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Can a new clotting factor offer better bleed protection for severe hemophilia?

NCT ID NCT04644575

Disease control Sponsor: Bioverativ, 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jul 28, 2026 · Last updated Jul 29, 2026 · Updated 1 time

Summary

This phase 3 trial is investigating the long-term safety and effectiveness of an experimental clotting factor called BIVV001 in people with severe hemophilia A. The study aims to see if regular infusions of this therapy can prevent bleeding episodes, protect joint health, and improve quality of life. Participants will receive the treatment intravenously and be monitored for side effects, including the development of antibodies that could block the therapy.

What this could mean

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

Active substance
an experimental clotting factor called efanesoctocog alfa (BIVV001)
What this could lead to
If successful, this could offer a more effective and longer-lasting treatment option for people with severe hemophilia A, potentially reducing bleeding episodes and improving joint health.
What could go wrong
This is an early-stage trial focused on safety, so the treatment may not prove effective for everyone. There is a risk of developing antibodies that neutralize the therapy.

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

261 people

The number who actually took part.

Started

Feb 2021

Expected to finish

Jan 2027

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

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

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 : * For participants rolling over into Arm A * Participants who have completed the studies EFC16923, EFC16925, Arm B or Arm C of the current study, or any other potential BIVV001 study. * Male or Female * For participants new to BIVV001 (Arm B and C) * Participants who have severe hemophilia A, defined as \<1 IU/dL (\<1%) endogenous FVIII activity as documented either by central laboratory testing at screening or in historical medical records from a clinical laboratory demonstrating \<1% FVIII coagulant activity (FVIII:C) or a documented genotype known to produce severe hemophilia A. * Previous treatment for hemophilia A (prophylaxis or on-demand) with any recombinant and/or plasma-derived FVIII, or cryoprecipitate for at least 150 EDs or 50 EDs for participants aged \<6 years. * Platelet count ≥100 000 cells/μL at screening. * A participant known to be human immunodeficiency virus (HIV) antibody positive, either previously documented or identified from screening assessments, must have the following results prior to enrollment: CD4 lymphocyte count \>200 cells/mm³ and viral load of \<400 000 copies/mL * Male * Only for Arm B: Chinese participants * Only for Arm C: planned major surgery within 6 months after Day 1. Exclusion criteria: * For participants rolling over into Arm A * Positive inhibitor result, defined as ≥0.6 Bethesda units (BU)/mL. * Participation in another study. * For participants new to BIVV001 (Arm B and Arm C) * Any concurrent clinically significant liver disease that, in the opinion of the Investigator, would make the participant unsuitable for enrollment. This may include, but is not limited to cirrhosis, portal hypertension, and acute hepatitis. * Serious active bacterial, fungal, or viral infection (other than chronic hepatitis or HIV) present within 30 days of screening. * Other known coagulation disorder(s) in addition to hemophilia A. * History of hypersensitivity or anaphylaxis associated with any FVIII product. * History of a positive inhibitor (to FVIII) test defined as ≥0.6 BU/mL, or any value greater than or equal to the lower sensitivity cut-off for laboratories with cut-offs for inhibitor detection between 0.7 and 1.0 BU/mL, or clinical signs or symptoms of decreased response to FVIII administrations. Family history of inhibitors will not exclude the participant. * Positive inhibitor test (FVIII) result, defined as ≥0.6 BU/mL at screening. * Treatment with acetylsalicylic acid (ASA) or antiplatelet agents that are not nonsteroidal anti-inflammatory drugs (NSAIDs) within 2 weeks prior to screening. * Treatment with NSAIDs greater than the maximum dose specified in the regional prescribing information within 2 weeks prior to screening. * Systemic treatment within 12 weeks prior to Screening with chemotherapy and/or other immunosuppressive drugs (except for the treatment of hepatitis C virus \[HCV\] or HIV). * Emicizumab use within the 20 weeks prior to screening. * Major surgery within 8 weeks prior to screening. 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 A severe hemophilia A

