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

NCT01388764 | Completed Phase 1

• 1 other identifier: 2011D001591
• Study Type: interventional
• Target: 7
• Locations: 1 country
• Sites: 1

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.

Conditions

Dystrophinopathy; Duchenne Muscular Dystrophy; Becker's Muscular Dystrophy

Keywords

L-arginine supplement study

Outcome Measures

Primary Outcomes (1)

• MRI/MRS of calf muscle

  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.

  Day 0 and Day 30

Secondary Outcomes (3)

• Blood tests

  Day 0 and Day 30

• Assessment of muscle strength and function

  Day 0 and Day 30

• Pulmonary function tests

  Day 0 and Day 30

Study Arms (1)

L-arginine

EXPERIMENTAL

Drug: L-arginine

Interventions

L-arginine DRUG

Subjects will receive oral L-Arginine (0.3 grams/kg/day, divided 2 times per day, not to exceed 14 grams/day)

L-arginine

Eligibility Criteria

• Age: 7 Years - 11 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• Presence of metallic orthopedic hardware in the lower extremity that could affect MRI/MRS measurements
• 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

Sponsors & Collaborators

• Massachusetts General Hospital: lead

Study Sites (1)

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

Arginine

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Amino Acids, Basic; Amino Acids; Amino Acids, Peptides, and Proteins; Amino Acids, Diamino; Amino Acids, Essential

Study Officials

• Namita Goyal, MD

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Physician Neurologist

Study Record Dates

• First Submitted: July 5, 2011
• First Posted: July 7, 2011
• Study Start: January 1, 2012
• Primary Completion: May 1, 2012
• Study Completion: May 1, 2012
• Last Updated: July 3, 2012

Record last verified: 2012-06

Locations

US (1)