Not recruiting

Study on Health, Activity, and Joint Outcomes in Patients Aged 13-69 with Severe or Moderate Hemophilia A Using Emicizumab

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 a medication called Hemlibra (also known as emicizumab) in people with a condition known as Hemophilia A. Hemophilia A is a genetic disorder where the blood does not clot properly, leading to excessive bleeding. This study is specifically for individuals with severe or moderate forms of Hemophilia A who do not have inhibitors against a protein called FVIII, which is important for blood clotting.

The purpose of the study is to evaluate how emicizumab affects joint health, overall health, and physical activity in participants. Participants will receive emicizumab as a solution for injection under the skin. The study will observe changes in joint health, the number of bleeding episodes, and the quality of life over time. Participants will also use a wearable activity tracker to monitor physical activity levels throughout the study.

The study will last for a period of up to 36 months. During this time, researchers will collect information on joint health using various assessments, including imaging techniques like MRI. They will also gather data on the participants' physical activity and any changes in their health-related quality of life. The study will help understand the benefits and any potential side effects of using emicizumab for managing Hemophilia A.

The research process

The trial runs in 8 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 participant will begin receiving the medication Hemlibra (emicizumab).
The medication is administered as a subcutaneous injection, which means it is injected under the skin.
Step 2
Medication administration
The participant will receive Hemlibra at a dosage of 150 mg/mL.
The frequency and duration of the administration will be determined by the study protocol and the participant's specific needs.
Step 3
Monitoring joint health
The study will evaluate joint health over time using various assessments, including ultrasound and MRI scans.
Joint health will be assessed at the start of the study and at month 36.
Step 4
Tracking physical activity
Physical activity levels will be monitored using a wearable activity tracker, such as a Fitbit.
Changes in daily step count, active minutes, and types of physical activities will be recorded.
Step 5
Quality of life assessment
The study will assess the impact of Hemlibra on health-related quality of life using questionnaires.
Participants will provide feedback on their experience with the treatment and its impact on their daily life.
Step 6
Bleeding events monitoring
The study will track the number and type of bleeding events, including joint bleeds and spontaneous bleeds.
Participants will report any bleeding events using a specific questionnaire.
Step 7
Safety and side effects
The study will monitor for any adverse events or side effects, including injection-site reactions and hypersensitivity.
The incidence of anti-drug antibodies and other potential complications will also be assessed.
Step 8
Study completion
The study is expected to conclude by December 28, 2026.
Participants will have the opportunity to provide feedback on their preference for Hemlibra compared to previous treatments.

Who can join the trial?

6 criteria

Must have a diagnosis of **severe** or **moderate hemophilia A**. This means the body has very low levels of a protein called **factor VIII (FVIII)**, which helps blood to clot.
Must have a **negative test** for **FVIII inhibitor** during the screening period. This means the body should not have developed substances that block the action of FVIII.
No history of **FVIII inhibitory antibodies** in the last 5 years. These are substances that can interfere with FVIII. If the participant had a treatment called **immune tolerance induction (ITI)** at least 5 years ago and has not shown signs of these antibodies coming back, they can participate.
Must have been on **standard FVIII prophylaxis** for at least the last 24 weeks. This means regularly taking FVIII to prevent bleeding, regardless of how many bleeding episodes occurred during this time.
Must have **adequate hematologic, hepatic, and renal function**. This means the blood, liver, and kidneys should be working well.
For women who can have children: Must agree to either not have heterosexual intercourse or use **contraception** during the treatment period and for at least 24 weeks after the last dose of the study medication.

Who cannot join the trial?

10 criteria

Patients who do not have Severe or Moderate Hemophilia A cannot participate. Hemophilia A is a condition where blood doesn't clot properly, leading to excessive bleeding.
Patients who are not within the specified age range cannot participate. The age range includes children and adults.
Patients who are not willing to follow the study procedures cannot participate.
Patients who have other medical conditions that might interfere with the study cannot participate.
Patients who are pregnant or breastfeeding cannot participate.
Patients who are currently participating in another clinical trial cannot participate.
Patients who have had a recent surgery or are planning to have surgery soon cannot participate.
Patients who have a history of allergic reactions to the study medication cannot participate.
Patients who have a history of drug or alcohol abuse cannot participate.
Patients who have any other condition that the study doctors think might make it unsafe for them to participate cannot participate.

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Verified sites

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

Azienda Ospedaliera Universitaria Federico II Di Napoli
Verified
Naples, Italy
Azienda Ospedaliero Universitaria Careggi
Responsive
Florence, Italy
Fundacion Para La Investigacion Biomedica Del Hospital Universitario La Paz
Responsive
Madrid, Spain

Trial locations

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Investigated drugs

Emicizumab is a medication used in this clinical trial to help people with hemophilia A. It is designed to prevent bleeding by helping the blood to clot more effectively. This medication is given regularly to reduce the number of bleeding episodes and improve joint health and overall quality of life for participants.

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 approved for use in patients with hemophilia A, particularly those without inhibitors to factor VIII, and is well-documented in medical literature as an effective prophylactic treatment. The main therapeutic indication for emicizumab is to prevent bleeding episodes in individuals with severe or moderate hemophilia A. At the molecular level, emicizumab works by bridging activated factor IX and factor X, which mimics the function of missing factor VIII, thereby promoting blood clotting. It is classified pharmacologically as a bispecific monoclonal antibody.

Investigated diseases

Hemophilia A – Hemophilia A is a genetic disorder characterized by a deficiency in clotting factor VIII, which is essential for blood clotting. This condition leads to prolonged bleeding episodes, often occurring spontaneously or after minor injuries. Over time, repeated bleeding into joints can cause joint damage and pain, known as hemophilic arthropathy. Individuals with severe or moderate forms of Hemophilia A may experience frequent joint bleeds, which can lead to chronic joint issues and reduced mobility. The disease can significantly impact daily activities and quality of life due to the need for ongoing management of bleeding episodes. Hemophilia A is typically diagnosed in early childhood, and its severity can vary among individuals.