Creatine and Glutamine in Steroid-Naive Duchenne Muscular Dystrophy
|Muscular Dystrophy, Duchenne||Drug: Creatine Monohydrate Drug: Glutamine||Phase 2 Phase 3|
|Study Design:||Allocation: Randomized
Primary Purpose: Treatment
|Official Title:||A Multicenter Randomized Placebo-controlled Double-blind Study to Assess Efficacy and Safety of Glutamine and Creatine Monohydrate in Duchenne Muscular Dystrophy|
|Study Start Date:||June 2000|
|Estimated Study Completion Date:||December 2006|
PURPOSE OF THE STUDY The purpose of this study is to see if children with Duchenne muscular dystrophy (DMD) who are given glutamine (a nutritional supplement) or creatine monohydrate (a nutritional supplement) have changes in strength compared to children who are given no medicinal treatment. Children who are chosen to be in this research study will be diagnosed with DMD, be between the ages of 5 and 10 years old, and will not have participated in any other DMD clinical trials within the last year, and will not have been on medications that disqualify them from participation in this trial. All children in this clinical trial are entered at the discretion of the doctors in charge of the study. During the first and second visits, children who fulfill the criteria for participation in the clinical trial will be determined. We expect 50 subjects to participate at CINRG centers worldwide.
Children will be randomized (a procedure like flipping a coin) into one of three groups: treated with glutamine, treated with creatine monohydrate, or untreated (placebo: a sugar pill with no medication). Neither parents, children, nor the doctors treating the children will know the group each child has been assigned to. Because of the study design, there is a possibility that children will receive no treatment (placebo group) during the clinical trial. During the trial children must take one supplement mixed with water in the morning and supplements mixed with water each night.
Nine (9) clinic visits are required to complete this clinical trial. During visits to the clinic, each child will be evaluated by members of the research team to determine the child?s strength. Strength will be measured in different ways: manual testing, quantitative testing, and the usual measures used during visits to the neurologist (child will be asked to rise from the floor, walk across room, etc.). Manual testing is the way strength is usually measured by your child?s doctor. The quantitative muscle strength test (QMT) is a mechanical way to measure strength: the child sits/lays on an examining table and is asked to pull/push a strap. This test will determine the strength of different muscle groups in kilograms of force. An electrocardiogram (ECG, a heart test) will be performed during screening visit 2 and month 6 and blood (a small amount equal to about 2 to 3 tablespoons) will be drawn on screening visit 2 and months 1, 3 and 6. Urine tests will be done at screening visit 2 nd months 1, 3 and 6 to monitor kidney function.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00016653
|United States, District of Columbia|
|Children's National Medical Center|
|Washington, District of Columbia, United States, 20010|
|Study Director:||Diana M Escolar, MD||Cooperative International Neuromuscular Research Group|
|Study Director:||Gunnar Buyse, MD, PhD||Cooperative International Neuromuscular Research Group|