Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12610000462088
Ethics application status
Approved
Date submitted
21/05/2010
Date registered
7/06/2010
Date last updated
26/05/2022
Date data sharing statement initially provided
26/05/2022
Type of registration
Prospectively registered

Titles & IDs
Public title
Nutriceuticals in Duchenne muscular dystrophy
Scientific title
Nutriceuticals 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.
Secondary ID [1] 251837 0
nil
Universal Trial Number (UTN)
U1111-1115-0920
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Duchenne muscular dystrophy 257436 0
Condition category
Condition code
Neurological 257584 257584 0 0
Other neurological disorders
Human Genetics and Inherited Disorders 257670 257670 0 0
Other human genetics and inherited disorders
Musculoskeletal 257671 257671 0 0
Other muscular and skeletal disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
The enhanced dietary supplement regime will consist of a shake made from 22g whey protein isolate powder administered to provide 20g protein; creatine monohydrate (5g); and glutamine (0.6g/kg fat free mass/day). The enhanced dietary will also include beta-hydroxy-beta-methylbutyrate (38mg/kg/day rounded to nearest 500mg) given in capsule form. Inactive ingredients used in the shake are maltodextrin powder (8g) and sucralose (5g). The shake will be made on 250ml skim milk and will be consumed once per day. The supplement regime also includes a paediatric multivitamin (Vitamin A 582.5 IU, Vitamin B1 350 microg, Vitamin B2 550 microg, Vitamin B3 6 mg, Vitamin B5 1.5 mg, Vitamin B6 650 microg, Vitamin B12 750 microg, Folic acid 50 microg, Vitamin C 20 mg, Vitamin D3 100 IU, Natural Vitamin E 15 IU, Biotin 25 microg, Calcium 5 mg, Iodine 75 microg, Iron 1.3 mg, Magnesium 5 mg, Manganese 12.5 microg, Zinc 1mg; 1 chewable capsules per day for participants aged 5 to 6 years; 2 chewable capsules per day for participants aged 7-12 years), fish oil (1050mg per day in 2 chewable capsules), and vitamin D (2000 IU/day in two gel capsules). This regime will be consumed for 20 weeks. During the two week washout period, participants will be supplied with a standard supplement regime. This consists of a shake made from 22g whey protein isolate powder administered to provide 20g protein. The shake will be made on 250ml skim milk and will be consumed once per day. The standard regime also includes a paediatric multivitamin (Vitamin A 582.5 IU, Vitamin B1 350 microg, Vitamin B2 550 microg, Vitamin B3 6 mg, Vitamin B5 1.5 mg, Vitamin B6 650 microg, Vitamin B12 750 microg, Folic acid 50 microg, Vitamin C 20 mg, Vitamin D3 100 IU, Natural Vitamin E 15 IU, Biotin 25 microg, Calcium 5 mg, Iodine 75 microg, Iron 1.3 mg, Magnesium 5 mg, Manganese 12.5 microg, Zinc 1mg; 1 chewable capsules per day for participants aged 5 to 6 years; 2 chewable capsules per day for participants aged 7-12 years), fish oil (1050mg per day in 2 chewable capsules) , and vitamin D (2000 IU/day in two gel capsules).
Intervention code [1] 256535 0
Treatment: Drugs
Intervention code [2] 256605 0
Treatment: Other
Comparator / control treatment
The standard supplement regime consists of a shake made from 22g whey protein isolate powder administered to provide 20g protein. Inactive ingredients used in the shake are maltodextrin powder (16g) and sucralose (5g). The shake will be made on 250ml skim milk and will be consumed once per day. Gel caps with maltodextrin (38mg/kg/day rounded to nearest 500mg) will be used for the beta-hydroxy-beta-methylbutyrate placebo. The standard regime also includes a paediatric multivitamin (Vitamin A 582.5 IU, Vitamin B1 350 microg, Vitamin B2 550 microg, Vitamin B3 6 mg, Vitamin B5 1.5 mg, Vitamin B6 650 microg, Vitamin B12 750 microg, Folic acid 50 microg, Vitamin C 20 mg, Vitamin D3 100 IU, Natural Vitamin E 15 IU, Biotin 25 microg, Calcium 5 mg, Iodine 75 microg, Iron 1.3 mg, Magnesium 5 mg, Manganese 12.5 microg, Zinc 1mg; 1 chewable capsules per day for participants aged 5 to 6 years; 2 chewable capsules per day for participants aged 7-12 years), fish oil (1050mg per day in 2 chewable capsules) , and vitamin D (2000 IU/day in two gel capsules). This regime will be consumed for 20 weeks.
Control group
Active

