The trial will enroll 16 ambulatory boys with Duchenne muscular dystrophy (DMD) and initially evaluate multiple intravenous doses of AVI-4658 between 0.5-4mg/kg. This is an open label, 12 week safety trial, which includes measures of drug efficacy and pharmacokinetics.
The clinical study started in London, UK at the UCL Institute of Child Health/Great Ormond Street Hospital NHS Trust facilities by members of the Mdex Consortium led by Francesco Muntoni and will shortly start to recruit patients in Newcastle Upon Tyne.
Avi BioPharma is the sponsor for the trial and Dr Muntoni has been awarded funding support of $1.3 million from the UK Medical Research Council to offset some of the clinical costs of the trial.
Stephen Shrewsbury, chief medical officer of Avi BioPharma, said: We are very pleased to begin the systemic evaluation of our exon skipping drug – AVI-4658 – for the treatment of DMD. We believe that this trial will build significantly on the data generated by the successful recent trial evaluating intramuscular administration of the same drug in DMD boys.