utriceuticals in Duchenne muscular dystrophy: a double blind, randomised controlled trial investigating the use of a multicomponent nutritional supplement to maintain or improve function as measured by StepWatch Step Activity Monitors.

The Royal Children's Hospital0 个研究点目标入组 48 人开始时间: 2010年6月7日最近更新: 2022年5月30日

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•Boys will be considered for inclusion if they have a definite diagnosis: phenotypic evidence of Duchenne muscular dystrophy (DMD) based on the onset of characteristic clinical symptoms or signs (ie, proximal muscle weakness, waddling gait, and Gowers’ maneuver) by 9 years of age, an elevated serum creatine kinase (CK), and ongoing difficulty with ambulation, with documentation of a deletion or duplication in the dystrophin gene, or absence of Dystrophin on muscle biopsy. Boys also need to be sufficiently ambulatory to walk = 75 meters unassisted during a 6 minute walk test (6MWT). Other personal assistance or use of assistive devices for ambulation (eg, short leg braces, long leg braces, or walkers) is not permitted. Parents/guardians need to be able to give full informed written consent.

•use of a wheelchair/motorised scooter/similar mobility aid for >75 % of activity during the day; cows milk protein allergy or lactose intolerance; history of kidney or liver disease; unable to attend the seven required hospital appointments within the 12 month period; unable to swallow tablets; parents wish to continue using other complementary therapies; participation in other muscular dystrophy trials involving an active intervention; use of medications which are contraindicated due to possible drug interactions.

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