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Study on Emicizumab and Drug Combination for Patients with Haemophilia A and FVIII Inhibitors

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What is this trial about?

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

This clinical trial focuses on studying different treatment approaches for patients with Hemophilia A, a condition where blood does not clot properly due to a lack of a specific protein called factor VIII. The study involves several treatments, including Hemlibra (also known as emicizumab), Nuwiq (simoctocog alfa), FEIBA (factor VIII inhibitor bypassing fraction), Wilate (human coagulation factor VIII and human von Willebrand factor), OCTANATE (human coagulation factor VIII), and NovoSeven (eptacog alfa, activated). These treatments are administered either through injections or infusions.

The purpose of the study is to evaluate the effectiveness of these treatments in patients with Hemophilia A who have developed inhibitors, which are antibodies that reduce the effectiveness of factor VIII treatments. The study is divided into three groups. Groups 1 and 2 will focus on evaluating the success of immune tolerance induction (ITI), a process aimed at reducing inhibitor levels, while Group 3 will assess the annualized bleeding rate (ABR) compared to the other groups. Participants will receive their assigned treatments over a period of up to 60 weeks, with regular monitoring to assess their response to the treatment.

Throughout the study, participants will be monitored for the frequency and severity of bleeding episodes, the number of infusions needed to control bleeding, and any side effects or adverse reactions. The study aims to provide valuable insights into the best treatment strategies for managing Hemophilia A in patients with inhibitors, ultimately improving their quality of life and treatment outcomes.

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, participants are assigned to one of three groups based on their treatment needs and history with hemophilia A.

    Participants must have a historical inhibitor titre of at least 0.6 Bethesda units per milliliter (BU/mL).

  2. Step 2

    Initial treatment phase

    Participants in Groups 1 and 2 undergo immune tolerance induction (ITI) therapy using products such as Nuwiq, Octanate, or Wilate.

    During ITI, some participants may also receive prophylactic therapy with emicizumab, activated prothrombin complex concentrate (aPCC), or recombinant activated factor VII (rFVIIa).

  3. Step 3

    Medication administration

    Emicizumab is administered subcutaneously, with available dosages including 30 mg/mL and 150 mg/mL solutions for injection.

    Nuwiq, Octanate, and Wilate are administered intravenously, with various dosages available, such as 250 IU, 500 IU, 1000 IU, 2000 IU, 2500 IU, and 3000 IU.

  4. Step 4

    Monitoring and evaluation

    Participants are regularly monitored to evaluate the success of ITI, which includes achieving an inhibitor titre of less than 0.6 BU/mL for at least two consecutive measurements.

    The recovery and half-life of factor VIII are also assessed to determine the effectiveness of the treatment.

  5. Step 5

    Bleeding rate assessment

    For Group 3, the annualized bleeding rate (ABR) is evaluated and compared with the ABR in Groups 1 and 2.

    The frequency and severity of bleeding episodes, including joint bleeds, are recorded over time.

  6. Step 6

    Follow-up and outcomes

    Participants who achieve complete ITI success are followed up to monitor for any relapses of factor VIII inhibitors.

    The study also tracks adverse drug reactions (ADRs), thrombotic events, and treatment costs.

Who can join the trial?

7 criteria

  • Participants can be of any age at the time of joining the trial.
  • Must be male with **Hemophilia A**, which is a condition where blood doesn't clot properly, of any severity.
  • Must have a past **inhibitor titre** of at least 0.6 Bethesda units per milliliter (BU/mL). An inhibitor titre is a measure of substances in the blood that can interfere with treatment.
  • Can include those who have not succeeded in previous **ITI** attempts. ITI stands for **Immune Tolerance Induction**, a treatment to help the body accept clotting factor treatments.
  • Must be undergoing ITI with specific treatments like Nuwiq®, octanate®, or wilate®.
  • Can also be receiving preventive therapy with emicizumab, aPCC, or rFVIIa. These are medications used to help prevent bleeding episodes.
  • Participants or their parent(s)/legal guardian(s) must be able to give signed informed consent, meaning they understand and agree to the trial's terms.

Who cannot join the trial?

4 criteria

  • Only male participants are allowed, so females cannot participate.
  • Participants must have Haemophilia A, a condition where blood doesn't clot properly.
  • Participants must be within certain age ranges, which are not specified here.
  • Participants must not belong to a vulnerable population, which means they should not be in a group that needs special protection or care.
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Investigated drugs

  • Emicizumab

    is a medication used in the trial to help prevent bleeding episodes in patients with Hemophilia A. It works by mimicking the activity of a protein called factor VIII, which is missing or not working properly in these patients. This medication helps the blood to clot more effectively, reducing the risk of bleeding.

  • Factor VIII Concentrate

    is used in the trial to replace the missing or deficient factor VIII in patients with Hemophilia A. This therapy helps to control and prevent bleeding episodes by providing the necessary clotting factor that the body lacks. It is an essential part of the treatment for managing Hemophilia A.

  • ITI Therapy

    (Immune Tolerance Induction) is a treatment approach used in the trial for patients who have developed inhibitors against factor VIII. The goal of ITI therapy is to train the immune system to accept factor VIII without reacting against it, allowing patients to use factor VIII concentrates effectively to manage their condition.

What is already known about the treatment

Factor VIII – This medication is administered intravenously and is used to treat Haemophilia A, a condition where blood does not clot properly due to a deficiency of Factor VIII. It is currently well-established in medical literature as a critical treatment for managing bleeding episodes in individuals with this condition. The main therapeutic indication is to replace the missing or deficient Factor VIII, thereby helping the blood to clot more effectively. At the molecular level, Factor VIII works by binding to another protein called von Willebrand factor in the bloodstream, which helps stabilize it and allows it to participate in the clotting process. It is classified pharmacologically as a coagulation factor replacement therapy.

Investigated diseases

Haemophilia A – Haemophilia A is a genetic disorder characterized by a deficiency in clotting factor VIII, which leads to prolonged bleeding. This condition primarily affects males and is inherited in an X-linked recessive pattern. Individuals with Haemophilia A may experience spontaneous bleeding episodes, particularly into joints and muscles, which can cause pain and swelling. Over time, repeated bleeding into joints can lead to chronic joint damage and reduced mobility. The severity of the disease varies, with some individuals experiencing frequent bleeding episodes and others having milder symptoms. Regular monitoring and management are essential to prevent complications associated with bleeding.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-516741-39-00Protocol codeMOTIVATEEstimated enrolment123 patientsSponsorEmory University

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).