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