Active not recruitingOBSERVATIONAL

Defining Endpoints in Becker Muscular Dystrophy

This study is called 'Defining Endpoints in Becker Muscular Dystrophy'. It's an observation-only study, meaning participants won't receive new treatments, but will be carefully monitored over 24 months. The main goal is to understand how Becker muscular dystrophy (BMD) affects people differently and how it progresses over time. BMD is a condition that causes muscles to weaken, but how quickly this happens varies greatly from person to person. By gathering detailed information from 50 participants, researchers hope to get a clearer picture of this progression. This knowledge is crucial for planning and testing new medicines for BMD more effectively in the future, helping to ensure that new treatments can be properly evaluated.

At a glance

Status

Active not recruiting

Sponsor

Virginia Commonwealth University

Enrolment target

Start

13 Apr 2022

Estimated completion

01 May 2026

Becker Muscular Dystrophy Muscular Dystrophies Muscular Dystrophy in Children Muscular Dystrophy, Becker

What is this study about?

Becker muscular dystrophy (BMD) is a condition that causes progressive weakness in the muscles. It's caused by changes in a specific gene, but the effects can be very different from one person to another. Some people might notice symptoms when they're young, while for others, it might not be until much later in life. This wide range in how the condition progresses makes it difficult for researchers to test new treatments.

This study aims to get a better understanding of how BMD changes over time in different people. By carefully observing 50 individuals with BMD for two years, researchers hope to identify specific milestones or 'endpoints' that show how the disease is progressing. This will create a very detailed picture of the natural course of the condition.

Why is this important? Right now, because BMD affects people so differently, it's hard to tell if a new treatment is really working or if the changes are just part of the natural variation of the disease. By understanding the natural progression better, future studies testing new medicines will be much more effective. This study doesn't involve any new medicines; it's purely about watching and learning from people living with BMD to improve how treatments are developed.

Key takeaways

This is an observation-only study, no new medications.
The study lasts for 24 months (two years).
Aims to understand how Becker muscular dystrophy progresses.
Information gathered will help future treatment development.
Open to males aged 6 and above with confirmed BMD.
Participation includes regular clinic visits and assessments.

Who may be eligible?

This study is looking for males with Becker muscular dystrophy, aged 6 years and older. To be considered, you must have a clear diagnosis of BMD, confirmed by a genetic test showing a particular type of change in the dystrophin gene. You also need to be able to understand the study and agree to take part, and be able to follow all the study's requirements.

There are some specific requirements based on age. For those aged 6 to 12, you must still be able to walk independently. For those 13 and older, most participants will still be walking, but a small number who no longer walk might be included. There are also specific rules about certain medications like steroids, and any other health conditions that might make it unsafe to take part or make the study results unclear.

In some cases, participants might also be asked to take part in an additional MRI scan. If this is the case, you'll need to be able to walk, and you can't have anything in your body (like certain medical devices) that would prevent you from having an MRI, or suffer from severe claustrophobia.

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 male?
Are you aged 6 years or older?
Do you have a confirmed diagnosis of Becker muscular dystrophy with a specific genetic change?
For ages 6-12, can you still walk independently?
Are you able to understand the study and give your permission to take part?

Answer every question to see your result.

What does participation involve?

If you join this study, you will be observed for a period of 24 months, which is two years. This is an observational study, meaning you won't be given any new medication or experimental treatments. Instead, the research team will regularly check your health and how your Becker muscular dystrophy is progressing.

Participation will involve regular visits to the study clinic, where various assessments will be carried out. These might include physical tests to check your muscle strength and movement, and other standard health checks. Some participants might also have MRI scans as part of the study. The study doesn't specify the exact number of visits, but it will be spread out over two years. You'll be asked to follow all the study procedures throughout this time.

Potential risks and benefits

The main benefit of taking part in this study is contributing to a better understanding of Becker muscular dystrophy. This knowledge is vital for helping researchers design more effective clinical trials for new treatments in the future, potentially benefiting many people with BMD. As this is an observational study, there are no direct medical benefits like receiving an experimental treatment. The risks are generally low, mainly involving the time commitment for visits and the discomfort associated with physical assessments or potential MRI scans. However, all procedures will be explained in detail, and your safety is a priority. Remember, participating is always voluntary, and you are free to withdraw from the study at any time without giving a reason.

Locations (11)

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

University of California, Irvine
Verified postcode
Orange, United States
University of Colorado Anschutz Medical Campus
Verified postcode
Aurora, United States
University of Iowa
Verified postcode
Iowa City, United States
University of Kansas Medical Center
Verified postcode
Kansas City, United States
Kennedy Krieger Institute
Verified postcode
Baltimore, United States
University of Minnesota
Verified postcode
Minneapolis, United States
Washington University School of Medicine
Verified postcode
St Louis, United States
Nationwide Children's Hospital
Verified postcode
Columbus, United States
Virginia Commonwealth University
Verified postcode
Richmond, United States
University of Auckland
Verified postcode
Auckland, New Zealand
John Walton Muscular Dystrophy Research Centre
Verified postcode
Newcastle upon Tyne, United Kingdom

Common questions

What is an 'observational study'?

An observational study means doctors will watch and learn about your condition without giving you any new medicines or treatments. They will just collect information.

Will I receive new medicine in this study?

No, this study does not involve giving new medicines. It's about understanding how Becker muscular dystrophy progresses naturally.

How long will I need to be in the study?

You will be part of this study for 24 months, which is two years.

Why is this study important?

It's important because understanding how Becker muscular dystrophy changes over time will help researchers develop and test new treatments more effectively in the future.

Will I need to visit a clinic often?

Yes, you will have regular visits to the clinic over the two years for check-ups and assessments.