Rare Disease Research, LLC | Atlanta, GA
Status and phase
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About
This study will be comprised of 2 parts: Part 1 (dose escalation) will be conducted to evaluate the safety and tolerability of 2 doses (100 milligrams/kilogram [mg/kg] and 200 mg/kg) of eteplirsen in approximately 10 participants with DMD; Part 2 (dose finding and dose comparison) will be conducted for the selection of a high dose (100 mg/kg versus 200 mg/kg) and its comparison with the 30 mg/kg dose of eteplirsen, in approximately 144 participants with genetically confirmed deletion mutations amenable to treatment by skipping exon 51.
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Other inclusion/exclusion criteria apply.
Primary purpose
Allocation
Interventional model
Masking
160 participants in 4 patient groups
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Central trial contact
Sarepta Therapeutics Inc. For Clinical Trial Information, Select Option 4
Data sourced from clinicaltrials.gov
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