Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy

Summary

The primary objective of this study is to assess the ongoing efficacy, safety, and tolerability of an additional 212 weeks of treatment with eteplirsen injection 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.

Timeline

Start

2012-02-27

Primary completion

2016-04-15

Completion

2017-08-16

Outcome

Met primary endpoint

paper At 36 months, eteplirsen-treated patients (n = 12) demonstrated a statistically significant advantage of 151m (p < 0.01) on 6MWT PMID 26573217 ↗

release “As previously reported, Study 202 met its primary endpoint of increased novel dystrophin as assessed by muscle biopsy at week 48” sarepta.com ↗

release “As previously reported, Study 202 met its primary endpoint of increased novel dystrophin as assessed by muscle biopsy at Week 48” sarepta.com ↗

release “As previously reported, Study 202 met its primary endpoint of increased novel dystrophin as assessed by muscle biopsy at Week 48.” sarepta.com ↗

release “At 36 months, eteplirsen-treated patients demonstrated a statistically significant difference of 151 meters in six minute walk test (6MWT), compared to the external cohort.” sarepta.com ↗

Publications

• Mendell JR, Goemans N, Lowes LP, Alfano LN, Berry K, Shao J, Kaye EM, Mercuri E; Eteplirsen Study Group and Telethon Foundation DMD Italian Network. Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy. Ann Neurol. 2016 Feb;79(2):257-71. doi: 10.1002/ana.24555. Epub 2016 Jan 8.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Eteplirsen	Antisense oligonucleotide	30 mg/kg	Intravenous
Subject	Eteplirsen	Antisense oligonucleotide	50 mg/kg	Intravenous

Indications