Phase 2
Completed N=3
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.
Source: ClinicalTrials.gov NCT04179409 ↗Enrolled (actual)
3
Serious AEs
0.0%
Results posted
Oct 2023
Primary outcomePrimary: Change in Dystrophin Expression From Baseline Following Treatment With Either AMONDYS 45 (Previously Casimersen), EXONDYS 51 (Previously Eteplirsen ), or VYONDYS 53 (Previously Golodirsen) — 3.1; 6.1 Percent of Healthy Control
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Dystrophin Expression From Baseline Following Treatment With Either AMONDYS 45 (Previously Casimersen), EXONDYS 51 (Previously Eteplirsen ), or VYONDYS 53 (Previously Golodirsen) |
3.1; 6.1 | — |
| PRIMARY Monitoring for the Development of Unacceptable Toxicity. |
17; 11 | — |
| SECONDARY Change in Dystrophin Expression From Baseline Following Treatment With Either AMONDYS 45 (Previously Casimersen), EXONDYS 51 (Previously Eteplirsen ), or VYONDYS 53 (Previously Golodirsen). |
38; 30.8 | — |
Eligibility Criteria
Inclusion Criteria
- Is a male with DMD and has an out-of-frame duplication of either exon 45, 51, or 53, with a normal copy number of all other DMD exons.
- Is above age 6 months of age.
- Has sufficient muscle mass in a pair of bilateral muscles that will allow for pre- and post-treatment muscle biopsies per PI discretion.
- If the subject is ambulant and 4 years old or greater and has been on a stable dose or dose equivalent of oral corticosteroids for at least 12 weeks prior to Week 1 the dose is expected to remain constant (except for modifications to accommodate changes in weight) throughout the study.
Exclusion Criteria
- Any additional missing exon for DMD that cannot be treated with study drugs.
Other inclusion/exclusion criteria apply
Data sourced from ClinicalTrials.gov (NCT04179409). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.