Safety, Tolerability and Effects of L-Arginine in Boys With Dystrophinopathy on Corticosteroids (NCT01388764) | Clinical Trial Compass
CompletedPhase 1
Safety, Tolerability and Effects of L-Arginine in Boys With Dystrophinopathy on Corticosteroids
United States7 participantsStarted 2012-01
Plain-language 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.
Who can participate
Age range
7 Years – 11 Years
Sex
MALE
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.