Safety, Tolerability and Effects of L-Arginine in Boys With Dystrophinopathy on Corticosteroids

Duchenne Muscular Dystrophy Clinical Trial

Official title: Pilot Study: To Assess the Safety, Tolerability and Effects of L-Arginine on Muscles in Boys With Dystrophinopathy on Corticosteroids

Status Completed

Clinical Trial Summary

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.

Clinical Trial Description

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 ;

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label

Related Conditions & MeSH terms

• Becker's Muscular Dystrophy
• Duchenne Muscular Dystrophy
• Dystrophinopathy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

• NCT number: NCT01388764
• Study type: Interventional
• Source: Massachusetts General Hospital
• Status: Completed
• Phase: Phase 1
• Start date: January 2012
• Completion date: May 2012