8 to 17 years old
duchenne muscular dystrophy
Drug study, Phase I/II
Medical History Physical/Neurological Examination Review of Medications Urinalysis Blood draw Electrocardiogram Cardiac function testing Strength, Range of motion, mobility testing Body compositon testing Study medication Medication diary
Age 8 years to 17 years
Non-Ambulatory (unable to complete 10m run/walk under 10s)
Diagnosis of DMD confirmed by at least one the following:
Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, and clinical picture consistent with typical DMD, or
Gene deletions test positive (missing one or more exons) of the dystrophin gene, where reading frame can be predicted as 'out-of-frame', and clinical picture consistent with typical DMD, or
Complete dystrophin gene sequencing showing an alteration (point mutation, duplication, or other mutation resulting in a stop codon mutation) that can be definitely associated with DMD, with a typical clinical picture of DMD, or
Positive family history of DMD confirmed by one of the criteria listed above in a sibling or maternal uncle, and clinical picture typical of DMD.
Cardiac ejection fraction >55% on echocardiogram
Use of nutritional, herbal and antioxidant supplements taken with the intent of maintaining or improving skeletal muscle strength or functional mobility has been discontinued at least 4 weeks prior to screening (daily multivitamin use is acceptable).
Glucocorticoid therapy, if used, must have a stable weight-based dose for at least 3 months prior to enrollment
Cardiac therapy, if used, includes prophylactic ACE inhibitors, aldosterone receptor antagonists (e.g.
spironolactone, eplerenone, etc.), and/or beta-blocker therapy, and must be stable for 3 months prior to enrollment.
Hematology profile within normal range.
Baseline laboratory safety chemistry profile within typical range for DMD (elevated ALT / AST acceptable in the absence of elevated GGT, elevated CK acceptable).
Inability to complete cardiac or strength, range of motion and mobility assessments per protocol
Current enrollment in another treatment clinical trial.
History of significant concomitant illness or significant impairment of renal or hepatic function.
Use of regular daily aspirin or other medication with antiplatelet effects within 3 weeks of first dose of study medication.
Cardiac symptoms that, in the opinion of the investigator, may be suggestive of imminent moderate to severe cardiac events, irrespective of LVEF.
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