Clinical trial being planned for congenital muscular dystrophy

Published Date
Kristina Elvidge

A clinical trial to test a drug called omigapil in Ullrich congenital muscular dystrophy, Bethlem myopathy and merosin-deficient congenital muscular dystrophy (MDC1A) is being planned for 2012.

Omigapil is an oral drug which was originally developed for the treatment of neurological disorders including Parkinson’s disease and amyotrophic lateral sclerosis (ALS). In 2007 Santhera Pharmaceuticals obtained a licence  to develop omigapil for neuromuscular conditions. Testing in a mouse model of congenital muscular dystrophy has shown that this compound may be able to reduce the severity of symptoms of this condition.

The trial organisers are planning to test omigapil in patients with several different types of congenital muscular dystrophy. Some of the conditions are collectively known as the ‘collagen VI-related myopathies’ and are caused by mutations in collagen VI genes. This includes Ullrich congenital muscular dystrophy, intermediate collagen VI myopathy and Bethlem myopathy.  The other condition to be included is merosin-deficient congenital muscular dystrophy (MDC1A) which is caused by mutations in the laminin alpha 2 gene.

This first clinical trial of omigapil for congenital muscular dystrophy  is known as a ‘pharmacokinetic study,’ which means that the trial aims to measure how much of the drug reaches the blood stream, which may help to determine the optimal dose for a subsequent clinical trial. Assessing participants for evidence of  short-term side effects of this compound will be a primary goal of this study.  

A small number of patients will participate in this trial which is proposed to be conducted at two study sites, one in the UK: Great Ormond Street Hospital (London)  and the other in the USA: National Institute of Neurological Disorders and Stroke/National Institute of Health  (Bethesda, Maryland).

The Muscular Dystrophy Campaign is supporting the planning of this clinical trial by funding Dr Reghan Foley, a Clinical Research Fellow at the Dubowitz Neuromuscular Centre. Dr Foley has helped to coordinate international collaborations for studying ways of measuring potential effects of treatments in individuals with collagen VI myopathies. This is essential for future clinical trials. We also fund a clinical trial coordinator at the centre who will be involved in setting up the clinical trial. 

There is an international patient registry for individuals with congenital muscular dystrophies and myopathies called the CMD International Registry (CMDIR). To register for the CMDIR, patients and/or family members are asked to answer questions in a secure online form. The information collected through this registry is essential both for achieving a better understanding of the congenital muscular dystrophies and congenital myopathies  as well as for contacting patients and families who may be eligible for and interested in participating in clinical trials. Another aim of the registry is to provide access to the latest information about international clinical trials. Find out more about the registry here.

Further information and links

Read more about the preclinical research into omigapil.

Join the discussion about this trial on our forum TalkMD.

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