • Investigational Site Number : 7240002

    A Coruña, A Coruña [La Coruña], 15006, Spain

  • Investigational Site Number : 7240003

    Zaragoza, 50009, Spain

  • Investigational Site Number : 7920001

    Çapa, 34390, Turkey (Türkiye)

  • Investigational Site Number : 7920002

    Adana, 01130, Turkey (Türkiye)

  • Investigational Site Number : 7920003

    Izmir, TR-35100, Turkey (Türkiye)

  • Investigational Site Number : 7920004

    Akdeniz, 07059, Turkey (Türkiye)

  • M Health Fairview University of Minnesota Medical Center - West Bank- Site Number : 8400016

    Minneapolis, Minnesota, 55454, United States

  • Northwell Health Hemostasis and Thrombosis Center Site Number : 8400015

    New Hyde Park, New York, 11040, United States

  • UPMC Children's Hospital of Pittsburgh-4401 Penn Ave Site Number : 8400017

    Pittsburgh, Pennsylvania, 15224-1334, United States

  • University Hospitals of Cleveland Site Number : 8400001

    Cleveland, Ohio, 44106, United States

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