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

NCT ID NCT04431726

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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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

55 people

The number who actually took part.

Started

Feb 2021

Expected to finish

May 2030

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

0 to 12 months

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: * Age from birth to ≤12 months at time of informed consent * Body weight ≥3 kilograms (kg) at time of informed consent. Patients with a lower body weight can be enrolled after they have reached a body weight of 3 kg. Premature babies (gestational age \<38 weeks) may be enrolled as long as they have reached a body weight of 3 kg. For premature babies, the corrected gestational age should be reported. * Mandatory receipt of vitamin K prophylaxis according to local standard practice * Diagnosis of severe congenital hemophilia A (intrinsic FVIII level \<1%) * A negative test for FVIII inhibitor (i.e., \<0.6 Bethesda units \[BU\]/mL) locally assessed during the 2-week screening period * No history of documented FVIII inhibitor (i.e., \<0.6 BU/mL), FVIII drug-elimination half-life \<6 hours, or FVIII recovery \<66% * Previously untreated patients or minimally treated patients (i.e., up to 5 days of exposure with hemophilia-related treatments, such as plasma-derived FVIII, recombinant FVIII, fresh frozen plasma, cryoprecipitate, or whole blood products) * Documentation of the details of the hemophilia-related treatments received since birth * Documentation of the details of the bleeding episodes since birth * For patients from birth to \<3 months of age at the time of study entry: no evidence of active intracranial hemorrhage, as confirmed by a negative cranial ultrasound at screening irrespective of delivery mode * Adequate hematologic, hepatic, and renal function, as defined in the protocol * For parents/caregivers: willingness and ability to comply with the study protocol requirements, scheduled visits, treatment plans, laboratory tests, completion of applicable questionnaires, and other study procedures Exclusion Criteria: * Inherited or acquired bleeding disorder other than severe hemophilia A * Use of systemic immunomodulators (e.g., interferon) at enrollment or planned use during the study * Receipt of any of the following: Prior use of emicizumab prophylaxis including investigational or commercial emicizumab; An investigational drug to treat or reduce the risk of hemophilic bleeds within 5 drug-elimination half-lives of last drug administration; A non-hemophilia-related investigational drug within the last 30 days or 5 drug-elimination half-lives, whichever is shorter; An investigational drug concurrently * Current active severe bleed, such as intracranial hemorrhage * Planned surgery (excluding minor procedures, e.g., circumcision, CVAD placement) during the study * History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection * Patients who are at high risk for thrombotic microangiopathy (TMA) (e.g., have a previous medical or family history of TMA, such as thrombotic thrombocytopenic purpura, atypical hemolytic uremic syndrome) in the investigator's judgment * Previous or current treatment for thromboembolic disease (with the exception of previous catheter-associated thrombosis in patients for whom anti-thrombotic treatment is not currently ongoing) or signs of thromboembolic disease * Any hereditary or acquired maternal condition that may predispose the patient to thrombotic events (e.g., inherited thrombophilias antiphospholipid syndrome) * Other diseases (e.g., certain autoimmune diseases) that may increase risk of bleeding or thrombosis * Known infection with HIV, hepatitis B virus, or hepatitis C virus * Serious infection requiring antibiotics or antiviral treatments within 14 days prior to screening * Concurrent disease, treatment, abnormality in clinical laboratory tests, vital signs measurements, or physical examination findings that could interfere with the conduct of the study or that would, in the opinion of the investigator or Sponsor, preclude the patient's safe participation in and completion of the study or interpretation of the study results * Unwillingness of the parent or caregiver to allow receipt of blood or blood products, or any standard-of-care treatment for a life-threatening condition * Any other medical, social, or other condition that may prevent adequate compliance with the study protocol in the opinion of the investigator

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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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