Queen Square Centre for Neuromuscular Diseases



SMT C1100 - A Phase I, Open-label, Single and Multiple Oral Dose, Safety, Tolerability and Pharmacokinetic Study in Paediatric Patients with Duchenne Muscular Dystrophy

Sponsor / Funder Summit
CI Professor Francesco Muntoni
sites London GOSH
More information


SMT C1100 is a new oral drug that acts to modulate transcriptional control of utrophin. SMT C1100 has the potential to treat DMD independent of the dystrophin mutation.

It maintains production of utrophin to compensate, at least in part, for the loss of the dystrophin protein. Results from laboratory studies showed that SMT C1100 increases utrophin levels and improves muscle structure and function.

Trial information

This was an open-label, single and multiple oral dose study in patients with DMD.

Primary Objective

To determine the safety and tolerability of single and multiple doses of the drug (SMT C1100).

Secondary objectives

To determine pharmacokinetics of the drug and its metabolites for single and multiple doses. 


Up to 12 patients with DMD.