A study to test the safety of the SMT C1100 in boys with DMD.

• Research type

  Research Study

• Full title

  SMT C11003 - A Phase 1b placebo-controlled, multi-centre, randomized, double-blind 3-period dose escalation study to evaluate the pharmacokinetics (PK) and safety of SMT C1100 in paediatric patients with Duchenne Muscular Dystrophy (DMD) who follow a balanced diet.

• IRAS ID

  163972

• Contact name

  Francesco Muntoni

• Contact email

  f.muntoni@ucl.ac.uk

• Sponsor organisation

  Summit (Oxford) Limited

• Eudract number

  2014-003100-78

• Duration of Study in the UK

  0 years, 4 months, 0 days

• Research summary

  Duchenne muscular dystrophy (DMD) is a progressive, lethal disease with an estimated incidence of between 1 in 3500 and 1 in 5000 live male births. As the disease progresses, the muscle weakness extends and ultimately cardiac and respiratory difficulties present, leading to death. Due to recent medical advances in the treatment of respiratory infections, surgery for curvature of the spine and methods of assisted ventilation, life expectancy has extended to the middle-late twenties.

  SMT C1100 is the first in a new pharmacological class of orally available utrophin (a protein involved in muscle function) modulators which in non­clinical studies demonstrated improvement of muscle structure and function.

  This Phase 1, study has been designed to determine the safety and tolerability of single and multiple oral doses of SMT C1100 and its metabolites in male children aged 5 to 13 years with DMD who follow a balanced diet.

  Up to 12 patients will be equally randomized to one of 3 treatment groups. Each patient will receive both doses of SMT C1100 (1250 mg and 2500 mg) in a dose escalating fashion, with placebo in the other study period.

  The expected duration of each patient on the study will be approximately 16 weeks. Patients will be screened no more than 3 weeks prior to a 7 day Dietary run in period, which will then be followed by 3 treatment periods. There will be a 2 week washout period between each Treatment Period, to allow for study drug washout and safety review. All patients will visit the study site 2 weeks after treatment period 3 for a follow up visit.

• REC name

  London - West London & GTAC Research Ethics Committee

• REC reference

  14/LO/1773

• Date of REC Opinion

  1 Dec 2014

• REC opinion

  Further Information Favourable Opinion