As listed by the trial registrant

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

Hemophilia A

Contacts and locations

Locations

  • Bloodworks Northwest Site Number : 8400005

    Seattle, Washington, 98104, United States

  • Children's Healthcare of Atlanta Site Number : 8400016

    Atlanta, Georgia, 30322, United States

  • Children's Hospital Los Angeles Site Number : 8400009

    Los Angeles, California, 90027, United States

  • Children's Hospital Of Iowa Site Number : 8400011

    Iowa City, Iowa, 52242, United States

  • Children's Hospital of Wisconsin Site Number : 8400014

    Milwaukee, Wisconsin, 53226-0509, United States

  • Children's Research Institute Site Number : 8400013

    Columbus, Ohio, 43205-2696, United States

  • Cincinnati Children's Hospital Medical Center Site Number : 8400012

    Cincinnati, Ohio, 45229, United States

  • East Carolina University -2390 Hemby Ln Site Number : 8400015

    Greenville, North Carolina, 27834, United States

  • Hemocentro Campinas - UNICAMP Site Number : 0760001

    Campinas, São Paulo, 13083-970, Brazil

  • Hemostasis and Thrombosis Center of Nevada Site Number : 8400001

    Las Vegas, Nevada, 89113, United States

  • Investigational Site Number : 0320001

    CABA, Buenos Aires F.D., C1425BWE, Argentina

  • Investigational Site Number : 0320002

    Godoy Cruz, Mendoza Province, M5504FKD, Argentina

  • Investigational Site Number : 0320003

    Buenos Aires, C1015ABO, Argentina

  • Investigational Site Number : 0360001

    Westmead, New South Wales, 2145, Australia

  • Investigational Site Number : 0360002

    South Brisbane, Queensland, 4101, Australia

  • Investigational Site Number : 0360003

    Murdoch, Western Australia, 6961, Australia

  • Investigational Site Number : 0360004

    Camperdown, New South Wales, 2050, Australia

  • Investigational Site Number : 0560003

    Woluwe-Saint-Lambert, 1200, Belgium

  • Investigational Site Number : 1000171

    Plovdiv, 4002, Bulgaria

  • Investigational Site Number : 1000172

    Sofia, 1756, Bulgaria

  • Investigational Site Number : 1240001

    Toronto, Ontario, M5G 1X8, Canada

  • Investigational Site Number : 1240002

    Ottawa, Ontario, K1H 8L1, Canada

  • Investigational Site Number : 1240004

    Hamilton, Ontario, L8N 3Z5, Canada

  • Investigational Site Number : 1240005

    Hamilton, Ontario, L8L 8E7, Canada

  • Investigational Site Number : 1560001

    Guangzhou, 510515, China

  • Investigational Site Number : 1560002

    Beijing, 100045, China

  • Investigational Site Number : 1560003

    Hangzhou, 310003, China

  • Investigational Site Number : 1560004

    Hangzhou, 310003, China

  • Investigational Site Number : 1560005

    Jinan, 250013, China

  • Investigational Site Number : 1560006

    Beijing, 100730, China

  • Investigational Site Number : 1560007

    Suzhou, 215006, China

  • Investigational Site Number : 1560009

    Kunming, 650032, China

  • Investigational Site Number : 1560010

    Kunming, 650101, China

  • Investigational Site Number : 1560013

    Lanzhou, 730000, China

  • Investigational Site Number : 1580001

    Taichung, 40201, Taiwan

  • Investigational Site Number : 1580002

    Taipei, 10002, Taiwan

  • Investigational Site Number : 1580003

    Taichung, 407, Taiwan

  • Investigational Site Number : 1580004

    Taipei, 11031, Taiwan

  • Investigational Site Number : 1580005

    Changhua County, 500, Taiwan

  • Investigational Site Number : 2500001

    Le Kremlin-Bicêtre, 94275, France

  • Investigational Site Number : 2500003

    Lille, 59037, France

  • Investigational Site Number : 2500004

    Bron, 69500, France

  • Investigational Site Number : 2500005

