Understanding Duchenne muscular dystrophy in males unable to walk

Start Date
01/03/2010
Expected end Date
01/10/2012
Location
USA
Status
Recruiting

What is the aim of the trial?

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

This study aims to provide a better understanding of disease progression in boys and men diagnosed with Duchenne muscular dystrophy who are unable to walk without assistance. This information is crucial for the design of future clinical trials for this patient group.

Most of the clinical trials for Duchenne muscular dystrophy do not accept individuals with Duchenne muscular dystrophy who are unable to walk without assistance. One reason for this is that currently the standard way to assess whether a treatment is working is to measure how fast the trial participants can walk.

Therefore, new ways to assess whether a treatment is working are required for those patients with Duchenne muscular dystrophy who are full-time wheelchair users. To develop measures of quality of life and muscle strength for clinical trials in this patient group, an understanding of the natural disease progression is needed, and this study aims to gather this information.

Who can be involved in the trial?

This trial aims to recruit males between the ages of seven and 22 with Duchenne muscular dystrophy who have been unable to walk without assistive devices such as crutches for at least one year.

What happens during the trial?

If necessary a blood sample or biopsy will be taken to confirm the diagnosis of Duchenne muscular dystrophy before enrolling in the study. Participants will be observed for the progression of the disease for a three year period and any clinical complications such as weakening of the heart muscle will be reported.

Where is the study taking place?

Washington University, St. Loius, Missouri, 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?

This study could gather information to enable future clinical trials to involve patients with more advanced stages of Duchenne muscular dystrophy who are currently unable to participate in most trials. This would mean that more patients could benefit from any successful new treatments.

Contact Details:

Pallavi Anand

Tel: +1 314 362 2490

Email: anandp@neuro.wustl.edu

Who is funding this study?

  • Washington University School of Medicine

Official name of the Trial:

Clinical outcomes validation in non ambulatory and young boys/men with Duchenne muscular dystrophy (DMD)

Trial study number:

NCT01098708

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.

www.clinicaltrials.gov/show/NCT01098708

Background information and related links:

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

This study aims to provide a better understanding of disease progression in boys and men diagnosed with Duchenne muscular dystrophy who are unable to walk without assistance. This information is crucial for the design of future clinical trials for this patient group.

Most of the clinical trials for Duchenne muscular dystrophy do not accept individuals with Duchenne muscular dystrophy who are unable to walk without assistance. One reason for this is that currently the standard way to assess whether a treatment is working is to measure how fast the trial participants can walk.

Therefore, new ways to assess whether a treatment is working are required for those patients with Duchenne muscular dystrophy who are full-time wheelchair users. To develop measures of quality of life and muscle strength for clinical trials in this patient group, an understanding of the natural disease progression is needed, and this study aims to gather this information.

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