Creatine and Glutamine in Steroid-Naive Duchenne Muscular Dystrophy

• Status: Completed
• Trial end date: 2006-12-01
• Target enrollment: 0
• Participant gender: Male

Summary

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.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Cooperative International Neuromuscular Research Group

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)