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.

Plain-language summary

This 48-week study evaluates the efficacy and safety of the drugs Amondys 45, Exondys 51, and Vyondys 53 in people with Duchenne muscular dystrophy who carry eligible DMD gene duplications. It enrolled male patients with DMD who had a duplication of exon 45, 51, or 53. The study was conducted in Phase 2 and has been completed.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 2

Status

Completed

Sponsor

Kevin Flanigan

Intervention

Amondys 45, Exondys 51, Vyondys 53

Start date

2/18/2020

Estimated completion

9/1/2023

Countries

United States

Related genes

Locations

• Columbus, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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