Safety, Tolerability and Effects of L-Arginine in Boys With Dystrophinopathy on Corticosteroids
| Status: | Archived |
|---|---|
| Conditions: | Neurology, Orthopedic |
| Therapuetic Areas: | Neurology, Orthopedics / Podiatry |
| Healthy: | No |
| Age Range: | Any |
| Updated: | 7/1/2011 |
| Start Date: | January 2012 |
| End Date: | June 2012 |
| Contact: | Namita Goyal, MD |
| Phone: | 617-726-8736 |
Pilot Study: To Assess the Safety, Tolerability and Effects of L-Arginine on Muscles in Boys With Dystrophinopathy on Corticosteroids
The purpose of the study is to assess the safety, tolerability, and effects of L-Arginine on
muscles in boys with dystrophinopathy on corticosteroids. Specifically, to see if L-arginine
reduces muscle signal abnormalities on MRI done pre and post 30 days of L-arginine
administration.
Dystrophinopathy is a muscular dystrophy (includes Duchenne or Becker's Muscular Dystrophy)
that can be a lethal muscle disorder resulting from defects in the gene for dystrophin, a
structural protein required to maintain muscle integrity. Absence of functional dystrophin
leaves the muscle membrane vulnerable to damage during contraction. This damage can be
exacerbated by an inflammatory response leading to myofiber necrosis.
L-arginine is a widely available dietary supplement amino acid postulated to affect
dystrophinopathy in several favorable ways: upregulation of utrophin, vasodilation in muscle
via nitric oxide, enhanced synthesis of creatine, increase levels of growth hormone.
We hypothesize that administration of L-arginine may increase levels of creatine and growth
hormone and in turn reduce the extent of myofiber damage in our patients with
dystrophinopathy
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