Active not recruitingPHASE2INTERVENTIONAL

Study to Evaluate the Efficacy and Safety of Satralizumab in FSHD1

This research is investigating a new treatment named Satralizumab for Facioscapulohumeral Muscular Dystrophy 1 (FSHD1). FSHD1 causes muscle weakness, and recent findings suggest that inflammation plays a role in its progression. Satralizumab works by targeting a specific inflammation pathway in the body, which researchers hope could reduce both muscle and overall body inflammation. The study will compare Satralizumab to a placebo (a dummy treatment) to understand if it's effective and safe. They're looking for adults aged 18 to 65 with a confirmed diagnosis of FSHD1 who meet certain criteria related to their muscle health and walking ability. The goal is to see if reducing inflammation can help manage FSHD1 symptoms and prevent further muscle damage.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Centre Hospitalier Universitaire de Nice

Enrolment target

Start

24 Jan 2024

Estimated completion

01 Mar 2027

Facioscapulohumeral Muscular Dystrophy 1

What is this study about?

Facioscapulohumeral Muscular Dystrophy, or FSHD, is a condition that causes muscles to weaken, most commonly in the face, shoulders, and upper arms, but it can affect other muscles too. The most common type is called FSHD1. Scientists have found that a specific gene, called DUX4, doesn't work correctly in people with FSHD1, leading to muscle damage. While there isn't a specific treatment for FSHD1 yet, new research suggests that inflammation, which is the body's natural response to injury or irritation, plays a big part in how the disease progresses in its early stages.

This study, called ReInForce, is looking into a new medication called Satralizumab. This medicine works by blocking a key inflammation signal in the body, specifically called IL-6. Our bodies naturally produce chemicals called cytokines, and some of these can cause inflammation. In people with FSHD1, studies have shown they often have higher levels of these inflammation-causing cytokines. IL-6 is one of these, and its levels seem to be linked to how severe FSHD1 is.

By blocking IL-6 with Satralizumab, researchers hope to reduce inflammation in both the muscles and the rest of the body. The goal is to see if this can help slow down muscle damage and improve the symptoms of FSHD1. The study will involve giving some participants Satralizumab and others a placebo (a treatment with no active medicine) to carefully compare their effects.

Key takeaways

This study investigates Satralizumab for FSHD1.
It aims to reduce inflammation, which may play a role in FSHD1 progression.
Participants will receive either the study drug or a placebo.
The study involves regular clinic visits for assessments like MRI and blood tests.
It targets adults (18-65) with specific FSHD1 characteristics.
Your participation helps advance understanding and potential treatments for FSHD1.

Who may be eligible?

To be able to join this study, you need to be an adult between 18 and 65 years old. You must have a confirmed diagnosis of FSHD1, which means genetic tests have shown you have the condition. Importantly, your FSHD1 should not be too mild or too severe; doctors will use a special score to check this. You should also be able to walk without needing support.

The research team will also look at your muscles using an MRI scan. They need to see evidence that some of your muscles have a certain amount of fat replacement, which is common in FSHD. You should also be willing to keep your current exercise routine the same throughout the study and be able to attend all appointments.

There are also some reasons why you might not be able to join. For example, if you have other serious health problems that might interfere with the study or the treatment. The doctors will carefully review your medical history to make sure joining is safe 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.

Are you between 18 and 65 years old?
Do you have a genetically confirmed diagnosis of FSHD1?
Can you walk without needing support?
Are you willing to maintain your current exercise level during the study?
Are you able to attend all scheduled study visits and follow instructions?
For women: Are you able to use reliable contraception if you could become pregnant?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you'll be given either the study medicine (Satralizumab) or a placebo (a dummy medicine) through an injection, which you'll give to yourself with a pre-filled syringe. You won't know which one you're receiving. You'll need to attend scheduled visits to the clinic for assessments, which will include physical examinations, blood tests, and MRI scans to check your muscles. You'll also need to keep track of your health and any changes you notice.

It's important that you commit to following the treatment plan, attending all appointments, and using adequate contraception if you are a woman of childbearing potential. The study expects you to maintain your usual level of exercise throughout. The total duration of your participation in the study, including all visits and follow-up, will be explained to you in detail by the study team.

Potential risks and benefits

Participating in this study might offer some potential benefits, such as contributing to medical knowledge about FSHD1 and potentially receiving a new treatment that could help reduce inflammation and improve your condition. However, there are also potential risks; you might experience side effects from the medication, or the experimental treatment might not be effective for you. You also might receive the placebo, which means you wouldn't get any active medication. The study team will explain all known side effects and risks. Remember, taking part in any clinical trial is always voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (2)

CHEO Research Institute Ottawa
Verified postcode
Ottawa, Canada
CHU de Nice
Verified postcode
Nice, France

Common questions

What is FSHD1?

FSHD1 is a type of muscular dystrophy that causes muscles to weaken, often starting in the face, shoulders, and upper arms.

What is Satralizumab?

Satralizumab is a new experimental medicine that aims to reduce inflammation in the body by blocking a specific signal called IL-6.

Will I get the actual medicine or a dummy treatment?

Participants will be randomly assigned to receive either Satralizumab or a placebo (a dummy treatment), and you won't know which one you are getting.

What is inflammation and why is it important for FSHD1?

Inflammation is your body's response to injury or irritation. Studies suggest it plays a role in how FSHD1 progresses, so reducing it might help.

What tests will be involved if I join?

You'll have physical exams, blood tests, and MRI scans to check your muscles and monitor your health throughout the study.