Active not recruitingNAINTERVENTIONAL

Clinical Trial Readiness Network FSHD France: Prospective 24 Months MRI Study

This study is looking at new ways to measure how Facioscapulohumeral Muscular Dystrophy (FSHD) affects people over time, using MRI scans and other tests. The main goal is to speed up the development of new treatments for FSHD. Researchers want to create better tools to see if new medicines are working. They will follow 30 people with FSHD for 24 months to understand how the disease progresses and how different tests can show these changes. This information will help drug companies design better clinical trials and make sure the right people are included, bringing new therapies closer to those who need them. It's about laying the groundwork for future medical breakthroughs in FSHD.

At a glance

Status

Active not recruiting

Phase

Sponsor

Centre Hospitalier Universitaire de Nice

Enrolment target

100

Start

16 Sep 2019

Estimated completion

03 Sep 2026

Muscular Dystrophy Facioscapulohumeral

What is this study about?

This study is important for people living with Facioscapulohumeral Muscular Dystrophy (FSHD). FSHD is a condition that causes muscles to weaken over time. Researchers have recently learned a lot about what causes FSHD at a genetic level. This new understanding means that exciting new treatments are being developed to target the root cause of the disease. However, before these new medicines can be widely used, they need to be tested thoroughly in clinical trials.

One of the biggest challenges in running these trials is having clear and reliable ways to measure if a new treatment is actually helping. Imagine trying to test a medicine, but not having a good way to tell if a person's muscles are getting stronger or if the disease is slowing down. This study aims to create and test new tools for measuring these changes. They will be looking at things like how well muscles work and using special scans (MRI) to see muscle health.

By carefully following people with FSHD for 24 months, the study hopes to create better ways for doctors to track the disease. This will help future clinical trials be more effective, allowing scientists to see more clearly if new drugs are working. Ultimately, this research will help speed up the process of getting new and effective treatments for FSHD into the hands of patients. It's all about building a stronger foundation for future medical progress.

Key takeaways

This study helps develop better ways to measure FSHD.
It aims to speed up finding new treatments for FSHD.
Participation involves regular visits and assessments over 24 months.
You won't receive new medication; it's an observational study.
Your involvement could help many others with FSHD in the future.

Who may be eligible?

This study is looking for adults aged 18 to 75 who have a confirmed diagnosis of FSHD. You need to have some muscle weakness in your limbs, but you should still be able to walk, either on your own or with a walking aid. Your leg muscles also need to have a certain level of strength.

There are also some reasons why you might not be able to join. For example, if you have severe heart or breathing problems, or other serious health conditions that might make testing unsafe. If you're pregnant, or if you're taking certain medications like strong steroids or testosterone, you wouldn't be able to take part. It's also not suitable if you've been in another FSHD drug trial recently, or if you can't have an MRI scan.

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 75 years old?
Do you have a confirmed diagnosis of FSHD?
Do you have some muscle weakness but can still walk (with or without aid)?
Do you have basic social security coverage?
Are you able to have an MRI scan?
Are you able to commit to 24 months of study visits?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would visit Nice University Hospital periodically over 24 months. During these visits, the research team would conduct various tests. This might include checking your muscle strength, assessing how you move, and performing special MRI scans to look at your muscles. They will also collect information about your medical history and any supplements you might be taking. The study doesn't involve any new medication, but focuses on thoroughly assessing your FSHD over time. The total duration of your participation would be 24 months.

Potential risks and benefits

The main benefit of taking part is that you would be contributing significantly to our understanding of FSHD, which could lead to better treatments for everyone in the future. You'd also receive regular assessments of your condition by expert doctors. As for risks, you might feel a bit tired or have some minor discomfort from the exercises and tests. There's also the standard safety aspect of MRI scans, which the team will discuss with you. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (3)

CHRU de Lille
Verified postcode
Lille, France
CHU de Nice
Verified postcode
Nice, France
Myology institute Association
Verified postcode
Paris, France

Common questions

What is FSHD?

FSHD stands for Facioscapulohumeral Muscular Dystrophy. It's a genetic condition that causes muscles to weaken over time, particularly in the face, shoulders, and upper arms, but can affect other parts of the body too.

Why is this study important for FSHD patients?

This study is crucial because it's helping researchers find better ways to measure how FSHD affects people. This will make it easier to test new medicines and find out if they are working, ultimately speeding up the development of new treatments.

Will I receive any new medicine in this study?

No, this study is about observing and measuring FSHD progression, not testing a new drug. You won't be given any experimental medication as part of this research.

How long will I be in the study if I participate?

If you join, you would be part of the study for 24 months, attending regular appointments at Nice University Hospital.

What are 'clinical outcome assessments'?

These are simply the ways doctors and researchers measure if a disease is getting better, worse, or staying the same. For FSHD, it could involve tests of muscle strength, walking ability, or special scans.