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New drug could help kids with hemophilia a bleed less

NCT ID NCT04759131

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 study tested a new drug called BIVV001 in 74 children under 12 with severe hemophilia A, a condition where blood doesn't clot properly. The drug is given through a vein and aims to prevent bleeding episodes. The trial found that the drug was safe and effective at reducing bleeds, with most children not developing antibodies against it.

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 to prevent bleeding episodes in young children with severe hemophilia A, potentially improving joint health and quality of life.
What could go wrong
This is a phase 3 trial with 74 participants, so results may not apply to all patients. There is a risk of developing antibodies that block the drug's effect, and long-term safety data are still limited.

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

74 people

The number who actually took part.

Started

Feb 2021

Finished

Jan 2023

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

Up 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 : * Participant must be younger than 12 years of age, at the time of signing the informed consent. * Severe hemophilia A defined as \<1 international units per deciliter (IU/dL) (\<1 percent \[%\]) endogenous Factor VIII (FVIII) 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 (EDs) for participants aged 6 to \<12 years and above 50 EDs for participants aged \<6 years. * Weight above or equal to 10 kg. Exclusion criteria: * History of hypersensitivity or anaphylaxis associated with any FVIII product. * History of a positive inhibitor (to FVIII) test defined as greater than or equal to (\>=) 0.6 Bethesda units (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 would not exclude the participant. * Positive inhibitor test result, defined as \>=0.6 BU/mL at 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.

Hemophilia A

Contacts and locations

Locations

  • Children's Healthcare of Atlanta-Site Number:8400019

    Atlanta, Georgia, 30322, United States

  • Children's Hospital Los Angeles-Site Number:8400006

    Los Angeles, California, 90027, United States

  • Children's Hospital Of Wisconsin-Site Number:8400005

    Milwaukee, Wisconsin, 53226-0509, United States

  • Childrens Hospital Of Columbus-Site Number:8400012

    Columbus, Ohio, 43205, United States

  • Cincinnati Children's Hospital Medical Center-Site Number:8400008

    Cincinnati, Ohio, 45206, United States

  • East Carolina University/Brody Medical Sciences Building-Site Number:8400015

    Greenville, North Carolina, 27834, United States

  • Investigational Site Number :0360001

    Westmead, New South Wales, 2145, Australia

  • Investigational Site Number :0360002

    South Brisbane, Queensland, 4101, Australia

  • Investigational Site Number :1240001

    Toronto, Ontario, M5G 1X8, Canada

  • Investigational Site Number :1240002

    Ottawa, Ontario, K1H 8L1, Canada

  • Investigational Site Number :1240003

    London, Ontario, N6A 4G5, Canada

  • Investigational Site Number :1240004

    Hamilton, Ontario, L8N 3Z5, Canada

  • Investigational Site Number :1580001

    Taichung, 402, 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 :2500001

    Le Kremlin-Bicêtre, 94275, France

  • Investigational Site Number :2500003

    Lille, 59037, France

  • Investigational Site Number :2500004

    Bron, 69500, France

  • Investigational Site Number :2760001

    Frankfurt am Main, 60590, Germany

  • Investigational Site Number :2760002

    München, 80337, Germany

  • 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, 80123, Italy

  • 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, WC1N3JH, United Kingdom

  • Investigational Site Number :8260003

    Birmingham, B4 6NH, United Kingdom

  • NY Presbyterian - Weill Cornell Medical Center-Site Number:8400020

    New York, New York, 10021, United States

  • Rush University Medical Center-Site Number:8400001

    Chicago, Illinois, 60612-3833, United States

  • University of Florida-Site Number:8400009

    Gainesville, Florida, 32610, United States

  • University of Iowa Hospitals and Clinics-Site Number:8400002

    Iowa City, Iowa, 52242, United States

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