Creatine and Glutamine in Steroid-Naive Duchenne Muscular Dystrophy

This study has been completed.
Sponsor:
Information provided by:
Cooperative International Neuromuscular Research Group
ClinicalTrials.gov Identifier:
NCT00016653
First received: May 21, 2001
Last updated: October 26, 2011
Last verified: October 2011
  Purpose

This study will help to determine the effectiveness of glutamine and creatine as a possible therapy for DMD. Boys with DMD who are enrolled in this trial will be randomly chosen to receive creatine monohydrate or glutamine or an inactive placebo orally for six months. Once a month during the six-month treatment period, the study participants will have their muscle strength evaluated using manual and computerized testing methods. This study will be conducted at several CINRG Centers throughout the U.S., Belgium, Israel and Puerto Rico. This study is supported by the Muscular Dystrophy Association.


Condition Intervention Phase
Muscular Dystrophy, Duchenne
Drug: Creatine Monohydrate
Drug: Glutamine
Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Masking: Double-Blind
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

Resource links provided by NLM:


Further study details as provided by Cooperative International Neuromuscular Research Group:

Estimated Enrollment: 48
Study Start Date: June 2000
Estimated Study Completion Date: December 2006
Detailed Description:

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.

PROCEDURES

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.

  Eligibility

Ages Eligible for Study:   5 Years to 9 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

INCLUSION CRITERIA

  • Aged 5 - 9 years old
  • Able to walk without assistance
  • Diagnosis of DMD confirmed by one of the following:
  • a) Positive X-linked family history; or
  • b) Dystrophin immunofluorescence and/or immunoblot, which shows complete dystrophin deficiency, and clinical picture consistent with DMD; or
  • c) Gene deletion test positive in the central rod domain (exons 25 - 60) of dystrophin, where reading frame can be predicted as 'out-of-frame', and clinical picture consistent with DMD.
  • Glucocorticosteroid-naive (i.e. has not been treated with prednisone or deflazacort within 1 year before the study began), or has been involved in other therapeutic research protocol within the last year
  • Forced Vital Capacity (a lung function test) > 50% of predicted value
  • Evidence of muscle weakness by MRC score or clinical functional evaluation
  • MRC (manual muscle test) score variability no greater than 10% between screening visits 1 and 2

EXCLUSION CRITERIA

  • Failure to achieve any of the criteria listed above
  • Symptomatic DMD carrier
  • Symptomatic cardiomyopathy or ventricular arrhythmias
  • Previous (6 months or less) or current use of glutamine or creatine (for DMD or any other indication)
  • Use of carnitine, other amino acids, coenzyme Q10, or any herbal medicines within the last month
  • History of significant concomitant illness or significant impairment of renal or hepatic function
  • Evidence of allergy to chocolate or milk solids (substances will be delivered in a powdered hot cocoa mixture)
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00016653

Locations
United States, District of Columbia
Children's National Medical Center
Washington, District of Columbia, United States, 20010
Sponsors and Collaborators
Cooperative International Neuromuscular Research Group
Investigators
Study Director: Diana M Escolar, MD Cooperative International Neuromuscular Research Group
Study Director: Gunnar Buyse, MD, PhD Cooperative International Neuromuscular Research Group
  More Information

Additional Information:
Publications:
ClinicalTrials.gov Identifier: NCT00016653     History of Changes
Other Study ID Numbers: CNMC0599
Study First Received: May 21, 2001
Last Updated: October 26, 2011
Health Authority: United States: Food and Drug Administration

Keywords provided by Cooperative International Neuromuscular Research Group:
Duchenne
muscular
dystrophy
treatment

Additional relevant MeSH terms:
Muscular Dystrophy, Duchenne
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on August 20, 2014