Purpose:

In this study Pentoxifylline will be added as a rescue treatment to patients who are taking steroids (the same amount for at least 1 year), but are either stable (no improvement of muscle strength over three months) or have continued to slowly deteriorate. Patients are not excluded if they are taking any combination of herbal supplements or vitamins as long as the dosing has been stable for 2 months.

Further Study Details:

Duchenne muscular dystrophy (DMD) is the most common lethal inherited disorder worldwide. Despite the exponential increase in our understanding of the disorder since the discovery and characterization of the causative gene and its product dystrophin in 1987, current therapeutic management remains largely supportive. Awaiting a final genetic cure to be available in the future, further investments in developing better drug therapies for DMD remain important. This study will be designed as a randomized, placebo controlled study. Patients will be enrolled in the study after showing similar muscle testing results during the screening visit. Sixty four patients will be randomized into the pentoxifylline arm or a placebo arm. Subjects will be evaluated by quantitative muscle testing (QMT), manual muscle testing (MMT), timed function testing, functional evaluation and pulmonary function tests, and measurement of contractures. At each visit, subjects will also undergo medical history review and safety evaluation.

Subject Inclusion Criteria:

• Age: 7 years to 100 years
• Ability to ambulate for 10 meters. The use of assistive devices is allowed.
• 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) 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, 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.
• On stable dose of prednisone, prednisolone or deflazacort for at least 12 months prior to screening.
• Participants who are on stable dose of any combination of the following compounds (creatine, glutamine, coenzyme Q10, vitamin E, C or D, JUVEN, arginine, calcium) must have taken these medications for at least 2 months prior to screening. Subjects are not required to take these medications to participate in the study.
• All other herbs, supplements or green tea (other than those noted above) have been discontinued 3 months prior to screening.
• Ability to provide reproducible QMT bicep score with no more than 15% variation between scores during screening.
• Normal blood clotting ability evidenced by a platelet function assessment (PFA). Subject

Subject Exclusion Criteria:

• Currently enrolled in another treatment clinical trial.
• History of significant concomitant illness or significant impairment of renal or hepatic function
• History of impairment of blood clotting ability (as evidenced by increased PT/PTT or PFA over the upper limit of normal (ULN)).
• Recent cerebral or retinal hemorrhage.
• History of bleeding diathesis or gastric ulcer.