Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy
The primary objective of this study is to assess the ongoing efficacy, safety, and tolerability of an additional 80 weeks of treatment with eteplirsen (AVI-4658) in Duchenne muscular dystrophy (DMD) subjects who have successfully completed the 28-week eteplirsen study: Study 4658-US-201. This study will also evaluate the correlation between biomarkers for DMD and the clinical status of participating DMD subjects.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Open-Label, Multiple-Dose, Efficacy, Safety, and Tolerability Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy Who Participated in Study 4658-US-201|
- The primary efficacy endpoint will be the change from baseline in the percent of dystrophin positive fibers in muscle biopsy tissue as measured by immunohistochemistry (IHC). [ Time Frame: 20 Weeks ] [ Designated as safety issue: Yes ]The primary efficacy endpoint will be based on the pre-treatment and post-treament percent of dystrophin positive fibers as measured in the muscle biopsy tissue on(IHC).
- The secondary efficacy endpoints will be the change from baseline in: CD3, CD4, and CD8 lymphocyte counts in muscle biopsy tissue; 6-Minute Walk Test (6-MWT) distance. [ Time Frame: 20,80 Weeks ] [ Designated as safety issue: Yes ]A key secondary efficacy endpoint will be based on the pre-treatment and post-treatment CD3, CD4, and CD8 lymphocyte counts as measured in the muscle biopsy tissue as well as a 6-Minute Walk Test(6-MWT)distance.
|Study Start Date:||February 2012|
|Estimated Study Completion Date:||December 2013|
|Estimated Primary Completion Date:||September 2013 (Final data collection date for primary outcome measure)|
Experimental: AVI-4658 (Eteplirsen)
Multiple-Dose Extension Study
Drug: AVI-4658 (Eteplirsen)
Eteplirsen will be administered once weekly via an IV infusion over a period of at least 60 minutes. Their are two treatment groups, 30 mg/kg and 50 mg/kg.
|United States, Ohio|
|Nationwide Children's Hospital|
|Columbus, Ohio, United States, 43205|
|Principal Investigator:||Jerry R Mendell, MD||Nationwide Childtren's Hospital|