RecruitingPHASE4INTERVENTIONAL

Efanesoctocog Alfa Prophylaxis in Patients With Hemophilia A With Synovial Hypertrophy

This study is for people with haemophilia A, a condition where blood doesn't clot properly. The main goal is to check if a new medicine called efanesoctocog alfa can help improve joint swelling. Participants will take this medicine once a week for about a year. Researchers want to see if the swelling (called synovial hypertrophy) in joints gets better over these 12 months. This is an important step to understand how this new treatment might help people manage their haemophilia A and improve their joint health.

At a glance

Status

Recruiting

Phase

PHASE4

Sponsor

Sanofi

Enrolment target

Start

23 Sep 2025

Estimated completion

25 Oct 2027

Factor VIII Deficiency

What is this study about?

Imagine your body has tiny workers called clotting factors that help stop bleeding. In people with haemophilia A, there aren't enough of these workers (specifically, Factor VIII), which means they can bleed more easily and for longer. This bleeding often happens into joints, especially if they're not protected enough. Over time, this can cause the lining of the joints (called the synovium) to become thick and swollen, a condition known as synovial hypertrophy, which can lead to pain and damage.

This study, called a Phase 4 trial, is looking at a medicine named efanesoctocog alfa. If you have haemophilia A, you might already take medicine regularly to prevent bleeds – this is called prophylaxis. The study wants to see if using efanesoctocog alfa once a week can reduce the swelling in joints that are already affected by synovial hypertrophy. It's like checking if a new approach can help calm down irritated joints.

The main aim is to understand how well this treatment works over a year to make joints feel and look better. This kind of research helps doctors find new and better ways to manage haemophilia A, aiming to improve the quality of life for people living with the condition.

Key takeaways

This study evaluates efanesoctocog alfa for haemophilia A.
It aims to reduce joint swelling (synovial hypertrophy).
Treatment is once per week for around 12 months.
Participants must have existing joint swelling and moderate to severe haemophilia A.
The study helps improve understanding of new haemophilia treatments.

Who may be eligible?

To join this study, you need to have moderate to severe haemophilia A, meaning your natural Factor VIII levels are 5% or less. You also need to be at least 12 years old. A key part of joining is having at least one joint with some existing swelling (synovial hypertrophy), which will be checked using a special ultrasound.

It's important that you haven't had any major joint surgery on the affected joint in the last three months, and you shouldn't have any planned during the study. You should also have been taking regular preventative haemophilia medicine (prophylaxis) in the past year. Women who could become pregnant will need to use effective birth control during the study.

There are also reasons why you might not be able to join. For example, if you have other bleeding disorders besides haemophilia A, or if you are already taking part in another study that might affect this one, you wouldn't be eligible. The study team will go through all the details to make sure it's the right fit for you.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Do you have moderate to severe haemophilia A?
Are you 12 years old or older?
Do you have at least one joint with existing swelling (synovial hypertrophy) that has been checked by a doctor?
Have you been having regular preventative haemophilia medicine (prophylaxis) in the last year?
Have you *not* had major joint surgery on the affected joint in the last 3 months, and don't have one planned soon?

Answer every question to see your result.

What does participation involve?

If you decide to take part, the study will last for about 12 months. Throughout this time, you will receive the study medicine, efanesoctocog alfa, once a week. The study team will regularly check how your joints are doing, especially the ones with swelling, using methods like ultrasound scans. They will also monitor your overall health and how you respond to the treatment. You will have regular visits to the clinic for these checks, and the team will explain everything in detail, including how to take the medicine and what to expect.

Potential risks and benefits

Taking part in any study has potential benefits and risks. A potential benefit could be an improvement in your joint swelling and overall joint health. However, as with any new medicine, there might be side effects, though the study team will monitor you closely for these. You will be given full information about known effects of the medicine. It's very important to remember that joining is completely voluntary, and you can change your mind and leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (13)

Orthopedic Institute for Children- Site Number : 8400009
Verified postcode
Los Angeles, United States· Recruiting
Innovative Hematology, Inc.- Site Number : 8400006
Verified postcode
Indianapolis, United States· Recruiting
University of Iowa- Site Number : 8400004
Verified postcode
Iowa City, United States· Recruiting
Ohio State University Hospital East- Site Number : 8400001
Verified postcode
Columbus, United States· Recruiting
Investigational Site Number : 1240003
Verified postcode
Hamilton, Canada· Recruiting
Investigational Site Number : 1240004
Verified postcode
Hamilton, Canada· Recruiting
Investigational Site Number : 3920003
Verified postcode
Kawasaki, Japan· Recruiting
Investigational Site Number : 3920002
Verified postcode
Hiroshima, Japan· Recruiting
Investigational Site Number : 3920001
Verified postcode
Saitama, Japan· Recruiting
Investigational Site Number : 3920004
Verified postcode
Tokyo, Japan· Recruiting
Investigational Site Number : 1580003
Verified postcode
Kaohsiung City, Taiwan· Recruiting
Investigational Site Number : 1580001
Verified postcode
Taichung, Taiwan· Recruiting

Common questions

What is haemophilia A?

Haemophilia A is a genetic bleeding disorder where your blood doesn't clot properly due to a lack of a specific protein called Factor VIII.

What is synovial hypertrophy?

It's when the lining of your joints (the synovium) becomes thick and swollen, often due to repeated bleeding into the joint. This can cause pain and lead to long-term joint damage.

What is efanesoctocog alfa?

It's a new type of medicine designed to replace the missing Factor VIII in people with haemophilia A. This study is testing how well it helps with joint swelling.

How long will I take part in the study?

If you join, you will be part of the study for about 12 months, receiving the treatment once a week.

Can I leave the study if I change my mind?

Yes, completely. Taking part is your choice, and you can withdraw from the study at any time without having to give a reason.

How to find out more

Trial Transparency email recommended (Toll free for US & Canada)

contact-us@sanofi.com 800-633-1610 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.