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

• Status: Completed
• Trial end date: 2012-05-01
• Target enrollment: 0
• Participant gender: Male

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.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Massachusetts General Hospital

Criteria

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