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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01388764
Recruitment Status : Completed
First Posted : July 7, 2011
Last Update Posted : July 3, 2012
Information provided by (Responsible Party):
Namita Goyal, Massachusetts General Hospital

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.

Condition or disease Intervention/treatment Phase
Dystrophinopathy Duchenne Muscular Dystrophy Becker's Muscular Dystrophy Drug: L-arginine Phase 1

Detailed 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

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 7 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Official Title: Pilot Study: To Assess the Safety, Tolerability and Effects of L-Arginine on Muscles in Boys With Dystrophinopathy on Corticosteroids
Study Start Date : January 2012
Actual Primary Completion Date : May 2012
Actual Study Completion Date : May 2012

Arm Intervention/treatment
Experimental: L-arginine Drug: L-arginine
Subjects will receive oral L-Arginine (0.3 grams/kg/day, divided 2 times per day, not to exceed 14 grams/day)

Primary Outcome Measures :
  1. 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.

Secondary Outcome Measures :
  1. 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.

  2. 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.

  3. Pulmonary function tests [ Time Frame: Day 0 and Day 30 ]
    Subjects will have pulmonary function studies to assess forced vital capacity

Information from the National Library of Medicine

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Ages Eligible for Study:   7 Years to 11 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Confirmation of diagnosis of dystrophinopathy, documented by clinical exam and dystrophin DNA mutation analysis
  • Ambulatory male subjects between the ages of 7-11 years
  • Stable dosage of corticosteroids for 3 months prior to entry (Screening/Baseline Day 0) and during treatment period
  • Able to follow instructions and give assent
  • Able to complete nonsedated MR

Exclusion Criteria:

  • Presence of metallic orthopedic hardware in the lower extremity that could affect MRI/MRS measurements
  • Routine MRI exclusion criteria such as the presence of a pacemaker, cochlear implant, or cerebral aneurysm clip
  • Subjects not capable of cooperating during MR examination
  • Known hypersensitivity to L-arginine
  • Exposure to another investigational agent, investigational supplements, growth hormone within 3 months prior to entry (Screening/Baseline Day 0) or during treatment period
  • Subjects must not be taking L-arginine for at least 4 weeks prior to entry (Day 0)
  • Subjects who are non-ambulatory or with daytime ventilatory dependence

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01388764

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United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Sponsors and Collaborators
Massachusetts General Hospital
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Principal Investigator: Namita Goyal, MD Massachusetts General Hospital
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Responsible Party: Namita Goyal, Physician Neurologist, Massachusetts General Hospital Identifier: NCT01388764    
Other Study ID Numbers: 2011D001591
First Posted: July 7, 2011    Key Record Dates
Last Update Posted: July 3, 2012
Last Verified: June 2012
Keywords provided by Namita Goyal, Massachusetts General Hospital:
L-arginine supplement study
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked