Queen Square Centre for Neuromuscular Diseases


A study of SMT C1100 in children with Duchenne muscular dystrophy (DMD) who follow a balanced diet.

Sponsor Summit Corporation plc
PI Francesco Muntoni

Newcastle: becky.davis@ncl.ac.uk

London: c.uzowuru@ucl.ac.uk

More information

Trial information

This is a randomised double-blind placebo control study of the drug SMT C11003.

Trial design

Boys will be put in three different treatment sequence groups. Each patient will receive two doses of the drug (increased amounts per dose) with a placebo also being used. The length of each study depends on which treatment the patient will receive.

Primary objective

To find out the required dose of the drug in patients with a balanced diet.