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Study of new treatment ACE-031 in subjects with Duchenne muscular dystrophy

  • Condition: Duchenne muscular dystrophy
  • Start Date: April 2010
  • Expected End Date: February 2012
  • Status: Terminated
  • Location of trial: North America

What is the aim of the trial?

The aim of this study is to see if ACE-031 is safe and well tolerated in children with Duchenne muscular dystrophy. In addition, the study will characterise the effects of ACE-031 on lean tissue (muscle) mass, muscle strength, muscle function, lung function and quality of life. ACE-031 is a new molecule which has been developed to help treat muscle weakness and deterioration, a symptom of many neuromuscular diseases including muscular dystrophy.

ACE-031 works by blocking the action of certain proteins in the body such as myostatin which work as natural ‘brakes’ to limit muscle growth and size. When these substances are prevented from acting on muscle cells by ACE-031, the muscle is able to grow larger and stronger. It is hoped ACE-031 will help maintain or improve muscle in patients with muscular dystrophy.

This early phase 2 trial will look at up to four different doses of ACE-031 to try and find out which is the best dose to use in further trials. The most important aim of this trial is to monitor safety and tolerability of ACE-031 in patients, and to measure how well the molecule is handled by the body. However some measurements will also be done to look for any potential changes in muscle size and strength.

Who can be involved in the trial?

This trial is for boys of 4 years or older who have been diagnosed with Duchenne muscular dystrophy. The patient must be able to walk independently and must already be showing signs of muscle weakness. The patient can only be included in the trial if he has had steroid treatment for at least one year before the study and has been on a stable steroid dose for at least 6 months before the start of the study. If the patient has already taken part in another trial up to 6 months before, or has any other medical problems which are deemed incompatible with this trial then they will not be able to enroll.

A maximum of 76 patients will be recruited. For more details of who can participate in the trial, see ‘Further trial details’ below.

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

What happens during the trial?

Patients entered into the trial will be randomly assigned to receive either ACE-031 or placebo; patients will have a 1 in 4 chance of getting placebo. They will be given an injection of ACE-031 or placebo every 4 weeks for 12 weeks (4 doses). There may be up to 4 groups at 4 different dosing levels. Results will be measured over 6 months.

Safety and tolerability of ACE-031 will be monitored at regular intervals over the length of the trial, including for example, blood tests, blood pressure, pulse and electrocardiograms and other methods of checking the heart. Antibody levels in the blood will be measured to monitor how well the immune system is tolerating ACE-031. A detailed record of anything which could be a treatment side effect will also be kept.

To measure any relative muscle size changes MRI scans of the thighs and a type of x-ray called a ‘whole body dual x-ray absorptiometry scan’ will be used. Changes in muscle function and strength, and also lung function will be measured.

Where is the study taking place?

This trial is taking place in six to eight study sites across Canada. The coordinating site is at the London Health Sciences Centre in Ontario Canada.

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?

It is hoped that ACE-031 can help to slow down or prevent some of the muscle deterioration which happens with Duchenne muscular dystrophy.

This treatment has already been shown to increase amounts of muscle in both healthy animals and animal models of muscle wasting diseases. ACE-031 has also shown encouraging results in early trials in healthy humans; there was an increase in muscle two weeks after one injection of ACE-031.

This trial will now help to determine if ACE-031 is safe for use in patients with Duchenne muscular dystrophy and will produce some of the first results showing if the molecule is effective in maintaining muscle in these patients. It is also hoped results from this trial can be used to define the best dose of ACE-031 to use in other larger trials.

Contact Details:

Rhiannon Taranik
Tel: +1 519 685 8441
Email: Rhiannon.Taranik@lhsc.on.ca

Who is funding this study?

Official name of the Trial:

A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ACE-031 (ActRIIB-IgG1) in Subjects With Duchenne Muscular Dystrophy

Trial study number:


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.


Link to terms in the glossary:

Background information and related links:

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