RecruitingOBSERVATIONAL

Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD)

The MOVE FSHD study wants to learn more about how Facioscapulohumeral Muscular Dystrophy (FSHD) impacts movement and daily activities. By carefully observing and measuring how people with FSHD change over a period of time, researchers hope to improve the quality of care available and help develop new medications faster. They also want to figure out what level of change in movement is truly important to people living with FSHD. Around 450 people with FSHD will be involved for at least three years. A smaller group, about 200 participants, will also take part in a related study called MOVE+, which involves more detailed scans and movement assessments, and possibly muscle biopsies or wearable devices if in the US.

At a glance

Status

Recruiting

Sponsor

University of Kansas Medical Center

Enrolment target

450

Start

15 Dec 2020

Estimated completion

01 Jan 2028

FSHD

What is this study about?

The MOVE FSHD study aims to understand how Facioscapulohumeral Muscular Dystrophy (FSHD) affects movement and daily life over time. FSHD is a condition that causes muscle weakness. By carefully tracking changes in how people move and perform everyday tasks, researchers hope to achieve two main goals. Firstly, this information will help doctors provide the best possible care for individuals with FSHD. Secondly, a clearer understanding of how the condition progresses physically will assist in developing new treatments more quickly.

Researchers will be looking at how FSHD impacts various aspects of life, including motor function (how you move), breathing, and if or when someone might start using a wheelchair. They also want to understand what kind of improvement in movement would be considered truly meaningful and helpful to someone living with FSHD. This is important for designing future drug trials and understanding if a new treatment is really making a difference to patients.

Around 450 people with FSHD will take part in the main MOVE FSHD study for at least three years. A subgroup of about 200 participants will also join a related study called MOVE+. This part of the study involves more detailed assessments, such as whole body MRI scans to look at muscles, and tests to measure how far you can reach. For participants in the US, there might also be options for muscle biopsies (taking a small sample of muscle tissue) and using wearable devices to track activity.

Key takeaways

A research study to understand how FSHD affects movement over time.
Aims to improve patient care and speed up treatment development for FSHD.
Involves regular visits over at least three years.
No new medications are given; it's an observational study.
Looking for diverse participants, including both adults and children with FSHD.

Who may be eligible?

To be considered for the MOVE FSHD study, you must have a confirmed diagnosis of Facioscapulohumeral Muscular Dystrophy (FSHD). This means you either have genetic proof (such as a blood test showing the specific gene changes for type 1 or type 2 FSHD) or you have been diagnosed by a doctor based on typical symptoms and either a parent or a child also has FSHD.

There are also some reasons why you might not be able to join. For example, if you are unable or unwilling to give your informed consent, which means understanding what the study involves and agreeing to take part freely. Also, if you have any other health condition that your study doctor believes would make it difficult or unsafe for you to participate fully in the study, you might not be eligible.

This study welcomes adults and children of all ages, and both men and women can participate. The most important factor for eligibility is a clear diagnosis of FSHD.

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 a genetically confirmed diagnosis of FSHD (type 1 or 2)?
Or, do you have a clinical diagnosis of FSHD with typical symptoms and an affected parent or child?
Are you able and willing to understand and give your consent to participate?
Do you have any other medical conditions that might make it difficult to take part?

Answer every question to see your result.

What does participation involve?

If you take part in the MOVE FSHD study, you will be followed for a minimum of three years. This means you will have regular visits to the study centre over this period. During these visits, researchers will perform various assessments to track how your FSHD is affecting your movement and other health aspects. These assessments will help them understand the progression of the condition over time.

For those who join the MOVE+ sub-study, there will be additional tests. These may include whole-body MRI scans, which are detailed pictures of your muscles, and tests to measure your 'reachable workspace' – how far you can comfortably move your arms. If you are in the US, there might also be an option to have a muscle biopsy (a small sample of muscle tissue taken) and to wear a device that tracks your activity.

The study does not involve taking any new medications, as its main goal is to observe and understand the natural progression of FSHD. You will continue to receive your usual medical care from your own doctors throughout your participation.

Potential risks and benefits

Participating in this study might not directly benefit you, as it's an observational study focused on understanding FSHD, not testing a new treatment. However, the information gathered will be incredibly valuable in helping researchers understand FSHD better, improve care for future patients, and speed up the development of new treatments. The main risks involve the time commitment for study visits and potential discomfort from procedures like MRI scans or, if applicable, muscle biopsies. All procedures will be explained in detail, and you'll have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (21)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

David Geffen School of Medicine at UCLA
Verified postcode
Los Angeles, United States· Recruiting
Neuromuscular Disorders Program at Stanford University School of Medicine
Verified postcode
Stanford, United States· Recruiting
University of Colorado Anschutz Medical Campus
Verified postcode
Aurora, United States· Recruiting
Univeristy of Florida Gainesville
Verified postcode
Gainesville, United States· Recruiting
University of Iowa
Verified postcode
Iowa City, United States· Recruiting
Univeristy of Kansas Medical Center
Verified postcode
Kansas City, United States· Recruiting
Kennedy Krieger Institute
Verified postcode
Baltimore, United States· Recruiting
University of Rochester Medical Center
Verified postcode
Rochester, United States· Recruiting
Duke University - Lenox Baker Children's Hospital
Verified postcode
Durham, United States· Recruiting
The Ohio State University Medical Center
Verified postcode
Columbus, United States· Recruiting
Austin Neuromuscular Center
Verified postcode
Austin, United States· Recruiting
Univeristy of Texas Southwestern Medical Center
Verified postcode
Dallas, United States· Recruiting

Common questions

What is the main goal of the MOVE FSHD study?

The main goal is to understand how FSHD affects movement and daily activities over time, to improve care and help develop new treatments.

How long will I need to be in the study?

If you participate, you will be followed for at least three years, with regular visits during that time.

Will I be given new medication as part of this study?

No, this study is about observing FSHD progression, not testing new medications. You will continue with your usual medical care.

What is the MOVE+ sub-study?

MOVE+ is a part of the study with more detailed tests, like MRI scans and movement assessments. Some US participants might also have optional muscle biopsies or wear tracking devices.

Can children participate in this study?

Yes, children of all ages are welcome to participate, as long as they meet the other eligibility criteria.

How to find out more

Michaela Walker, MPH

mwalker20@kumc.edu 913-945-9920 Official trial record

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