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Study on the Effects and Safety of Emicizumab for Infants with Hemophilia A (Ages Birth to 12 Months)

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying Hemophilia A, a genetic disorder that affects the blood's ability to clot properly, leading to excessive bleeding. The treatment being tested is called Emicizumab, which is also known by its code names RO5534262 and ACE910. Emicizumab is administered as a solution for injection under the skin, known as subcutaneous use. The purpose of this study is to evaluate how effective and safe Emicizumab is for patients from birth to 12 months of age who have Hemophilia A without inhibitors, which are substances that can interfere with treatment.

Participants in the study will receive Emicizumab injections over a period of time, and researchers will monitor various aspects of their health. This includes checking how well the treatment works in preventing bleeding episodes, as well as observing any side effects or reactions to the medication. The study will also look at how the body processes Emicizumab, which is referred to as its pharmacokinetic profile, and how the drug affects the body, known as pharmacodynamics. Additionally, the study will assess the immune response to the treatment.

The trial is designed to gather comprehensive data on the number of bleeding events, joint health, and any adverse events that may occur. Researchers will use tools like the Hemophilia Joint Health Score and MRI to evaluate joint health over time. The study aims to provide valuable insights into the long-term effects and benefits of Emicizumab for young patients with Hemophilia A, contributing to better management of the condition.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, the participant is confirmed to have a diagnosis of severe congenital hemophilia A without inhibitors. This means the body has very low levels of a protein called factor VIII, which is important for blood clotting.

    The participant must have no history of factor VIII inhibitors, which are substances that can interfere with treatment.

  2. Step 2

    Screening period

    A screening period of two weeks is conducted to ensure eligibility. During this time, a test confirms the absence of factor VIII inhibitors.

    The participant's medical history, including previous treatments and bleeding episodes, is documented.

  3. Step 3

    Treatment administration

    The participant receives emicizumab, a medication administered through subcutaneous injection, which means it is injected under the skin.

    The medication is available in two forms: Hemlibra 30 mg/mL and Hemlibra 150 mg/mL solutions for injection. The specific dosage and frequency are determined by the study protocol.

  4. Step 4

    Monitoring and follow-up

    Throughout the study, the participant is monitored for any bleeding episodes, including treated and spontaneous bleeds.

    Joint health is assessed using specific scoring systems and imaging techniques at certain time points during a long-term follow-up period of up to seven years.

  5. Step 5

    Safety assessments

    The study evaluates the safety of the treatment by monitoring for adverse events, which are any unwanted effects that may occur.

    The severity of these events is assessed, and any reactions at the injection site are recorded.

  6. Step 6

    End of study

    The study is estimated to conclude by May 18, 2030. At this point, all data collected will be analyzed to determine the efficacy and safety of the treatment.

Who can join the trial?

7 criteria

  • The patient must have no history of a documented **FVIII inhibitor**. This means their FVIII inhibitor level should be less than 0.6 BU/mL. FVIII inhibitors are substances that can interfere with the treatment for hemophilia A.
  • The patient must have received **vitamin K prophylaxis** as per local standard practice. Prophylaxis means preventive treatment to avoid bleeding problems.
  • The patient must have a diagnosis of severe **congenital hemophilia A**, which means their natural FVIII level is less than 1%. FVIII is a protein important for blood clotting.
  • The patient must have a negative test for FVIII inhibitor, meaning less than 0.6 Bethesda units (BU)/mL, during the 2-week screening period before the trial starts.
  • The patient should be either a **previously untreated patient (PUP)** or a **minimally treated patient (MTP)**. This means they have had up to 5 days of exposure to hemophilia-related treatments like plasma-derived FVIII, recombinant FVIII, fresh frozen plasma, cryoprecipitate, or whole blood products.
  • The patient must have documentation of the details of the hemophilia-related treatments they have received since birth and details of any bleeding episodes they have experienced since birth.
  • The trial is only for male patients.

Who cannot join the trial?

7 criteria

  • Patients who have any other bleeding disorders besides Hemophilia A cannot participate. Hemophilia A is a condition where blood doesn't clot properly.
  • Patients who have had a recent surgery or are planning to have surgery soon are not eligible.
  • Patients who have an active infection or are currently being treated for an infection cannot join the study.
  • Patients who have a history of allergic reactions to similar treatments are excluded.
  • Patients who are currently participating in another clinical trial are not allowed to join this study.
  • Patients who have any serious medical conditions that might interfere with the study are not eligible.
  • Patients who are unable to follow the study procedures or attend the required visits cannot participate.
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Investigated drugs

Emicizumab is a medication used in this clinical trial to help manage Hemophilia A in young patients. It is designed to prevent or reduce bleeding episodes by mimicking the activity of a protein that helps blood to clot. This medication is given as an injection under the skin and is being studied for its effectiveness and safety in children from birth to 12 months old who do not have inhibitors.

What is already known about the treatment

Emicizumab – Emicizumab is administered as a subcutaneous injection, meaning it is injected under the skin. It is currently being studied in clinical trials and is recognized in medical literature for its potential benefits in treating Hemophilia A, particularly in patients without inhibitors. The main therapeutic indication for Emicizumab is to prevent or reduce bleeding episodes in individuals with Hemophilia A. At the molecular level, Emicizumab works by bridging activated factor IX and factor X, which are proteins involved in the blood clotting process, thereby mimicking the function of missing factor VIII. It is classified pharmacologically as a bispecific monoclonal antibody.

Investigated diseases

Hemophilia A – Hemophilia A is a genetic disorder characterized by a deficiency in clotting factor VIII, which is essential for blood clotting. This condition leads to prolonged bleeding after injuries, surgeries, or even spontaneously without any apparent cause. Individuals with Hemophilia A may experience frequent nosebleeds, easy bruising, and bleeding into joints and muscles, which can cause pain and swelling. Over time, repeated bleeding into joints can lead to joint damage and reduced mobility. The severity of symptoms can vary, with some individuals experiencing mild symptoms and others having more severe bleeding episodes. Hemophilia A is typically diagnosed in childhood, often after a bleeding episode or during routine medical tests.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IIITrial ID2023-505964-13-00Protocol codeMO41787Estimated enrolment65 patientsSponsorF. Hoffmann-La Roche AG

sourced from the EU Clinical Trials Register and site verification

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