Modified Diet Trial: A Study of SMT C1100 in Paediatric Patients With DMD Who Follow a Balanced Diet

Summary

Placebo-controlled, multi-centre, randomized, double-blind dose escalation study. The aim is to evaluate the pharmacokinetics (PK) and safety of SMT C1100 in paediatric patients with Duchenne Muscular Dystrophy (DMD) who follow a balanced diet.

Timeline

Start

2015-02

Primary completion

2015-08

Completion

2015-08

Publications

• Ricotti V, Spinty S, Roper H, Hughes I, Tejura B, Robinson N, Layton G, Davies K, Muntoni F, Tinsley J. Safety, Tolerability, and Pharmacokinetics of SMT C1100, a 2-Arylbenzoxazole Utrophin Modulator, following Single- and Multiple-Dose Administration to Pediatric Patients with Duchenne Muscular Dystrophy. PLoS One. 2016 Apr 7;11(4):e0152840. doi: 10.1371/journal.pone.0152840. eCollection 2016.

Drugs

Indications