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Study on Emicizumab for Patients with Mild or Moderate Hemophilia A Without 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 is focused on studying the effects of a medication called Hemlibra, which contains the active substance emicizumab. The study is specifically for patients with hemophilia A, a condition where the blood does not clot properly, leading to excessive bleeding. This trial is for those with mild or moderate forms of hemophilia A who do not have inhibitors against factor VIII, a protein that helps blood clot.

The purpose of the study is to evaluate the safety and effectiveness of emicizumab in preventing bleeding episodes in these patients. Participants will receive emicizumab as a solution for injection under the skin. The study will monitor the number of bleeding episodes over time and assess any side effects or reactions to the medication. The trial will also look at how the medication affects joint health, quality of life, and physical activity.

Throughout the study, participants will be regularly assessed to ensure their safety and to gather information on how emicizumab is working in their bodies. The trial will also explore the presence of any antibodies that might develop against the medication. This research aims to provide valuable insights into the management of hemophilia A and improve treatment options for those affected by this condition.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Upon joining the study, the patient will be assessed to confirm eligibility based on specific criteria, such as having mild or moderate hemophilia A without FVIII inhibitors and a weight of at least 3 kg.
A negative test for inhibitors is required within 8 weeks prior to enrollment, and there should be no documented inhibitors or specific FVIII issues in the last 5 years.
Step 2
Initial assessment
The patient will undergo a comprehensive initial assessment, including documentation of previous treatments and bleeding episodes for at least the last 24 weeks.
Baseline measurements will be taken, including physical examination findings, vital signs, and ECG parameters.
Step 3
Medication administration
The patient will receive emicizumab, administered as a subcutaneous injection. The specific dosage and frequency will be determined by the study protocol.
The medication is available in two forms: Hemlibra 150 mg/mL and Hemlibra 30 mg/mL solutions for injection.
Step 4
Monitoring and follow-up
Regular monitoring will occur to evaluate the safety and efficacy of the treatment. This includes tracking the number of treated bleeds over time and assessing any adverse events.
The patient's joint health and quality of life will be assessed at specified timepoints using standardized tools.
Step 5
End of study
The study is estimated to conclude by December 31, 2025. At the end of the study, a final assessment will be conducted to evaluate the overall impact of the treatment.
The study aims to gather data on the incidence of adverse events, changes in physical health, and the patient's preference for emicizumab compared to previous treatments.

Who can join the trial?

6 criteria

Have a diagnosis of **mild** or **moderate hemophilia A**. This means your **FVIII level** should be between **more than 5% and less than 40%** for mild, or **between 1% and 5%** for moderate. **FVIII** is a protein important for blood clotting.
Weigh at least **3 kg** (about 6.6 pounds).
Need regular treatment to prevent bleeding, as decided by the doctor.
Have a **negative test for inhibitors** within 8 weeks before joining the study. An inhibitor is a substance that can stop the treatment from working.
No record of having inhibitors, a **FVIII half-life** of less than 6 hours, or **FVIII recovery** of less than 66% in the past 5 years. **Half-life** is the time it takes for the amount of FVIII to reduce by half in the body. **Recovery** is how well FVIII works after treatment.
Have records of your treatment details and the number of bleeding episodes for at least the last 24 weeks before joining the study.

Who cannot join the trial?

5 criteria

Patients with **severe hemophilia A** cannot participate. This means the study is only for those with mild or moderate forms of the condition.
Patients who have **FVIII inhibitors** are excluded. FVIII inhibitors are substances in the blood that can stop the treatment from working properly.
Patients who are not within the specified age range cannot participate. The study is for certain age groups only.
Patients who are not part of the specified clinical trial groups are excluded. This means only certain groups of patients are eligible.
Patients who are considered part of a **vulnerable population** may be excluded. Vulnerable populations include groups that might need special protection or care.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Responsive
Cliniques Universitaires Saint-Luc
Woluwe-Saint-Lambert, Belgium
Verified
Hopital Beaujon
Clichy, France
Responsive
Katholieke Universiteit te Leuven
Leuven, Belgium

Trial locations

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Investigated drugs

Emicizumab is a medication used in this clinical trial to help manage hemophilia A, a bleeding disorder. It works by mimicking the activity of a protein called factor VIII, which is missing or not working properly in people with hemophilia A. This medication helps to prevent bleeding episodes by promoting blood clotting. The trial aims to assess how safe and effective emicizumab is for patients with mild or moderate hemophilia A who do not have inhibitors against factor VIII.

Emicizumab

What is already known about the treatment

Emicizumab – Emicizumab is administered as an injection under the skin, typically on a weekly basis. It is currently being studied in clinical trials for its safety and effectiveness in treating mild or moderate hemophilia A in patients who do not have inhibitors against factor VIII. The main therapeutic use of Emicizumab is to prevent bleeding episodes in these patients. It works by mimicking the activity of factor VIII, a protein that helps blood to clot, thereby bridging activated factor IX and factor X to restore the blood clotting process. Emicizumab is classified as a monoclonal antibody and represents a novel approach in the management of hemophilia A.

Investigated diseases

Hemophilia A – Hemophilia A is a genetic disorder where the blood does not clot properly due to a deficiency of clotting factor VIII. This condition leads to prolonged bleeding after injuries, surgeries, or even spontaneously without any apparent cause. In mild or moderate cases, bleeding episodes may occur less frequently and are often associated with trauma or surgery. Individuals with this condition may experience joint bleeds, which can lead to joint damage over time if not managed properly. The severity of the disease is determined by the level of factor VIII activity in the blood, with mild cases having higher levels than severe cases. Regular monitoring and management are essential to prevent complications associated with bleeding episodes.