A phase III, randomized, double blind, placebo-controlled clinical study to assess the efficacy and safety of GSK2402968 in subjects with Duchenne muscular dystrophy. - N/A

INTERVENTION: Product Code: GSK2402968 Pharmaceutical Form: Injection* Current Sponsor code: GSK2402968 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 200‐ Pharmaceutical form of the placebo: Injection* Route of administration of the placebo: Subcutaneous use CONDITION: Duchenne muscular dystrophy ; MedDRA version: 12.1 Level: LLT Classification code 10013801 Term: Duchenne muscular dystrophy PRIMARY OUTCOME: Main Objective: To assess the efficacy of subcutaneous 6 mg/kg GSK2402968 versus placebo; administered over 48 weeks in ambulant subjects with DMD. Primary end point(s): The primary endpoint is the mean change from baseline in the 6MWD and results will be compared between active and placebo groups using statistical hypothesis testing.; The primary time point of interest will be the end of treatment (Week 48). Confirmatory statistical testing will be performed only for the primary variable, analysed using the ITT analysis population, therefore, no adjustments for multiplicity are required. A 5%; significance level will be used to determine a statistically significant difference. Secondary Objective: To evaluate the safety and tolerability of subcutaneous 6 mg/kg GSK2402968; versus placebo administered over 48 weeks in ambulant subjects with DMD.; • To evaluate the PK of subcutaneous 6 mg/kg GSK2402968 administered over 48; weeks in ambulant subjects with DMD.; • To evaluate the impact on quality of life of GSK2402968 administered over 48; weeks in ambulant subjects with DMD. INCLUSION CRITERIA: 1. Ambulant subjects with Duchenne muscular dystrophy resulting from a mutation/deletion within the DMD gene, confirmed by a state‐of‐the‐art DNA diagnostic technique covering all DMD gene exons, including but not limited to MLPA (Multiplex Ligation‐dependent Probe Amplification), CGH (Comparative Genomic Hybridisation), SCAIP (Single Condition Amplification/Internal Primer) or H‐RMCA (High‐Resolution Melting Curve Analysis), and correctable by GSK2402968‐induced DMD exon 51 skipping. 2. Males, aged at least 5 years, 3. Life expectancy of at least 1 year, 4. Able to complete 6MWD test with minimal distance of at least 75m at each predrug visit. In addition, results of 6MWD must be within 20% of each other at each pre‐drug visit. 5. Receiving glucocorticoids for a minimum of 6 months immediately prior to screening, with no significant change in total daily dosage or dosing regimen for a minimum of 3 months