At a glance
ClinicalIndex Comparison Record- ✓Male with DMD and out-of-frame duplication of exon 45, 51, or 53 with normal copy number of all other DMD exons
- ✓Age greater than 6 months
- ✓Sufficient muscle mass in bilateral muscles allowing pre- and post-treatment biopsies per PI discretion
- ✓If ambulant and ≥4 years old on corticosteroids, must be on stable dose for ≥12 weeks prior to Week 1 with expectation of remaining constant
- ✕Any additional missing exon for DMD that cannot be treated with study drugs
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications.
In Brief
A Phase 2 clinical trial evaluating Amondys 45, Exondys 51, and 1 other intervention for Duchenne Muscular Dystrophy. Completed, enrolled 3 participants across 1 site.
Detailed Summary
This is an 48-week open-label study to determine the efficacy and safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 for the treatment of boys with duchenne muscular dystrophy who have a single exon duplication of either exon 45, 51 or 53, respectively. There will be weekly infusions and two muscle biopsies at baseline and at month 12.
Study Details
Timeline
Interventions
This drug is used to target skipping of exon 45 of the dystrophin gene.
This drug is used to target skipping of exon 51 of the dystrophin gene.
This drug is used to target skipping of exon 53 of the dystrophin gene.