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New drug shows promise in reducing bleeding for hemophilia patients

NCT ID NCT03754790

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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

Summary

This study looks at the long-term safety and effectiveness of a drug called fitusiran in people with hemophilia A or B, including those with inhibitors that make standard treatments less effective. The main goal is to see if fitusiran is safe over time and if it reduces bleeding episodes, including spontaneous and joint bleeds. Participants are males aged 12 and older who have already completed a Phase 3 fitusiran trial. The study does not aim to cure hemophilia but to control the disease by lowering bleeding risk.

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

281 people

The number who actually took part.

Started

Jan 2019

Expected to finish

Nov 2026

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 : * Participant must be at least 12 years of age inclusive, at the time of signing the informed consent * Participants with severe hemophilia A or B who have completed a Phase 3 fitusiran clinical trial * Male * Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol. In countries where legal age of majority is above 18 years, a specific ICF must also be signed by the participant's legally authorized representative Exclusion criteria: * Completion of a surgical procedure within 14 days prior to screening, or currently receiving additional factor concentrate or BPA infusion for postoperative hemostasis * Current participation in immune tolerance induction treatment (ITI) * Current use of factor concentrates or bypassing agents (BPAs) as regularly administered prophylaxis designed to prevent spontaneous bleeding episodes except for participants requiring factor concentrates or BPAs prophylaxis during the study dosing pause period * Use of compounds other than factor concentrates or BPAs for hemophilia treatment * Current or prior participation in a gene therapy trial * Alanine aminotransferase (ALT) and/or Aspartate aminotransferase (AST) \>1.5 × upper limit of normal reference range (ULN) for patients who are naïve to fitusiran at study start; ALT and/or AST \> 5 x ULN for patients who were in the fitusiran arm in the parent study * Additional exclusions for participants not currently participating in a fitusiran trial at the time of enrollment in the lower dose cohort: * Clinically significant liver disease * History of arterial or venous thromboembolism 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

