Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy
Trial ID or NCT#
This is an open-label study to assess the safety, tolerability, efficacy and pharmacokinetics of eteplirsen in patients with early stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
An Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early Stage Duchenne Muscular Dystrophy
- - Male 4-6 years of age. - Diagnosis of DMD, genotypically confirmed. - Stable dose of oral corticosteroids for at least 12 weeks or has not received corticosteroids for at least 12 weeks. - Intact right and left biceps muscles or two alternative upper arm muscle groups. - Parent that is willing to provide consent and comply with study procedures.
- - Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks that may have an effect on muscle strength or function (e.g., growth hormone, anabolic steroids). - Previous or current treatment with any other experimental treatments within 12 weeks or participation in any other clinical trial within 6 months. - Major surgery within 3 months prior to the first dose of study drug, or planned surgery during this study which would interfere with the ability to perform study activities. - Presence of other clinically significant illness.
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