SMT C1100 in Ambulatory Boys with DMD

• Research type

  Research Study

• Full title

  A Phase 2 Clinical Study to Assess the Activity and Safety of Utrophin Modulation with SMT C1100 in Ambulatory Paediatric Male Subjects with Duchenne Muscular Dystrophy (C11005)

• IRAS ID

  193136

• Contact name

  Francesco Muntoni

• Contact email

  f.muntoni@ucl.ac.uk

• Sponsor organisation

  Summit (Oxford) Limited

• Eudract number

  2015-004333-27

• Duration of Study in the UK

  1 years, 5 months, days

• Research summary

  This study will examine how safe and effective a new medication called SMT C1100 is as a treatment for boys aged 5-10 who suffer from Duchenne Muscular Dystrophy (DMD). Approximately 40 boys will take part in the study in the USA and UK.
  
  DMD often begins by affecting a particular group of muscles, before affecting the muscles more widely. It is characterised by generalised weakness, worsening muscle function over time and will eventually affect the heart or the muscles used for breathing, at which point it becomes life-threatening. DMD affects around 1 in 3500 boys and currently there is no effective treatment for DMD. The average life expectancy is approximately mid-twenties.

  The disorder is caused by a change (mutation) in the gene which codes for the protein dystrophin. Dystrophin is an essential protein for healthy muscle function. A related protein, utrophin, fulfils a similar role and is responsible for muscle growth in foetal and early child development. As a healthy child develops, utrophin levels diminish and dystrophin levels increase, taking on utrophin’s role in muscle development. However, DMD boys do not generate enough functional dystrophin to replace utrophin leading to muscle damage and a gradual weakening of the muscles.

  SMT C1100 is predicted to be a treatment for DMD by increasing the amount of utrophin in the muscle cells and therefore acting as a functioning replacement for the low levels of dystrophin.

  Participants will need to come to the clinic 10 times over the course of the study, about 13 months in total. The study treatment phase lasts 48 weeks. During which participants will receive the study medication, SMT C1100, which will be taken as an oral suspension twice a day after a meal with milk.

• REC name

  London - West London & GTAC Research Ethics Committee

• REC reference

  15/LO/2045

• Date of REC Opinion

  19 Jan 2016

• REC opinion

  Further Information Favourable Opinion