Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
Register:
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ClinicalTrials.gov |
Last refreshed on:
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19 February 2015 |
Main ID: |
NCT01388764 |
Date of registration:
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05/07/2011 |
Prospective Registration:
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Yes |
Primary sponsor: |
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Public title:
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Safety, Tolerability and Effects of L-Arginine in Boys With Dystrophinopathy on Corticosteroids
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Scientific title:
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Pilot Study: To Assess the Safety, Tolerability and Effects of L-Arginine on Muscles in Boys With Dystrophinopathy on Corticosteroids |
Date of first enrolment:
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January 2012 |
Target sample size:
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7 |
Recruitment status: |
Completed |
URL:
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http://clinicaltrials.gov/show/NCT01388764 |
Study type:
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Interventional |
Study design:
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Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label
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Phase:
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Phase 1
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Countries of recruitment
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United States
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Contacts
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Name:
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Namita Goyal, MD |
Address:
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Telephone:
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Email:
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Affiliation:
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Massachusetts General Hospital |
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Key inclusion & exclusion criteria
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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
Age minimum:
7 Years
Age maximum:
11 Years
Gender:
Male
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Health Condition(s) or Problem(s) studied
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Becker's Muscular Dystrophy
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Duchenne Muscular Dystrophy
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Dystrophinopathy
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Intervention(s)
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Drug: L-arginine
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Primary Outcome(s)
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MRI/MRS of calf muscle
[Time Frame: Day 0 and Day 30]
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Secondary Outcome(s)
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Pulmonary function tests
[Time Frame: Day 0 and Day 30]
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Blood tests
[Time Frame: Day 0 and Day 30]
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Assessment of muscle strength and function
[Time Frame: Day 0 and Day 30]
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Secondary ID(s)
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2011D001591
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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