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Treatment Study for Boys with Duchenne Syndrome

Clinical Trial Title: 
Clinical research trial to determine if their new drug improves muscle function compared to placebo and to determine that it is safe and to document side effects if any.
Clinical Trial Protocol ID: 
Clinical Trial Investigator Name: 
Peter T. Heydemann, MD
Clinical Trial Protocol Description: 

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.

Clinical Trial Eligibility Criteria: 

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.

Clinical Trial Area: 
Pediatric Neurological Disorders
Contact Phone: 
(312) 942-0079
Contact Name: 
Susan Rohde