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New hope for babies with severe hemophilia: Once-a-Week shot may prevent bleeding

NCT ID NCT04431726

Ongoing Disease control Sponsor: Hoffmann-La Roche Source: ClinicalTrials.gov ↗

First seen Nov 01, 2025 · Last updated Jun 23, 2026 · Updated 32 times

Summary

This study is testing a medicine called emicizumab (Hemlibra) in babies from birth to 12 months old with severe hemophilia A. The goal is to see if a weekly or biweekly shot under the skin can prevent bleeding episodes and protect joints. About 55 infants will receive the drug for a year, with long-term follow-up for up to 7 years. The study measures how many bleeds occur and checks for side effects.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Locations

  • AORN Santobono Pausilipon

    Naples, Campania, 80123, Italy

  • AOU Careggi

    Florence, Tuscany, 50134, Italy

  • AOU di Parma

    Parma, Emilia-Romagna, 43126, Italy

  • Adana Acibadem Hospital

    Adana, 01130, Turkey (Türkiye)

  • Charlotte Maxeke Johannesburg Hospital

    Johannesburg, 2193, South Africa

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • Children's Hospital of Eastern Ontario

    Ottawa, Ontario, K1H 8L1, Canada

  • Cliniques Universitaires St-Luc

    Brussels, 1200, Belgium

  • Ege University, School of Medicine

    Izmir, 35100, Turkey (Türkiye)

  • Groupe Hospitalier Necker Enfants Malades

    Paris, 75015, France

  • Hacettepe University Medical Faculty

    Ankara, 06100, Turkey (Türkiye)

  • Hospital Sant Joan de Deu

    Esplugues de Llobregat, Barcelona, 08950, Spain

  • Hospital Universitario Virgen del Rocio

    Seville, 41013, Spain

  • Hospital Universitario la Paz

    Madrid, 28046, Spain

  • Hospital das Clínicas Faculdades Médicas de Ribeirão Preto

    Ribeirão Preto, São Paulo, 14051-140, Brazil

  • Hämophilie-Zentrum Rhein Main GmbH

    Mörfelden-Walldorf, 64546, Germany

  • IRCCS Ca' Granda Ospedale Maggiore Policlinico

    Milan, Lombardy, 20122, Italy

  • Medizinische Universität Wien

    Vienna, 1090, Austria

  • Ondokuz Mayis Univ. Med. Fac.

    Samsun, 55139, Turkey (Türkiye)

  • Perth Children's Hospital

    Nedlands, Western Australia, 6009, Australia

  • Phoenix Children's Hospital

    Phoenix, Arizona, 85016, United States

  • Queen Elizabeth University Hospital

    Glasgow, G51 4TF, United Kingdom

  • Royal Children's Hospital

    Parkville, Victoria, 3052, Australia

  • Seattle Children's Hospital

    Seattle, Washington, 98105, United States

  • Sheba Medical Center - National Hemophilia Center

    Tel Litwinsky, 5262100, Israel

  • The Children's Hospital at Westmead

    Westmead, New South Wales, 2145, Australia

  • The Hospital for Sick Children

    Toronto, Ontario, M5G 1X8, Canada

  • Tulane University Health Sciences Center

    New Orleans, Louisiana, 70112, United States

  • UZ Leuven Gasthuisberg

    Leuven, 3000, Belgium

  • University of Colorado Denver, Children's Hospital

    Aurora, Colorado, 80045, United States

  • University of Michigan

    Ann Arbor, Michigan, 48109-0934, United States

  • Universitätsklinikum Bonn

    Bonn, 53127, Germany

What this could mean

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

Active substance

emicizumab (Hemlibra)

What this could lead to

If successful, this could provide a safe and effective way to prevent bleeding in very young children with severe hemophilia A, reducing the need for frequent IV treatments and protecting joints from damage.

What could go wrong

This is a single-arm, open-label study with no comparison group, so results may be less definitive. Long-term safety and effectiveness in infants are still being studied, and there is a risk of side effects like injection site reactions or blood clots.

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.

SEVERE HEMOPHILIA A