Study on Synovial Hypertrophy in Patients with Hemophilia A Using Efanesoctocog Alfa Prophylaxis

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What is this study about?

This clinical trial is focused on studying Haemophilia A, a condition where blood does not clot properly, leading to excessive bleeding. The study will use a treatment called efanesoctocog alfa, also known by its code name BIVV001. This treatment is a type of protein that helps blood to clot and is given as an injection into a vein. The purpose of the study is to observe changes in joint health in patients with Haemophilia A who are receiving regular doses of efanesoctocog alfa.

Participants in the study will receive the treatment over a period of 12 months. During this time, doctors will use imaging techniques like ultrasound and MRI (Magnetic Resonance Imaging) to monitor the condition of the joints. These imaging methods help in detecting changes in the joint tissues, specifically looking for improvements in a condition called synovial hypertrophy, which is a thickening of the joint lining that can occur in people with Haemophilia A.

The study aims to see if the treatment can reduce this thickening and improve joint health over time. Participants will be monitored for any changes in their joint condition and overall health. The study will also collect information on how the treatment affects the frequency of bleeding episodes and the participants’ quality of life. This information will help in understanding the effectiveness and safety of efanesoctocog alfa for people living with Haemophilia A.

1 joining the study

Upon joining the study, the patient will begin a 12-month period of participation. The study focuses on the condition known as haemophilia A, which affects the blood’s ability to clot properly.

The main goal is to observe changes in joint health, specifically looking at synovial hypertrophy, which is a thickening of the joint lining.

2 treatment administration

The patient will receive a medication called efanesoctocog alfa as a preventive treatment. This medication is administered through an intravenous injection, which means it is injected directly into a vein.

The specific product used will be one of the following: ALTUVOCT 500 IU, 250 IU, 4,000 IU, 2,000 IU, or 3,000 IU, all of which are prepared as a solution for injection.

3 monitoring and assessments

Throughout the study, the patient’s joints will be monitored using ultrasound and MRI scans. These imaging techniques help in assessing the condition of the joints and any changes in synovial hypertrophy.

Assessments will occur at the start of the study, and then again at 6 months and 12 months to track any improvements or changes.

4 patient-reported outcomes

The patient will be asked to report on their experiences and preferences regarding the treatment. This includes completing questionnaires about their quality of life, pain levels, and physical function.

These reports will be collected at the beginning, at 6 months, and at the end of the study to understand the patient’s perspective on the treatment.

5 completion of the study

At the end of the 12-month period, the study will conclude. The final assessments will be conducted to evaluate the overall impact of the treatment on joint health and any changes in synovial hypertrophy.

The study aims to determine if there has been an improvement in joint conditions and to gather data on the safety and effectiveness of the treatment.

Who Can Join the Study?

Must be able to give signed informed consent, which means agreeing to follow the rules and requirements of the study. If the patient is under 18 years old or unable to give consent, a parent or legal representative must provide consent. Patients under 18 should also agree to participate if appropriate.
Must be a male or female patient who is at least 12 years old and diagnosed with moderate or severe haemophilia A. This means having 5% or less of the normal level of a blood clotting protein called FVIII.
If the patient is a female, she must not be pregnant when joining the study and should not plan to become pregnant during the study. Women who can have children must have a negative pregnancy test at the start of the study.
Must have been receiving regular preventive treatment with any approved FVIII product or a medication called emicizumab for at least 12 months before the study begins.
Must have at least one joint, such as an ankle, elbow, or knee, that qualifies for the study.
Must have 12 months of recorded information about haemophilia treatments and bleeding episodes before the study starts.
Must be willing and able, or have a legal representative who is willing and able, to learn how to use the study diary and to fill it out during the study.

Who Cannot Join the Study?

Patients who do not have Haemophilia A cannot participate. Haemophilia A is a condition where blood doesn’t clot properly.
Patients who are not within the specified age range cannot participate. The age range includes children and adults.
Patients who are part of a vulnerable population cannot participate. Vulnerable populations include groups that may need special protection.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

Site Name	City	Country
Oslo Universitetssykehus HF	Oslo	Norway
Hospital Universitario Y Politecnico La Fe	Valencia	Spain
IRCCS Humanitas Research Hospital	Rozzano	Italy
Azienda Ospedaliera Universitaria Federico II Di Napoli	Naples	Italy

Other Sites

Site Name	City	Country
Region Skane Skanes Universitetssjukhus	Lund	Sweden
Virgen del Rocío University Hospital	Sevilla	Spain
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico	Milan	Italy
Queen Silvia Childrens Hospital – Sahlgrenska University Hospital – Vaestra Goetalandsregionen	Gothenburg	Sweden
Flapjlhas Pzwz Ly Ifstdzuzjzset Bszhfqmig Dmd Hvipkwqb Utnfgjjzhwgyv Li Pcv	Madrid	Spain
Hosospyb Vdeo dzhexxne	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Italy	Not recruiting	11.11.2024
Norway	Not recruiting	11.11.2024
Spain	Not recruiting	11.11.2024
Sweden	Not recruiting	11.11.2024

Trial locations

Investigated drugs:

Efanesoctocog Alfa

Efanesoctocog alfa is a medication used in this clinical trial. It is designed for patients with hemophilia A and is used as a prophylactic treatment. The goal of this medication is to prevent bleeding episodes by providing a replacement for the missing or deficient factor VIII, which is essential for blood clotting. In this study, the focus is on observing how this medication affects synovial hypertrophy, which is a condition where the lining of the joints becomes thickened, often leading to joint pain and damage. The trial aims to see if regular use of efanesoctocog alfa can improve this condition as detected by joint ultrasound and MRI.

Investigated diseases:

Factor VIII deficiency

Haemophilia A – Haemophilia 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. Individuals with Haemophilia A may experience frequent nosebleeds, easy bruising, and joint bleeds, which can cause pain and swelling. Over time, repeated bleeding into joints can lead to chronic joint damage and reduced mobility. The severity of the disease can vary, with some individuals experiencing mild symptoms and others having severe bleeding episodes. It is a lifelong condition that primarily affects males, as it is linked to the X chromosome.

Trial ID:

2024-512066-33-00

Protocol code:

Sobi.BIVV001-004

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on Synovial Hypertrophy in Patients with Hemophilia A Using Efanesoctocog Alfa Prophylaxis

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