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|
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
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.
This was an open-label, single and multiple oral dose study in patients with DMD.
To determine the safety and tolerability of single and multiple doses of the drug (SMT C1100).
To determine pharmacokinetics of the drug and its metabolites for single and multiple doses.
Up to 12 patients with DMD.