Treatment Study for Boys with Duchenne Syndrome

Mallinckrodt’s goal is that this drug will replace steroids as standard course of treatment for boys with Duchenne. A goal of 132 boys will be recruited globally with Rush taking 1 or 2 subjects.

In order to participate you must meet the following criteria:

• Are male and between 4 and 8 years of age (inclusive) at the screening visit.
• Have a documented diagnosis of Duchenne muscular dystrophy (DMD) confirmed by complete dystrophin deficiency (by immunofluorescence and/or immunoblot), or identifiable mutation in the DMD gene where reading frame can be predicated as “out of frame”, or complete dystrophin gene sequencing consistent with DMD; AND in the opinion of the investigator, a typical clinical profile consistent with DMD.
• Are unable to complete the 10 meter walk/run test at the screening and/or baseline visit.

You will be excluded from the study if any of the following criteria apply to you:

• Have had previous systemic treatment with corticosteroids within 2 months prior to the screening visit.
  Exception: In subjects who were down-titrated to a physiological dose of corticosteroids a maximum of 1 month of no greater than a physiological dose followed by 1 month completely off corticosteroids prior to the screening visit will be acceptable for study entry. Transient previous use of corticosteroids will be evaluated on a case-by-case basis by the sponsor or designee. The use of topical or intra-articular corticosteroids is permitted during the study.
• Are unwilling to receive, or is intolerant of, SC injections.
• Have Type 1 or Type 2 diabetes mellitus.

This is a partial list of inclusion and exclusion criteria.