Clinical trial for Congenital MD

Swiss pharmaceutical company Santhera has announced that it will soon start a clinical trial of a drug called omigapil in children with congenital muscular dystrophy (CMD).

The study (called CALLISTO) will take place in the USA and will assess the safety, tolerability and pharmacokinetics of omigapil. Pharmacokinetics involves measuring how much of the drug reaches the blood stream, which will help to determine the optimal dose for a subsequent clinical trial.

Twenty participants, aged from five to 16 years of age with Ullrich CMD or merosin-deficient CMD (MDC1A) will take part in the trial for 12 weeks. Recruitment is expected to start later this year and further information about the trial is available on the website.

Omigapil is an oral drug which was originally developed for the treatment of neurological disorders including Parkinson’s disease and amyotrophic lateral sclerosis (ALS). It is thought to work by preventing cells dying. Omigapil does not target the primary genetic cause of CMD and so it cannot be considered a ‘cure’, but testing in a mouse model of MDC1A has shown that it may be able to reduce the severity of symptoms.

Further information

• Read Santhera’s press release
• The MDA Ullrich CMD factsheet contains more information about the condition including a research summary
• Information about the Congenital Muscle Disease International Registry (CMDIR) which will be used to recruit patients to the trial. Further information about patient registries is available on the MDA website.
Clinical trials – your questions answered
• You can get regular updates by becoming a friend of the MDA Facebook page

If you have any questions, please contact us:

Phone: +61 3 9320 9555

Uploaded 2 July 201