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New clotting factor shows promise in reducing bleeds for hemophilia a patients

NCT ID NCT04161495

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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

Summary

This phase 3 study tested a new medicine called BIVV001 (efanesoctocog alfa) in 159 people aged 12 and older with severe hemophilia A. The goal was to see if regular injections could prevent bleeding episodes, improve joint health, and enhance quality of life. Participants received the drug intravenously, and researchers measured how many bleeds occurred per year.

What this could mean

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

Active substance
efanesoctocog alfa (BIVV001)
What this could lead to
If successful, this could provide a more effective and longer-lasting treatment option for people with severe hemophilia A, reducing the frequency of bleeding episodes and improving daily life.
What could go wrong
This is a phase 3 trial, but results may not apply to all patients. There is a risk of side effects like allergic reactions or development of inhibitors that block the treatment's effect.

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

159 people

The number who actually took part.

Started

Nov 2019

Finished

Feb 2022

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

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: * Participant, male or female, must be equal to or greater than 12 years of age inclusive, at the time of signing the informed consent. * Severe hemophilia A, defined as less than (\<) 1 international units per deciliter (IU/dL) (\<1 percent \[%\]) 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 exposure days. * Current regimen included one of the following: * Prophylactic treatment regimen with a FVIII product or prophylactic emicizumab therapy for at least 6 months during the previous 12 months. Appropriate washout time needs to be taken into account. * On-demand regimen with a FVIII product with a history of at least 12 bleeding episodes in the previous 12 months or at least 6 bleeding episodes in the previous 6 months prior to study enrollment. * On-demand participant was accepted to move to a prophylaxis treatment regimen after 26-week on-demand period. * Willingness and ability of the participant or surrogate (a caregiver or a family member greater than or equal to \[\>=\] 18 years of age) to complete training in the use of the study electronic Patient Diary (ePD) and to use the ePD throughout the study. * Ability of the participant or his or her legally authorized representative (eg., parent or legal guardian) to understand the purpose and risks of the study, willing and able to comply with study requirements and provide signed and dated informed consent or assent (as applicable) and authorization to use protected health information in accordance with national and local participant privacy regulations. Exclusion criteria: * Clinically significant liver disease. * Serious active bacterial 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. * Positive inhibitor results, defined as \>=0.6 Bethesda unit per milliliter (BU/mL) at screening. History of a positive inhibitor test defined as \>=0.6 BU/mL. Family history of inhibitors would not exclude the participant. * Use of Emicizumab within the 20 weeks prior to screening. * Major surgery within 8 weeks prior to screening. The above information was not intended to contain all considerations relevant to a participant'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.

Factor viii deficiency

Contacts and locations

Locations

  • Investigational Site Number 121

    Perth, Australia

  • Investigational Site Number 122

    Sydney, Australia

  • Investigational Site Number 136

    Buenos Aires, Argentina

  • Investigational Site Number 137

    Buenos Aires, Argentina

  • Investigational Site Number 139

    Mendoza, Argentina

  • Investigational Site Number 161

    Brussels, Belgium

  • Investigational Site Number 171

    Plovdiv, Bulgaria

  • Investigational Site Number 172

    Sofia, Bulgaria

  • Investigational Site Number 181

    Campinas, Brazil

  • Investigational Site Number 202

    Hamilton, Canada

  • Investigational Site Number 205

    Hamilton, Canada

  • Investigational Site Number 281

    Brest, France

  • Investigational Site Number 282

    Lyon, France

  • Investigational Site Number 283

    Lille, France

  • Investigational Site Number 284

    Marseille, France

  • Investigational Site Number 302

    Bonn, Germany

  • Investigational Site Number 303

    Frankfurt, Germany

  • Investigational Site Number 304

    Berlin, Germany

  • Investigational Site Number 312

    Budapest, Hungary

  • Investigational Site Number 314

    Debrecen, Hungary

  • Investigational Site Number 321

    Athens, Greece

  • Investigational Site Number 401

    Vicenza, Italy

  • Investigational Site Number 402

    Milan, Italy

  • Investigational Site Number 421

    Tokyo, Japan

  • Investigational Site Number 422

    Nara, Japan

  • Investigational Site Number 423

    Kitakyushu, Japan

  • Investigational Site Number 424

    Tokyo, Japan

  • Investigational Site Number 425

    Nagoya, Japan

  • Investigational Site Number 426

    Kawasaki, Japan

  • Investigational Site Number 435

    Durango, Mexico

  • Investigational Site Number 521

    Madrid, Spain

  • Investigational Site Number 531

    Chang-hua, Taiwan

  • Investigational Site Number 532

    Taipei, Taiwan

  • Investigational Site Number 581

    Basingstoke, United Kingdom

  • Investigational Site Number 581

    London, United Kingdom

  • Investigational Site Number 600

    Seoul, South Korea

  • Investigational Site Number 601

    Seoul, South Korea

  • Investigational Site Number 603

    Daegu, South Korea

  • Investigational Site Number 641

    Utrecht, Netherlands

  • Investigational Site Number 902

    Seattle, Washington, 98101-3932, United States

  • Investigational Site Number 906

    Las Vegas, Nevada, 89113, United States

  • Investigational Site Number 908

    Lansing, Michigan, 48824, United States

  • Investigational Site Number 911

    San Diego, California, 92121, United States

  • Investigational Site Number 917

    Gainesville, Florida, 32610, United States

  • Investigational Site Number 919

    Ann Arbor, Michigan, 48109, United States

  • Investigational Site Number 920

    Los Angeles, California, 90007-2664, United States

  • Investigational Site Number 921

    Los Angeles, California, 90027, United States

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