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Study of Eteplirsen in DMD Patients
study id #: NCT02255552
condition: Duchenne Muscular Dystrophy (DMD)
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).
mechanism of action: Exon-skipping to promote dystrophin production
last updated: July 08, 2019
start date: November 17, 2014
estimated completion: June 14, 2019
phase of development: Phase 3
size / enrollment: 109
This is an open-label, multi-center study to evaluate the efficacy and safety of eteplirsen in patients with genotypically confirmed Duchenne muscular dystrophy (DMD) with genetic deletions amenable to exon 51 skipping (treated group), with a concurrent control arm of DMD patients not amenable to exon 51 skipping (untreated group). Following primary efficacy endpoints, dosing will continue to week 144 to evaluate the long term effects of eteplirsen.
Patients in the treated group will receive once weekly intravenous (IV) infusions of 30 mg/kg Eteplirsen for 96 weeks, followed by a safety extension (not to exceed 48 weeks). Patients in the untreated group will not receive treatment.
Clinical efficacy will be assessed at regularly scheduled study visits, including functional tests such as the six minute walk test. Patients in the treated group will undergo a muscle biopsy at Baseline and a second muscle biopsy over the course of the study. Patients in the untreated group will not undergo muscle biopsy.
Safety, including adverse event monitoring and routine laboratory assessments, will be continuously monitored for all patients.
- Change in 6-Minute Walk Test (6MWT) distance from baseline [ Time Frame: Change from Baseline to Week 96 ]
- The percentage of dystrophin-positive fibers [ Time Frame: Change from Baseline ]
- Maximum inspiratory/expiratory pressure percent predicted (MIP/MEP % predicted) [ Time Frame: Change from Baseline ]
• 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
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