Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy
Status:
Completed
Trial end date:
2009-03-31
Target enrollment:
Participant gender:
Summary
Duchenne muscular dystrophy (DMD), a fatal muscle degenerative disorder, arises from
mutations in the dystrophin gene. Antisense therapy with the use of antisense
oligonucleotides (AON) has the potential to restore effectively the production of dystrophin,
the defective protein, in >70% of DMD. This could result in increased life expectancy through
improved muscle survival and function. Recent scientific research has demonstrated the
potential of this technique to skip mutated dystrophin exons, restore the reading frame and
generate functional dystrophin protein. Having demonstrated proof-of-principle in human cell
culture and animal model studies, we now intend to determine efficacy and safety of this
approach to induce dystrophin exon skipping in children with DMD.
The specific aim of this phase I/II study is to assess efficacy (dystrophin production) and
safety of intramuscular administered morpholino oligomer directed against exon 51 (AVI-4658
PMO). We are performing parallel preclinical studies to develop methods of systemic delivery
that will be necessary for future phase II/III clinical studies.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
Imperial College London
Collaborators:
Department of Health, United Kingdom Sarepta Therapeutics, Inc.