Queen Square Centre for Neuromuscular Diseases


Dose-ranging study of AVI-4658 to induce dystrophin expression in selected Duchenne Muscular Dystrophy (DMD) patients - (Systemic study)

Sponsor AVI Biopharma
Funder Medical Research Council (MRC) and AVI Biopharma
CI Professor Kate Bushby / Professor Francesco Muntoni
sites Newcastle, London GOSH
More information


Studies have shown that AVI-4658 may be useful in managing DMD in boys with a frame-shifted mutation in dystrophin gene which could be corrected after removal of exon 51. That will happen if enough drug reaches the nucleus of the affected muscle cell.

Trial information

This was a safety study of AVI-4658 to skip exon 51 of the dystrophin gene in relevant patients with DMD.

This was an open-label, two-centre, dose-ranging comparative clinical study. All patients will receive 12 weekly intravenous infusions of AVI-4658.


  • to assess safety
  • to select the best dose

The best dose would have to elicit at least 10% new dystrophin and dystrophin-positive fibres in a sentinel muscle group after treatment.


  • Up to 16 ambulatory boys, aged ≥5 and ≤15 years
  • four treatment groups with four patients each (drug dose ranging from 0.5 mg/kg to 4 mg/kg)

This trial was conducted in London and Newcastle.