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Study on Joint Health in Hemophilia A and B Patients Using Efmoroctocog Alfa or Eftrenonacog Alfa

Verified siteRegistered drugNo 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 two treatments for patients with Hemophilia A and Hemophilia B. These are genetic disorders where blood does not clot properly, leading to excessive bleeding. The treatments being studied are called efmoroctocog alfa and eftrenonacog alfa. Efmoroctocog alfa is a recombinant fusion protein that acts like a human coagulation factor VIII, while eftrenonacog alfa is a similar protein that acts like factor IX. These treatments are given as injections and are used to prevent bleeding episodes in patients with hemophilia.

The purpose of this study is to assess the health of joints in patients with hemophilia who are receiving these treatments over an 18-month period. The study will use ultrasound, a type of imaging that uses sound waves to create pictures of the inside of the body, to evaluate joint health. Participants will receive either efmoroctocog alfa or eftrenonacog alfa as part of their regular treatment plan. Some participants may receive a placebo, which is a substance with no active medication, to compare the effects of the treatments.

Throughout the study, participants will have regular check-ups to monitor their joint health and overall well-being. The study aims to understand how these treatments affect joint health and to gather information that could help improve care for people with hemophilia. The study will last for 18 months, during which participants will continue their usual care while being monitored for any changes in their 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

    Enrollment and consent

    Upon joining the study, you will provide a signed and dated informed consent. If you are under the legal age, your legally authorized representative will provide consent, and your assent will be obtained as per local regulations.

  2. Step 2

    Baseline assessment

    You will undergo a baseline assessment, which includes documenting your treatment history and any bleeding episodes from the past six months. This information is crucial for establishing your starting point in the study.

  3. Step 3

    Start of prophylactic treatment

    You will begin or continue prophylactic treatment with either efmoroctocog alfa or eftrenonacog alfa. These medications are administered as a solution for injection. The specific dosage and frequency will be determined by your healthcare provider.

  4. Step 4

    Regular follow-up visits

    Throughout the 18-month study period, you will attend regular follow-up visits. During these visits, your joint health will be assessed using ultrasound to monitor any changes in your joint status.

  5. Step 5

    Data collection

    Data will be collected on various aspects of your health, including joint health scores, bleeding rates, and physical function. This information will help evaluate the effectiveness of the treatment.

  6. Step 6

    End of study assessment

    At the end of the 18-month period, a final assessment will be conducted to evaluate changes in your joint health and overall outcomes. This will include a comparison of your baseline and end-of-study data.

Who can join the trial?

7 criteria

  • Age must be 6 years or older.
  • Must have a diagnosis of hemophilia A or B (a condition where blood does not clot properly).
  • Must have at least 6 months of documented treatment history, including treatment prescriptions and bleeding episodes, before the start of the study.
  • Must have been previously treated for hemophilia A or B with any approved recombinant (lab-made) or plasma-derived FVIII or FIX concentrate for at least 6 months.
  • Must have started preventive treatment with rFVIIIFc or rFIXFc (specific medications for hemophilia) before joining the study or at the latest by the first study visit, following local rules.
  • Must provide signed and dated informed consent. If under the legal age, consent must be provided by a legally authorized representative, and agreement (assent) should be obtained from children, following local rules.
  • To join the florio HAEMO sub-study, must have used the florio HAEMO app (a medical device used in regular clinical practice) for at least 3 months and agree to have data collected from the app by providing separate consent or assent.

Who cannot join the trial?

5 criteria

  • Patients who do not have Hemophilia A or Hemophilia B cannot participate.
  • Patients who are not within the age range of 2 to 4 years old cannot participate.
  • Patients who are not part of the specified clinical trial group cannot participate.
  • Patients who are not male or female cannot participate.
  • Patients who are not considered part of a vulnerable population cannot participate.
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Investigated drugs

  • Efmoroctocog alfa

    is a medication used in the treatment of hemophilia A. Hemophilia A is a condition where the blood does not clot properly due to a lack of a specific protein. This medication helps to replace the missing protein, allowing the blood to clot more effectively. In this clinical trial, efmoroctocog alfa is used as a preventive treatment to help reduce bleeding episodes and improve joint health over time.

  • Eftrenonacog alfa

    is a medication used for treating hemophilia B, which is similar to hemophilia A but involves a different missing protein. This medication works by providing the body with the protein it lacks, helping the blood to clot properly. In the trial, eftrenonacog alfa is used as a preventive measure to help manage bleeding and protect joint health in patients with hemophilia B.

What is already known about the treatment

  • Efmoroctocog alfa

    Efmoroctocog alfa is administered through an injection into a vein, typically used for people with Hemophilia A. It is currently being studied in clinical trials to understand its effects on joint health over 18 months. This medication helps prevent bleeding by replacing a missing protein in the blood, known as factor VIII, which is crucial for blood clotting. It belongs to a class of drugs called clotting factor replacements.

  • Eftrenonacog alfa

    Eftrenonacog alfa is given as an injection into a vein and is used for individuals with Hemophilia B. It is part of ongoing research to evaluate its impact on joint health over an 18-month period. This medication works by providing the body with factor IX, a protein that is essential for blood clotting, helping to prevent bleeding episodes. It is classified as a clotting factor replacement therapy.

Investigated diseases

  • Hemophilia A

    Hemophilia 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. Over time, repeated bleeding episodes can cause damage to joints, muscles, and other tissues. The disease primarily affects males, as it is linked to the X chromosome. Individuals with Hemophilia A may experience frequent nosebleeds, easy bruising, and excessive bleeding from cuts or injuries. Joint bleeding is common, leading to pain and swelling.

  • Hemophilia B

    Hemophilia B is a genetic disorder resulting from a deficiency of clotting factor IX, which is crucial for proper blood coagulation. Similar to Hemophilia A, this condition causes prolonged bleeding after injuries or surgeries and can also occur spontaneously. The disease is inherited in an X-linked recessive pattern, predominantly affecting males. Repeated bleeding episodes can lead to joint damage and muscle hemorrhages over time. Symptoms include frequent nosebleeds, easy bruising, and excessive bleeding from minor cuts or injuries. Joint bleeding is a common issue, causing pain and swelling.

Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2022-502921-16-00Protocol codeSobi.HAEM89-007Estimated enrolment250 patientsSponsorSwedish Orphan Biovitrum AB (publ)

sourced from the EU Clinical Trials Register and site verification

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