Open label extension study of GSK2402968 in DMD (version 1)

• Research type

  Research Study

• Full title

  An open-label extension study of the long-term safety, tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy (DMD114349)

• IRAS ID

  82368

• Contact name

  Volker Straub

• Eudract number

  2011-001266-17

• ISRCTN Number

  n.a

• Clinicaltrials.gov Identifier

  n.a

• Research summary

  Duchenne Muscular Dystrophy (DMD) is the most frequent inherited fatal childhood disease, affecting 1 in every 3,500 boys born. Muscle weakness is usually seen before age 4, followed by progression to the muscles in the arms, legs and trunk. DMD patients are usually wheel-chair bound by their mid-teens and generally die in their twenties/early thirties. No treatment is currently available to prevent this progression. Steroid treatment has limited success and is considered the current best treatment; however it does not alter the course of the disease.DMD is caused by changes in the DMD gene ?? a key element that determines how a protein, called dystrophin, which is an important part of the structure of muscle cells, is produced. Loss of dystrophin leads to muscle fibre breakdown and muscle weakness. GSK2402968 is a novel drug that works by ??skipping? the changed part of the DMD gene and allowing the production of a version of dystrophin which, although only partially functioning, may lead to a less severe form of the condition.This Phase III open-label extension study will recruit patients who have completed one of two on-going ??feeder? studies investigating the safety, tolerability and efficacy of GSK2402968. It is expected that 200 patients will be enrolled in this study. In the primary treatment arm patients will receive GSK2402968 6mg/kg as an injection under the skin on a weekly basis for 104 weeks. A secondary dosing arm of intermittent injections (8 weeks on, 4 weeks off) is included for patients who have problems with side effects and a natural history observation arm is included for subjects who decline active treatment.At the end of the treatment period, subjects who have completed the study will be followed up for safety and progress for 20 weeks after the last dose of study drug.

• REC name

  East of England - Cambridge South Research Ethics Committee

• REC reference

  11/EE/0293

• Date of REC Opinion

  3 Oct 2011

• REC opinion

  Further Information Favourable Opinion