Duchenne Muscular Dystrophy Treatment With Eteplirsen – Phase III

Study with a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy

Protocol Description

The objective of this multicenter study is to confirm effectiveness of eteplirsen (AVI-4658) in improving muscle function in boys who have a certain type of Duchenne muscular dystrophy (DMD). Additional objectives include evaluation of safety, biomarkers, and the long-term effects of eteplirsen use.

Study Level: Phase III

Eligibility Criteria

Subject to certain exclusion criteria, the study is accepting male participants ages 7 to 16, who have a confirmed diagnosis of DMD with a type of genetic code error that may be treatable with eteplirsen using a principle called exon skipping – specifically exon 51 skipping. They will be included in the arm of the study that receives the investigational drug. A second study arm will enroll boys with DMD who have a genetic error that investigators believe is not treatable through exon 51 skipping. This group will not receive eteplirsen, but will be observed as a comparative control group.
Boys: Ages 7 to 16


Participants in the treatment arm of this study will receive weekly infusions of eteplirsen for 96 weeks. After 52 weeks, infusions may optionally be provided at the patient’s home by a visiting nurse, but monthly visits will still be required for assessments. Physical testing, health assessments, and biological sample collection will be done at specific, less-frequent intervals. Participants in the control arm will receive the same assessments, without the weekly infusions.
Visits: Up to 100; fewer if infusions are done at patient’s home
Duration: About 2 years and 2 months

Status: Open to Enrollment

Source(s) of Support

Sarepta Therapeutics

Primary Investigator

Hoda Abdel-Hamid, MD, MS

Contact Information

For more information about the study or enrollment, please contact:
Jennifer Monahan, Clinical Research Coordinator