At a glance
ClinicalIndex Comparison Record- ✓Male aged 10–17 years at study drug administration
- ✓Clinical diagnosis of DMD with documented out-of-frame deletions or absent dystrophin on muscle biopsy, specifically deletions skippable by exon 51 (45-50; 47-50; 48-50; 49-50; 50; 52; 52-63)
- ✓<5% revertant fibres on muscle biopsy
- ✓Unable to ambulate or stand independently
- ✕Prior EDB muscle removal or resection
- ✕Stage 4 EDB muscle preservation on MRI
- ✕Left ventricular shortening fraction <25% or ejection fraction <35% on echocardiography at Visit 1 or within 3 months
- ✕Nocturnal hypoventilation (mean oxygen saturation ≤94% on overnight sleep study)
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: A Phase I/II Clinical Trial Using AVI-4658
In Brief
A Phase 2 clinical trial evaluating AVI-4658 (PMO) for Duchenne Muscular Dystrophy. Completed, enrolled 7 participants across 1 site.
Detailed 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.
Study Details
Timeline
Interventions
morpholino antisense oligonucleotide