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

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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

Brief 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.

Detailed Summary:

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


Sponsor: Massachusetts General Hospital

Current Primary Outcome: MRI/MRS of calf muscle [ Time Frame: Day 0 and Day 30 ]

MRI/MRS will be performed of the calf muscle in all subjects (N=8) to assess muscle signal abnormalities on MRI and creatine levels on MRS, done at the start of the study (Day 0) and at the end of the study (Day 30), after 30 days of L-arginine administration.


Original Primary Outcome: MRI/MRS of calf muscle [ Time Frame: Day 0 and Day 30 ]

MRI/MRS will be performed of the calf muscle in all subjects (N=6-8) to assess muscle signal abnormalities on MRI and creatine levels on MRS, done at the start of the study (Day 0) and at the end of the study (Day 30), after 30 days of L-arginine administration.


Current Secondary Outcome:

  • Blood tests [ Time Frame: Day 0 and Day 30 ]
    We will obtain safety labs [complete blood count (CBC) and comprehensive metabolic panel (CMP)] from all subjects (N =8), at day 0 and day 30, after 30 days of oral L-argninine administration.
  • Assessment of muscle strength and function [ Time Frame: Day 0 and Day 30 ]
    Measurements of upper and lower extremity strength will be performed using a hand-held dynamometer. Functional tests will also be performed which include time to walk specified distances and time to climb stairs.
  • Pulmonary function tests [ Time Frame: Day 0 and Day 30 ]
    Subjects will have pulmonary function studies to assess forced vital capacity


Original Secondary Outcome:

  • Blood tests [ Time Frame: Day 0 and Day 30 ]
    We will obtain safety labs [complete blood count (CBC) and comprehensive metabolic panel (CMP)] from all subjects (N =6-8), at day 0 and day 30, after 30 days of oral L-argninine administration.
  • Assessment of muscle strength and function [ Time Frame: Day 0 and Day 30 ]
    Measurements of upper and lower extremity strength will be performed using a hand-held dynamometer. Functional tests will also be performed which include time to walk specified distances and time to climb stairs.
  • Pulmonary function tests [ Time Frame: Day 0 and Day 30 ]
    Subjects will have pulmonary function studies to assess forced vital capacity


Information By: Massachusetts General Hospital

Dates:
Date Received: July 5, 2011
Date Started: January 2012
Date Completion:
Last Updated: June 30, 2012
Last Verified: June 2012