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Study on How Concizumab Works for Patients with Hemophilia A or B with Inhibitors

Verified siteInvestigationalNo placebo
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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 the effects of a medication called concizumab in people with hemophilia A or hemophilia B who have developed inhibitors. Hemophilia is a condition where the blood does not clot properly, leading to excessive bleeding. Inhibitors are antibodies that can develop in some patients, making standard treatments less effective. The medication being tested, concizumab, is given as a solution for injection using a pen-injector device.

The purpose of the study is to see how well concizumab works in reducing the number of bleeding episodes compared to no regular preventive treatment. Participants will be divided into groups, with some receiving concizumab and others receiving their usual on-demand treatment. The study will last for several months, during which participants will receive regular injections of concizumab and be monitored for any changes in their bleeding patterns.

Throughout the study, researchers will collect information on the number of bleeding episodes, any side effects, and how the participants feel physically. This information will help determine if concizumab is a safe and effective treatment option for people with hemophilia A or B with inhibitors. The study aims to provide valuable insights into managing this condition and improving the quality of life for those affected.

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, informed consent is required. This means understanding the trial and agreeing to participate.

    Eligibility includes being male, at least 12 years old, weighing more than 25 kg, and having haemophilia A or B with inhibitors.

  2. Step 2

    Initial assessment

    An initial assessment is conducted to confirm eligibility and gather baseline health information.

  3. Step 3

    Randomization

    Participants are randomly assigned to one of two groups: one receiving concizumab and the other receiving on-demand treatment with bypassing agents.

  4. Step 4

    Treatment phase

    For those receiving concizumab, the medication is administered as a subcutaneous injection. The dosage is either 40 mg/mL or 100 mg/mL, depending on the specific needs.

    The treatment continues for at least 32 weeks, with regular monitoring of health and any bleeding episodes.

  5. Step 5

    Monitoring and evaluation

    Throughout the trial, the number of bleeding episodes is recorded, along with any side effects such as injection site reactions or hypersensitivity.

    Regular assessments are conducted to evaluate changes in physical functioning and pain levels.

  6. Step 6

    Conclusion of trial

    The trial is expected to conclude by the end of 2025, with a final analysis of the data collected to determine the efficacy and safety of concizumab.

Who can join the trial?

5 criteria

  • Must provide informed consent before any trial-related activities. This means you agree to participate after understanding the trial details.
  • Must be a male who is 12 years old or older at the time of signing the informed consent.
  • Must have a body weight greater than 25 kilograms at the time of screening.
  • Must have congenital haemophilia A or B of any severity. This means you were born with this condition. You must also have a documented history of an inhibitor with a level of 0.6 BU or higher. An inhibitor is a substance in the blood that can make treatment less effective.
  • Must have been prescribed, or need, treatment with bypassing agents in the last 24 weeks before screening. Bypassing agents are special treatments used when regular treatments don't work well due to inhibitors.

Who cannot join the trial?

3 criteria

  • Patients who do not have **haemophilia A** or **haemophilia B** with **inhibitors** cannot participate. **Haemophilia** is a condition where blood doesn't clot properly, and **inhibitors** are substances that stop treatments from working effectively.
  • Only **male** patients are eligible, so female patients cannot participate.
  • Patients who are part of a **vulnerable population** are not eligible. This term generally refers to groups who may need special protection, like children or those unable to give consent.
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Investigated drugs

Concizumab is a medication being studied for its ability to prevent bleeding episodes in patients with hemophilia A or B who have developed inhibitors. It is used as a prophylactic treatment, meaning it is given regularly to help prevent bleeding rather than treating it after it occurs. The trial aims to see how effective concizumab is compared to not using any regular preventive treatment.

What is already known about the treatment

Concizumab – Concizumab is administered as an injection under the skin, designed to be used regularly to prevent bleeding episodes in patients with hemophilia A or B who have developed inhibitors. It is currently being studied in clinical trials to assess its effectiveness and safety compared to traditional on-demand treatments. The main therapeutic indication for concizumab is to reduce the frequency of bleeding episodes in these patients. At the molecular level, concizumab works by inhibiting a protein called tissue factor pathway inhibitor (TFPI), which plays a role in the regulation of blood clotting. It is classified as an anticoagulant therapy, specifically targeting the coagulation pathway to enhance clot formation and reduce bleeding risks.

Investigated diseases

  • Hemophilia A with inhibitors

    Hemophilia A is a genetic disorder where the blood does not clot properly due to a deficiency of clotting factor VIII. In some patients, the body develops inhibitors, which are antibodies that attack the replacement clotting factor, making treatment more challenging. This condition leads to frequent bleeding episodes, which can occur spontaneously or due to injury. Bleeding often happens in joints and muscles, causing pain and swelling. Over time, repeated bleeding into joints can lead to joint damage and reduced mobility. Managing bleeding episodes becomes more complex when inhibitors are present, requiring alternative treatment strategies.

  • Hemophilia B with inhibitors

    Hemophilia B is a genetic disorder characterized by a deficiency of clotting factor IX, leading to improper blood clotting. Similar to Hemophilia A, some individuals develop inhibitors that neutralize the effectiveness of replacement factor IX therapy. This results in increased bleeding episodes, which can be spontaneous or triggered by trauma. Bleeding commonly affects joints and muscles, leading to pain and potential joint damage over time. The presence of inhibitors complicates the management of the disease, necessitating different therapeutic approaches to control bleeding. Regular monitoring and specialized care are essential to manage the condition effectively.

Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-506832-33-00Protocol codeNN7415-4311Estimated enrolment131 patientsSponsorNovo Nordisk A/S

sourced from the EU Clinical Trials Register and site verification

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