Purpose:

This study was to help determine whether a high-dose weekly course of prednisone therapy is safer than and at least as effective as daily dose therapy for people with Duchenne muscular dystrophy (DMD). Boys enrolled in this study had not taken carnitine, other amino acids, creatine, glutamine, Coenzyme Q10 or any herbal medicines within the last three months. In this study, participants were randomized into groups after being screened to determine eligibility. Participants were then followed for a 12-month period with follow-up visits at month one, three and then every three 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. The effect of a high dose prednisone regimen was evaluated in comparison to a daily dose regimen in a multi-center, randomized, double-blind placebo-controlled 4-arm study. Ambulant children aged 4-10 years with an established DMD diagnosis were studied. Patients underwent 2 screening evaluations within 1 week. Patients were randomized into treatment groups on the second screening visits, followed by a 12-month treatment period. During the treatment period, patients were evaluated at monthly intervals. The primary endpoints were percentage change in average muscle strength score and QMT performance for specific muscle groups. Secondary endpoints included timed function tests, functional grades for arms and legs, and pulmonary function tests.

Results

Please see http://www.mda.org/research/080415dmd_prednisone.html for the results of this study.