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 Jun 27, 2026 · Last updated Jul 08, 2026 · Updated 2 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.

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.

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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.

SEVERE HEMOPHILIA A

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

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