Muscular Dystrophy, Duchenne Clinical Trial
Official title:
A Double-Blinded Randomized Placebo Controlled Study of Daily Pentoxifylline as a Rescue Treatment in DMD
The purpose of this study is to see if male children with Duchenne muscular dystrophy (DMD)
have changes in strength when given the drug Pentoxifylline as a rescue treatment. A total
of 64 subjects are expected to participate through all other centers of the Cooperative
International Neuromuscular Research Group (CINRG) worldwide.
The primary purpose of this study is to see whether the addition of pentoxifylline to a
steroid regimen is effective in treating deteriorating muscle strength by comparing the
muscle strength of PTX treated subjects and placebo treated subjects.
DMD is the most common and devastating type of muscular dystrophy (incidence 1 in 3500 live
born males worldwide). DMD is characterized by a complete loss of dystrophin, leading to
progressive muscle weakness and wasting.
No cure is currently available despite our present understanding of the disorder and the
discovery and characterization of the causative gene and its protein product dystrophin in
1987. Corticosteroids (prednisone, deflazacort) may delay disease progression and until now
it is the only treatment that proved to be beneficial for patients with DMD. Other
alternative supplements like creatine and glutamine also delay diseased progression.
;
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator), Primary Purpose: Treatment
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