NCT01153932

Phase II Doubleblind Exploratory Study of GSK2402968 in Ambulant Subjects With Duchenne Muscular Dystrophy

A Phase II, Double Blind, Exploratory, Parallel-group, Placebocontrolled Clinical Study to Assess Two Dosing Regimens of GSK2402968 for Efficacy, Safety, Tolerability and Pharmacokinetics in Ambulant Subjects With Duchenne Muscular Dystrophy

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 53 (actual)
• Sponsor: GlaxoSmithKline · Industry
• Sex: Male
• Age: 5 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to determine whether GSK2402968 given as a continuous dose and as an intermittent dose is effective and safe in the treatment of Duchenne muscular dystrophy.

Detailed description

This is a phase II, double-blind, exploratory, parallel-group, placebo-controlled clinical study in ambulant subjects with DMD resulting from a mutation that can be corrected by exon skipping induced by GSK2402968. The study aims to randomise 54 subjects. There will be 2 parallel cohorts. Each cohort will include subjects on GSK2402968 and matched placebo in a 2:1 ratio.

Conditions

• Muscular Dystrophies

Interventions

Type	Name	Description
DRUG	GSK2402968	Subcutaneous injection
DRUG	matched placebo	Subcutaneous injection

Timeline

• Start date: 2010-09-01
• Primary completion: 2012-03-01
• Completion: 2012-09-01
• First posted: 2010-06-30
• Last updated: 2014-08-25

Locations

13 sites across 9 countries: Australia, Belgium, France, Germany, Israel, Netherlands, Spain, Turkey (Türkiye), United Kingdom