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

• Status: Completed
• Trial end date: 2012-09-01
• Target enrollment: 0
• Participant gender: Male

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: GlaxoSmithKline

Criteria

Inclusion Criteria:

• Ambulant subjects with Duchenne muscular dystrophy resulting from a mutation in 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) or H-RMCA (High-Resolution Melting Curve Analysis), and correctable by GSK2402968-induced DMD exon 51 skipping,

• Males, at least 5 years of age and with a life expectancy of at least 1 year

• Able to rise from floor in ≤7 seconds (without aids/orthoses),

• Able to complete the 6MWD test with a distance of at least 75m

• 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 immediately prior to screening and a reasonable expectation that total daily dosage and dosing regimen will not change significantly for the duration of the study

• QTc <450msec

• On adequate contraception

• Able to comply with and complete all protocol requirements

Exclusion Criteria:

• any additional missing exon for DMD

• Current of history of liver or renal disease or impairment

• Acute illness within 4 weeks of the first dose

• Use of prohibited meds within 6 months of fist dose

• Current participation in any other investigational clinical trial

• Positive hepatitis B surface antigen, hepatitis C antibody test, or human immunodeficiency virus (HIV) test at screening

• Symptomatic cardiomyopathy

• Children in Care