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Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy

Muscular Dystrophy, Duchenne Clinical Trial

Official title:
An Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Eteplirsen in Patients With Advanced Stage Duchenne Muscular Dystrophy

Clinical Trial Summary

The primary objective of this study is to explore safety and tolerability of eteplirsen in participants with advanced stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.

Clinical Trial Description

This is an open-label, multi-center study to explore the safety and tolerability of eteplirsen injection in participants with advanced stage DMD with confirmed genetic mutations amenable to treatment by exon 51 skipping.

Participants will be evaluated for inclusion during a Screening/Baseline period of up to 4 weeks. Eligible participants will receive once weekly intravenous (IV) infusions of 30 mg/kg eteplirsen for 96 weeks, followed by a safety extension (not to exceed 48 weeks).

Safety will be regularly assessed throughout the study via the collection of adverse events (AEs), laboratory tests, electrocardiograms (ECGs), echocardiograms (ECHOs), vital signs, and physical examinations. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT02286947
Study type Interventional
Source Sarepta Therapeutics, Inc.
Contact
Status Completed
Phase Phase 2
Start date November 2014
Completion date March 23, 2018
View Details
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