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Testing non-invasive imaging techniques to measure the effect of exon skipping drugs

  • Condition: Duchenne muscular dystrophy
  • Start Date: September 2011
  • Status: Active but not recruiting
  • Location of trial: North America

What is the aim of the trial?

This study is an observational study and no new or experimental drugs will be given to the participants.

The aim of this study is to test whether images taken using non-invasive techniques could be used to measure changes in muscles. These changes could show if a new drug is having a positive effect.

This study is part of a larger project which is testing whether a new drug (called GSK2402968) could treat Duchenne muscular dystrophy. GSK2402968 uses a technique called exon skipping – where small pieces of DNA hide the mutated part of the dystrophin gene – to restore the production of the dystrophin protein. It is hoped this will help to prevent muscle damage occurring.

To find out if the new drug works, researchers need to be able to measure the effects of the drugs in the muscle. One way to do this is to take muscle biopsies. This is an invasive procedure, however, and can be very uncomfortable for participants, so it is hard to take multiple samples from a muscle. This makes it difficult to detect changes that are happening over a long time as a result of drug treatment.

The researchers will use magnetic resonance imaging (MRI) and ultrasound to take images of different muscles in the body – including the heart, arms and legs. They will examine the images for changes – called biomarkers – that could show if there has been an improvement or worsening in the muscle. By comparing the images from healthy participants with those from people with Duchenne muscular dystrophy, researchers will be able to find out which method of taking images works best. This information can then be used to test how well the new exon skipping drug works.

Who can be involved in the trial?

The researchers aim to recruit 85 boys aged between five and 17. Participants must have Duchenne muscular dystrophy and must already be enrolled in the exon skipping drug (GSK2402968) trial. The researchers also aim to recruit healthy boys of a similar age who have no muscle disease.

All participants must be able to cope with being put in a magnetic resonance imaging (MRI) scanner without sedation, so people who fear small spaces will not be able to take part.

For more information about how to get involved in trials please read our FAQ.

What happens during the trial?

Participants will be given three tests which will take about two hours in total. The tests will include an MRI scan of the skeletal, heart, and diaphragm (breathing) muscles; and an ultrasound examination of the leg and arm muscles. Healthy participants will take these tests once, whilst participants with Duchenne muscular dystrophy (from the exon skipping drug trial) will take the tests four times:
• at the beginning of the study
• 3 months after starting treatment
• 6 months after starting treatment
• 6 months after finishing treatment

Where is the study taking place?

The study is taking place at the National Institutes of Health Clinical Center in Bethesda, Maryland (USA).

In most circumstances, for somebody to participate in a clinical trial, they need to live near the team of people who are conducting the research, because they need to be closely monitored.

How could the results of the trial benefit patients?

Although this trial will not offer new drugs or treatments to participants, it is part of a larger trial – which aims to test whether a new exon skipping drug can treat Duchenne muscular dystrophy. This trial is an important step for that study because it will tell researchers the best ways to measure changes in the muscles that will indicate whether or not the drug is having an effect.

The information collected in this study will also be useful for future trials into treatments for Duchenne muscular dystrophy as researchers may be able to use the same techniques to detect changes in the muscle. The knowledge gained from this study may also provide a method by which clinicians can monitor progression of the condition over time giving them more detailed information about how the disease is progressing in their patients.

Contact Details:

Alice B Schindler
National Institutes of Health, Maryland, USA
Tel: +1 301 496 8969
Email: schindlerab@mail.nih.gov

Who is funding this study?

  • National Institute of Neurological Disorders and Stroke (NINDS).

Official name of the Trial:

Evaluation of Skeletal Muscle, Cardiac, and Diaphragm Imaging Biomarkers for GSK2402968 Effects in Ambulatory Boys With Duchenne Muscular Dystrophy.

Trial study number:

NCT01451281

Further trial details:

For further information on the trial and detailed inclusion and exclusion please click on the link below. Sometimes these details can be quite technical. If you have any questions please discuss this with your clinician or contact the clinical trial organisers.

ww.clinicaltrials.gov/show/NCT01451281

Link to terms in the glossary:

Background information and related links:

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