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Study on Physical Activity and Joint Health in Patients Aged 12 and Older with Severe Hemophilia A Treated with Efanesoctocog Alfa for 24 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 severe hemophilia A, a condition where the blood does not clot properly, leading to excessive bleeding. The treatment being tested is called efanesoctocog alfa, also known by its code name BIVV001. This medication is a type of protein that helps the blood to clot and is given as a powder mixed into a solution for injection into a vein.

The purpose of the study is to observe changes in physical activity and joint health in patients aged 12 and older who have been previously treated for severe hemophilia A. Participants will receive the treatment once a week for a period of 24 months. During the study, participants will use an activity tracker to monitor their physical activity and heart rate. They will also report their physical activities and any changes in their joint health.

The study will last for 24 months, during which participants will be monitored for any changes in their condition. The study aims to understand how the treatment affects physical activity levels and joint health over time. Participants will be asked to use a smartphone or tablet to record their activities and will have regular check-ups to assess their progress. The study will also look at the number of bleeding episodes and any side effects that may occur during the treatment period.

The research process

The trial runs in 7 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, you will be required to provide informed consent. This means you agree to participate after understanding the study's purpose and procedures.

    You will be asked to confirm that you meet the eligibility criteria, such as being 12 years or older and having a diagnosis of severe haemophilia A.

  2. Step 2

    Initial assessment

    An initial assessment will be conducted to establish your baseline health status. This includes a review of your medical history and a physical examination.

    A blood test will be performed to check your platelet count and other relevant health markers.

  3. Step 3

    Training and equipment

    You will receive training on how to administer the study medication, efanesoctocog alfa, at home through an intravenous injection.

    You will be provided with an activity tracker to monitor your physical activity and heart rate throughout the study.

  4. Step 4

    Medication administration

    You will administer efanesoctocog alfa once weekly. This medication is a powder that you will dissolve in a solution for injection.

    The treatment will continue for a period of 24 months, during which you will record each administration in an electronic patient diary.

  5. Step 5

    Regular follow-up visits

    You will attend regular follow-up visits to monitor your health and the effects of the medication. These visits will include physical examinations and laboratory tests.

    You will be asked to report any side effects or changes in your health status during these visits.

  6. Step 6

    Physical activity monitoring

    Your physical activity will be monitored using the activity tracker provided. You will also complete questionnaires about your physical activity levels.

    The study aims to evaluate changes in your physical activity and joint health over the 24-month period.

  7. Step 7

    End of study assessment

    At the end of the 24-month period, a final assessment will be conducted to evaluate your overall health and the impact of the treatment.

    You will return any study equipment and complete a final set of questionnaires.

Who can join the trial?

12 criteria

  • Be at least 12 years old at the time of signing the consent form.
  • Have a diagnosis of severe haemophilia A, which means having less than 1% of normal clotting factor VIII activity in the blood.
  • Have been treated for haemophilia A with any approved recombinant or plasma-derived factor VIII for at least 150 exposure days (EDs).
  • Have been on regular preventive treatment for at least 12 months with any approved factor VIII product or emicizumab before joining the study. If using emicizumab, it must be stopped at least 26 weeks before joining the study.
  • Have 12 months of documented treatment history and 6 months of bleeding episode records before the start of the study.
  • Have a platelet count of at least 100,000 cells per microliter at the screening. Platelets are cells that help with blood clotting.
  • If HIV positive, must have a CD4 lymphocyte count greater than 200 cells per cubic millimeter and a viral load of less than 400 copies per milliliter. CD4 lymphocytes are a type of white blood cell important for the immune system, and viral load measures the amount of HIV in the blood.
  • Be able and willing to give the study medication, efanesoctocog alfa, through an intravenous (IV) injection at home.
  • Be willing and able to complete training and use the study's electronic patient diary (ePD) on a smartphone or tablet throughout the study.
  • Be willing and able to use the activity tracker (like a Fitbit) provided by the study to measure physical activity and heart rate.
  • If female, must not be pregnant or breastfeeding. Women of childbearing potential must use an acceptable method of birth control during the study and have a negative pregnancy test before starting the study.
  • Provide signed and dated informed consent, or have a legally authorized representative provide it if under the legal age. For children, assent should be obtained according to local laws.

Who cannot join the trial?

7 criteria

  • Patients with any other bleeding disorders besides Haemophilia A cannot participate.
  • Patients who have had a recent surgery or are planning to have surgery soon are not eligible.
  • Patients with severe liver or kidney disease cannot take part in the study.
  • Patients who are currently participating in another clinical trial are not allowed to join.
  • Patients who have a history of allergic reactions to the study medication or similar medications cannot participate.
  • Pregnant or breastfeeding women are not eligible for the study.
  • Patients with any condition that the study doctors believe would make it unsafe for them to participate are excluded.
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Investigated drugs

Efanesoctocog alfa is a medication used in this clinical trial to help people with severe hemophilia A. Hemophilia A is a condition where the blood does not clot properly, leading to excessive bleeding. This medication is designed to replace a missing protein in the blood called factor VIII, which is essential for blood clotting. By providing this protein, efanesoctocog alfa helps to prevent bleeding episodes. In this study, patients receive the medication through an intravenous infusion, which means it is given directly into a vein. The goal is to see how this treatment affects physical activity and joint health over a period of 24 months.

What is already known about the treatment

Efanesoctocog alfa – Efanesoctocog alfa is administered through an intravenous injection, meaning it is delivered directly into the bloodstream. It is currently being studied in clinical trials, particularly for its effects on physical activity and joint health in patients with severe Hemophilia A. This medication is primarily used to prevent bleeding episodes in individuals with Hemophilia A, a condition where blood does not clot properly. At the molecular level, efanesoctocog alfa works by replacing the missing or defective factor VIII, a protein essential for blood clotting, thereby helping to control bleeding. It is classified as a recombinant coagulation factor, specifically designed to mimic the natural clotting process in the body.

Investigated diseases

Haemophilia A – Haemophilia A is a genetic disorder caused by a deficiency of 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 Haemophilia A may experience frequent nosebleeds, easy bruising, and excessive bleeding from cuts or injuries. Over time, repeated bleeding episodes can cause damage to joints and muscles, leading to pain and reduced mobility. The severity of the disease can vary, with some individuals experiencing mild symptoms and others having severe bleeding episodes. The condition is typically inherited and primarily affects males, although females can be carriers and occasionally exhibit symptoms.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2022-500275-31-00Protocol codeSobi.BIVV001-001Estimated enrolment90 patientsSponsorSwedish Orphan Biovitrum AB (publ)

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