utriceuticals in Duchenne muscular dystrophy
- Conditions
- Duchenne muscular dystrophyNeurological - Other neurological disordersHuman Genetics and Inherited Disorders - Other human genetics and inherited disordersMusculoskeletal - Other muscular and skeletal disorders
- Registration Number
- ACTRN12610000462088
- Lead Sponsor
- The Royal Children's Hospital
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Male
- Target Recruitment
- 48
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.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method