PILOT TRIAL OF PENTOXIFYLLINE IN DUCHENNE MUSCULAR DYSTROPHY (DMD)

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. This will be an open-label pilot study of pentoxifylline in steroid-naive patients with DMD. The patient population will include boys, aged 4 to 7 years old, with an established DMD diagnosis, that have not received glucocorticosteroids within one year of their enrollment in the study. The primary endpoint is the change in QMT of 5 muscle groups (elbow flexion and extension, knee flexion and extension and grip). The secondary endpoint is the change on MMT score at 12 months compared to baseline, functional evaluation and timed function tests. Exploratory endpoints will include MRI, TGF beta levels and TNF alpha levels, which will be explored to assess baseline characteristics of MRI in DMD, MRI disease progression patterns over time and effect of therapy