NCT02286947

Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 24 (actual)
• Sponsor: Sarepta Therapeutics, Inc. · Industry
• Sex: Male
• Age: 7 Years – 21 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Muscular Dystrophy, Duchenne

Interventions

Type	Name	Description
DRUG	Eteplirsen	Eteplirsen solution for IV infusion

Timeline

• Start date: 2014-11-01
• Primary completion: 2017-04-21
• Completion: 2018-03-23
• First posted: 2014-11-10
• Last updated: 2020-03-30
• Results posted: 2019-02-20

Locations

9 sites across 1 country: United States

Regulatory

• FDA-regulated drug study