    Brest, 29200, France

  • Investigational Site Number : 2500006

    Marseille, 13385, France

  • Investigational Site Number : 2760001

    Frankfurt am Main, 60590, Germany

  • Investigational Site Number : 2760002

    München, 80337, Germany

  • Investigational Site Number : 2760302

    Bonn, 53127, Germany

  • Investigational Site Number : 2760304

    Berlin, 10249, Germany

  • Investigational Site Number : 3000001

    Athens, 11527, Greece

  • Investigational Site Number : 3480002

    Budapest, 1134, Hungary

  • Investigational Site Number : 3480004

    Debrecen, 4093, Hungary

  • Investigational Site Number : 3480005

    Pécs, 7623, Hungary

  • Investigational Site Number : 3720001

    Dublin, D12 N512, Ireland

  • Investigational Site Number : 3800001

    Milan, 20121, Italy

  • Investigational Site Number : 3800002

    Naples, Campania, 80123, Italy

  • Investigational Site Number : 3800003

    Vicenza, 36100, Italy

  • Investigational Site Number : 3920421

    Shinjuku-ku, Tokyo, 160-0023, Japan

  • Investigational Site Number : 3920422

    Kashihara-Shi, Niigata, 634-8521, Japan

  • Investigational Site Number : 3920423

    Kitakyushu-shi, Fukuoka, 807-8556, Japan

  • Investigational Site Number : 3920424

    Suginami-ku, Tokyo, 167-0035, Japan

  • Investigational Site Number : 3920425

    Nagoya, Aichi-ken, 466-0065, Japan

  • Investigational Site Number : 3920426

    Kawasaki-shi, Kanagawa, 216-8511, Japan

  • Investigational Site Number : 4100600

    Seoul, Seoul-teukbyeolsi, 05278, South Korea

  • Investigational Site Number : 4100601

    Seoul, Seoul-teukbyeolsi, 03722, South Korea

  • Investigational Site Number : 4100603

    Daegu, Daegu, 41404, South Korea

  • Investigational Site Number : 5280001

    Utrecht, 3584 CX, Netherlands

  • Investigational Site Number : 5280002

    Amsterdam, 1105 AZ, Netherlands

  • Investigational Site Number : 7240001

    Madrid, Madrid, Comunidad de, 28046, Spain

  • Investigational Site Number : 7240002

    Esplugues de Llobregat, Catalunya [Cataluña], 08950, Spain

  • Investigational Site Number : 7520001

    Malmö, 20502, Sweden

  • Investigational Site Number : 7560001

    Zurich, 8032, Switzerland

  • Investigational Site Number : 7920001

    Istanbul, 34390, Turkey (Türkiye)

  • Investigational Site Number : 7920003

    Izmir, TR-35100, Turkey (Türkiye)

  • Investigational Site Number : 7920004

    Antalya, 07059, Turkey (Türkiye)

  • Investigational Site Number : 8260001

    London, London, City of, WC1N 3JH, United Kingdom

  • Investigational Site Number : 8260003

    Birmingham, B4 6NH, United Kingdom

  • Investigational Site Number : 8260004

    Hampshire, RG24 9NA, United Kingdom

  • Investigational Site Number : 8260005

    London, London, City of, NW3 2QG, United Kingdom

  • Michigan State University School Of Med Site Number : 8400002

    East Lansing, Michigan, 48824, United States

  • New York Presbyterian Hospital/Weill Cornell Medical Center Site Number : 8400017

    New York, New York, 10021, United States

  • Orthopaedic Institute for Children Site Number : 8400003

    Los Angeles, California, 90007, United States

  • Rush University Medical Center Site Number : 8400010

    Chicago, Illinois, 60612-3833, United States

  • University of California San Diego Site Number : 8400007

    San Diego, California, 92121, United States

  • University of Florida Health Site Number : 8400008

    Gainesville, Florida, 32608, United States

  • University of Michigan Medical Center Site Number : 8400006

    Ann Arbor, Michigan, 48109, United States

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