Active not recruitingOBSERVATIONAL

Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD

This study focuses on improving how new medicines for Facioscapulohumeral Muscular Dystrophy (FSHD) are tested. Recently, scientists discovered what causes FSHD, opening doors for new treatments. Many drug companies are now working on these. However, to speed up this process, we need better ways to measure how well these new drugs work and to pick the best participants for trials. Researchers in this study are developing new tools: a 'functional rating scale' to assess how FSHD affects daily life and a method called 'Electrical Impedance Myography' to look at muscle changes. They will test these on 320 patients across 14 international sites over two years, also looking at patient characteristics to help decide who can join future trials. The goal is to make drug development for FSHD faster and more effective.

At a glance

Status

Active not recruiting

Sponsor

University of Kansas Medical Center

Enrolment target

324

Start

05 Mar 2018

Estimated completion

01 Dec 2027

Facioscapulohumeral Muscular Dystrophy

What is this study about?

You might have heard about new research into Facioscapulohumeral Muscular Dystrophy (FSHD), which has given us a better understanding of what causes it. This is really exciting because it means drug companies can now design new treatments that target the root cause of the condition.

Right now, many companies are working hard to develop these new medicines. However, before these drugs can be used by everyone, they need to be thoroughly tested in clinical trials. A big challenge is making sure these trials are set up in the best possible way. This includes having good methods to measure if a new drug is actually working and making sure we choose the right people to participate in the trials so the results are clear.

This particular study isn't testing a new drug itself. Instead, it's about making future drug trials better and faster. Researchers are creating two new tools: one is a way to measure how FSHD affects your daily activities and another involves a special test called 'Electrical Impedance Myography' to check muscle changes. They also want to understand more about people with FSHD to help decide who can take part in future trials. By doing this, they hope to speed up the process of getting effective new treatments for FSHD to patients who need them.

Key takeaways

This study aims to make future FSHD drug trials better and faster.
Researchers are developing new tools to measure FSHD's effects.
It will involve 320 patients over two years across several locations.
This study itself does not involve taking or testing a new medication.
Participation helps scientists understand FSHD better for future treatments.

Who may be eligible?

To be considered for this study, you would need to be aged between 18 and 75.

You would generally need to have a confirmed diagnosis of FSHD, either through a genetic test or based on your symptoms and a family history. You should also have some weakness in your limbs due to the condition, but still be able to walk about 10 meters (roughly 30 feet) without needing someone else's help or a walking frame (canes or braces are okay).

You wouldn't be able to join if you have serious heart or breathing problems, or other conditions that would make muscle function tests unsafe. Also, if you regularly use certain muscle-affecting medications, or have taken part in another FSHD drug trial in the last month, or are pregnant, you wouldn't be eligible. If you take over-the-counter supplements, you would need to be willing to keep your regimen consistent throughout the study.

Quick self-check

Are you between 18 and 75 years old?
Do you have a confirmed diagnosis of FSHD?
Can you walk about 10 metres (30 feet) without someone else's help or a walking frame?
Are you generally free from major heart or breathing problems?
Are you not currently pregnant?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

This study is not testing a new medication, so you won't be given any experimental drugs. Instead, it involves several visits over two years to assess your FSHD. Researchers will use the new FSHD-specific functional rating scale to understand how the condition affects your daily life. You'll also likely undergo Electrical Impedance Myography (EIM), a gentle test that assesses your muscle health. The study involves collecting information about your condition to help design better clinical trials for the future. You'll need to attend follow-up appointments over the 24-month period.

Potential risks and benefits

While this study doesn't offer direct therapy, a potential benefit is contributing valuable information that could speed up the development of effective treatments for FSHD for everyone in the future. The risks of participation are likely to be low, mainly from the procedures themselves, which include assessing your muscle function and possibly some gentle electrical tests, similar to what you might experience with an ECG. You are free to stop participating in the study at any time, for any reason, without it affecting your medical care.

Locations (14)

University of California Los Angeles
Los Angeles, United States
University of Kansas Medical Center
Kansas City, United States
Kennedy Krieger Institute
Baltimore, United States
University of Rochester Medical Center
Rochester, United States
The Ohio State University
Columbus, United States
University of Utah
Salt Lake City, United States
Virginia Commonwealth University
Richmond, United States
University of Washington
Seattle, United States
Chu De Nice
Nice, France
Institut de Myologie
Paris, France
Ludwig-Maximilians-Universität München
München, Germany
Centro Clinico NeMO
Milan, Italy

+2 more sites — see the official record for the full list.

Common questions

Is this study testing a new drug for FSHD?

No, this study is not testing a drug. It's focused on creating better ways to measure FSHD and plan future drug trials to make them more effective.

What methods are they using to measure FSHD?

They are developing a 'functional rating scale' to see how FSHD affects your daily life, and a technique called 'Electrical Impedance Myography' to look at muscle changes.

How long will the study last if I take part?

The study is planned to last for 24 months, which is two years.

Will I need to change my current medications or supplements?

You wouldn't need to change any prescribed medications. If you take over-the-counter supplements, you'd need to agree to keep taking them consistently throughout the study, without changing the amount or type.

Will I receive any direct medical benefits from participating?

You won't receive a new treatment or direct medical benefit. However, your participation will greatly help in speeding up the development of new treatments for FSHD in the future.