Muscular Dystrophies Clinical Trial
Official title:
An Exploratory Study to Assess Two Doses of GSK2402968 in the Treatment of Ambulant Boys With Duchenne Muscular Dystrophy
The purpose of this study is to determine if GSK2402968 is effective in the treatment of ambulant boys with Duchenne muscular dystrophy resulting from a mutation thought to be corrected by exon 51 skipping. Two doses of GSK2402968 and placebo will be used in this study.
Boys with Duchenne Muscular Dystrophy (DMD) suffer from a relentless, progressive and fatal
disease due to lack of dystrophin, a critical muscle protein. There is currently no known
cure for the disease. GSK2402968 is thought to correct several genetic mutations through
skipping of exon 51 and therefore targets only those boys with these mutations.
A reasonable hypothesis is that increasing dystrophin will result in clinical improvement,
and that the amount of dystrophin expressed will correlate with clinical improvement above a
threshold level (e.g. around 30% of control). The initial limited efficacy data from
completed and ongoing unblinded studies with GSK2402968 are encouraging as they have
demonstrated de novo production of dystrophin and improved walking ability (primary efficacy
endpoint) after 48 weeks of treatment which has been generally well tolerated.
This study is designed to explore the efficacy, safety and pharmacokinetics of two doses of
GSK2402968 given over 24 weeks. The two doses to be assessed are 6mg/kg/week and 3mg/kg/week.
Based on pharmacokinetic and pharmacodynamic modeling, it is predicted at steady-state that
the 6 mg/kg/week dose will induce dystrophin expression greater than 30% of control. The 3
mg/kg/week dose was chosen as modeling predicts 3 mg/kg/week of GSK2402968 will produce
dystrophin expression in the range of 18-22%. Potential variability between subjects could
theoretically produce higher expression and lead to a dystrophin level correlated with
clinical improvement.
Following the treatment period, the study has a 24 week post-treatment phase. The purpose of
the post-treatment phase is to model the half-life of dystrophin, assess maintenance of
response, and provide information about resolution of adverse event and laboratory
abnormalities following cessation of treatment.
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