As listed by the trial registrant

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

Hemophilia

Contacts and locations

Locations

  • Alliance for Childhood Diseases Site Number : 8400007

    Las Vegas, Nevada, 89135-3011, United States

  • Center for Inherited Blood Disorders (CIBD) Site Number : 8400016

    Orange, California, 92868, United States

  • Children's Hospital Los Angeles Site Number : 8400019

    Los Angeles, California, 90027, United States

  • Childrens Hospital Medical Center of Akron Site Number : 8400006

    Akron, Ohio, 44308, United States

  • Investigational Site Number : 0360001

    Camperdown, New South Wales, 2050, Australia

  • Investigational Site Number : 0360002

    Murdoch, Western Australia, 6961, Australia

  • Investigational Site Number : 0360003

    Prahran, Victoria, 3181, Australia

  • Investigational Site Number : 1240001

    Montreal, Quebec, H1T 2M4, Canada

  • Investigational Site Number : 1560002

    Guangzhou, 510515, China

  • Investigational Site Number : 1560003

    Shanghai, 200025, China

  • Investigational Site Number : 1560004

    Beijing, 100045, China

  • Investigational Site Number : 1560005

    Hangzhou, 89147, China

  • Investigational Site Number : 1560006

    Suzhou, 215006, China

  • Investigational Site Number : 1560007

    Beijing, 100730, China

  • Investigational Site Number : 1560008

    Jinan, 250013, China

  • Investigational Site Number : 1560009

    Chengdu, 610091, China

  • Investigational Site Number : 1560010

    Qingdao, 266555, China

  • Investigational Site Number : 1560011

    Guiyang, 550004, China

  • Investigational Site Number : 1560012

    Kunming, 650000, China

  • Investigational Site Number : 1560013

    Lanzhou, 730000, China

  • Investigational Site Number : 1560014

    Changsha, 410008, China

  • Investigational Site Number : 1580001

    Taipei, 110, Taiwan

  • Investigational Site Number : 1580002

    Changhua, 500, Taiwan

  • Investigational Site Number : 1580003

    Taipei, 100, Taiwan

  • Investigational Site Number : 1580004

    Taichung, 40705, Taiwan

  • Investigational Site Number : 1580005

    Taipei, 112, Taiwan

  • Investigational Site Number : 1580007

    Taoyuan City, 33305, Taiwan

  • Investigational Site Number : 1580008

    Taichung, 40201, Taiwan

  • Investigational Site Number : 2080001

    Copenhagen, 2100, Denmark

  • Investigational Site Number : 2500002

    Lyon, 69677, France

  • Investigational Site Number : 2500003

    Paris, 75015, France

  • Investigational Site Number : 2760001

    Berlin, 10249, Germany

  • Investigational Site Number : 2760003

    Leipzig, 04103, Germany

  • Investigational Site Number : 3480002

    Budapest, 1134, Hungary

  • Investigational Site Number : 3560001

    Bangalore, 560034, India

  • Investigational Site Number : 3560002

    Pune, 411001, India

  • Investigational Site Number : 3560004

    Ranipet, 632517, India

  • Investigational Site Number : 3560005

    Lucknow, 226003, India

  • Investigational Site Number : 3560007

    Mumbai, 400 022, India

  • Investigational Site Number : 3560010

    Pune, 411004, India

  • Investigational Site Number : 3720001

    Crumlin, Dublin, 12, Ireland

  • Investigational Site Number : 3720002

    Dublin, 8, Ireland

  • Investigational Site Number : 3760001

    Ramat Gan, 5262100, Israel

  • Investigational Site Number : 3800002

    Milan, Lombardy, 20122, Italy

  • Investigational Site Number : 3800004

    Padua, Veneto, 35128, Italy

  • Investigational Site Number : 3920001

    Nagoya, 4668560, Japan

  • Investigational Site Number : 3920002

    Nishinomiya, Hyōgo, 663-8501, Japan

  • Investigational Site Number : 3920003

    Kitakyushu, 8078555, Japan

  • Investigational Site Number : 3920004

    Saitama, 330-8777, Japan

  • Investigational Site Number : 3920005

    Isehara, Kanagawa, 2591193, Japan

  • Investigational Site Number : 3920008

    Tokyo, 1600023, Japan

  • Investigational Site Number : 4100001

    Busan, Busan, 602-739, South Korea

  • Investigational Site Number : 4100002

    Daejeon, Daejeon, 35233, South Korea

  • Investigational Site Number : 4100003

    Seoul, Seoul-teukbyeolsi, 03722, South Korea

  • Investigational Site Number : 4100004

    Seoul, 3722, South Korea

  • Investigational Site Number : 4580001

    Johor Bahru, 80100, Malaysia

  • Investigational Site Number : 4580002

    Kota Kinabalu, 88586, Malaysia

  • Investigational Site Number : 4580003

    Ampang, 68000, Malaysia

  • Investigational Site Number : 7100002

    Port Elizabeth, 6001, South Africa

  • 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)

  • Investigational Site Number : 7920005

    Istanbul, 34093, Turkey (Türkiye)

  • Investigational Site Number : 7920006

    Gaziantep, 27100, Turkey (Türkiye)

  • Investigational Site Number : 7920007

    Samsun, 55200, Turkey (Türkiye)

  • Investigational Site Number : 7920008

    Kayseri, 38039, Turkey (Türkiye)

  • Investigational Site Number : 7920012

    Bornova, 35100, Turkey (Türkiye)

  • Investigational Site Number : 8040001

    Kyiv, 04060, Ukraine

  • Investigational Site Number : 8040002

    Lviv, 79044, Ukraine

  • Investigational Site Number : 8040003

    Kyiv, 1135, Ukraine

  • Investigational Site Number : 8040004

    Mykolaiv, 54058, Ukraine

  • Investigational Site Number : 8260001

    London, London, City of, SE1 9RT, United Kingdom

  • Investigational Site Number : 8260004

    Glasgow, G4 0SF, United Kingdom

  • Nemours Children's Clinic Site Number : 8400008

    Jacksonville, Florida, 32207, United States

  • Phoenix Childrens Hospital Site Number : 8400009

    Phoenix, Arizona, 85016, United States

  • Rush University Medical Center -1725 W Harrison St Site Number : 8400001

    Chicago, Illinois, 60612-3863, United States

  • St Joseph's Children's Hospital of Tampa Site Number : 8400002

    Tampa, Florida, 33607, United States

  • University of Michigan Hospital - 1500 E Medical Center Dr Site Number : 8400012

    Ann Arbor, Michigan, 48109-5000, United States

  • ~Massachusetts General Hospital Site Number : 8400011

    Boston, Massachusetts, 02114, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.