In March 2011, ethical approval was obtained to start with the DEMAND III study in the Leiden University Medical Center (Principal investigator Erik Niks, pediatric neurologist). In this international phase III study, 180 boys with Duchenne muscular dystrophy will be randomized in a 2:1 ratio to assess the efficacy of systemic exon 51 skipping by subcutaneous administration of 6 mg/kg GSK2402968 versus placebo during 48 weeks. Boys need to be at least 5 years old and able to complete the 6 minute walking test with minimal distance of at least 75m as results of this test will be the primary endpoint. They must be on steroids for at least 6 months with no significant change in total daily dosage or dosing regimen for a minimum of 3 months.
Hopefully this phase III trial will demonstrate that treatment with this drug will result in a functional benefit for the boys with Duchenne.
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