Study of Eteplirsen in DMD Patients
Trial ID or NCT#
The main objective of this study is to provide evidence of efficacy of eteplirsen (AVI-4658) in Duchenne muscular dystrophy (DMD) patients that are amenable to skipping exon 51. Additional objectives include evaluation of safety, biomarkers and the long-term effects of eteplirsen up to 96 weeks, followed by a safety extension (not to exceed 48 weeks).
An Open-Label, Multi-Center, Study With a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy
- - Male 7-16 years old - Diagnosed with DMD, genotypically confirmed - Stable dose of corticosteroids for at least 24 weeks - Have intact right and left alternative upper muscle groups - Mean 6MWT greater than 300m (primary analysis on 300 to 450 meters) - Stable pulmonary and cardiac function: predicted FVC equal to or greater than 50% and LVEF of greater than 50%
- - Previous treatment with drisapersen or any other RNA antisense agent or any gene therapy within the last 6 months - Participation in any other DMD interventional clinical study within 12 weeks - Major surgery within 3 months - Presence of other clinically significant illness - Major change in the physical therapy regime within 3 months Other inclusion/exclusion criteria apply.
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John W. Day, MD, PhD