KUL0401: An Open-label Pilot Study of Oxatomide in Steroid-Naive Duchenne Muscular Dystrophy

Subject Inclusion Criteria

• 5 to 10 years of age
• ambulatory
• diagnosis of DMD confirmed by at least one of the following:

• Positive x-linked family history of DMD in older male relatives (onset by 5 years, wheelchair bound by 12 years), or;
• Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, and clinical picture consistent with typical DMD, or;
• Gene deletion test positive (missing one or more exons) in the central rod domain (exons 25-60) of dystrophin, where reading frame can be predicted as 'out of frame', and clinical picture consistent with typical DMD.
• glucocorticosteroid-naive (i.e. has not been treated with prednisone or deflazacort within the past year)
• Evidence of muscle weakness by MRC score or clinical functional evaluation
• QMT biceps score variability no greater than 10% between screening visits

Subject Exclusion Criteria

• Failure to achieve one or more of the inclusion criteria listed above
• Inability to suitably cooperate with strength assessments
• Symptomatic DMD carrier
• Use of oxatomide (or other anti-histamine drugs) within the last 6 months for DMD or any other disease
• Use of creatine monohydrate or glutamine within the last 6 months
• Use of carnitine, Coenzyme Q10, other amino acids or any herbal medications within the last 3 months
• History of symptomatic cardiomyopathy
• History of impairment of hepatic function
• History of significant concomitant illness or significant impairment of renal function.