Outcomes
Primary outcome [1] 258504 0
Inactive minutes as measured by StepWatch Step Activity Monitor.
Timepoint [1] 258504 0
Boys will by required to wear the monitor for five days each at five time points (25 days total): week 0 (baseline); week 8; week 28; week 30; week 50.
Secondary outcome [1] 264301 0
Distance covered in six minute walk test.
Timepoint [1] 264301 0
Week 0 (baseline); week 8; week 28; week 30; week 50.
Secondary outcome [2] 264302 0
Fat free mass as measured by Dual Energy X-ray Absorptiometry (DXA) scan.
Timepoint [2] 264302 0
Week 0; week 28; week 50.
Secondary outcome [3] 264303 0
ACTIVLIM survey on activity limitations
Timepoint [3] 264303 0
Week 0 (baseline); week 8; week 28; week 30; week 50.
Secondary outcome [4] 264304 0
PedsQL survey: general and neuromuscular models.
Timepoint [4] 264304 0
Week 0 (baseline); week 8; week 28; week 30; week 50.
Secondary outcome [5] 264305 0
Vitamin D measured by venous blood test for serum 25-hydroxy vitamin D.
Timepoint [5] 264305 0
Week 0 (baseline); week 8; week 28; week 30; week 50.

Eligibility
Key inclusion criteria
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.
Minimum age
5 Years
Maximum age
13 Years
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
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
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Potential participants will be approached by the Neuromuscular clinic at The Royal Children's Hospital. If interested, participants will be screened by an associate investigator. Upon obtaining consent, participants will be allocated a unique code. This code will be provided to the study statistician who is off site with the subjects age. The allocation will be provided by locked data file to an associate investigator who is not involved in assessments or data analysis. The associate investigator will provide allocation details to an offsite formula room that will prepare the supplement. Participants will receive individual shake sachets labelled only with their unique code.
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
A random sequence will be generated in Excel.
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?



Intervention assignment
Crossover
Other design features
The design features a two month lead in period and a two week wash out period at week 28 to week 30. The standard nutritional supplement will continue through the washout period. The participants will be allocated to treatment as aged matched pairs.
Phase
Phase 2
Type of endpoint/s
Safety/efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Date of first participant enrolment
Anticipated
1/11/2010
Actual
11/01/2011
Date of last participant enrolment
Anticipated
31/12/2016
Actual
4/09/2015
Date of last data collection
Anticipated
Actual
30/10/2016
Sample size
Target
48
Accrual to date
Final
38
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Recruitment hospital [1] 1644 0
The Royal Childrens Hospital - Parkville
Recruitment hospital [2] 1645 0
Royal Children's Hospital - Herston
Recruitment hospital [3] 1646 0
The Children's Hospital at Westmead - Westmead
Recruitment hospital [4] 3854 0
Sydney Children's Hospital - Randwick

Funding & Sponsors
Funding source category [1] 257025 0
Hospital
Name [1] 257025 0
Murdoch Children's Research Institute: Critical Care and Neurosciences Theme
Country [1] 257025 0
Australia
Funding source category [2] 258088 0
Charities/Societies/Foundations
Name [2] 258088 0
John T Reid Charitable Trust
Country [2] 258088 0
Australia
Funding source category [3] 258089 0
Charities/Societies/Foundations
Name [3] 258089 0
Muscular Dystrophy Australia
Country [3] 258089 0
Australia
Funding source category [4] 291376 0
Charities/Societies/Foundations
Name [4] 291376 0
Duchenne Foundation
Country [4] 291376 0
Australia
Funding source category [5] 291377 0
Charities/Societies/Foundations
Name [5] 291377 0
Muscular Dystrophy Foundation
Country [5] 291377 0
Australia
Primary sponsor type
Hospital
Name
The Royal Children's Hospital
Address
The Royal Children's Hospital
Flemington Road
Parkville
VIC 3052
Country
Australia
Secondary sponsor category [1] 256281 0
University
Name [1] 256281 0
Monash University; Department of Nutrition and Dietetics
Address [1] 256281 0
Monash Medical Centre
246 Clayton Road
Level 5; Block E
Clayton
VIC, 3168
Country [1] 256281 0
Australia
Other collaborator category [1] 252328 0
Hospital
Name [1] 252328 0
Royal Children's Hospital
Address [1] 252328 0
Royal Children's Hospital
Herston Road
HERSTON Queensland 4029
Country [1] 252328 0
Australia
Other collaborator category [2] 252329 0
Hospital
Name [2] 252329 0
Mater Children's Hospital
Address [2] 252329 0
Mater Children's Hospital,
Raymond Terrace,
South Brisbane Queensland 4101
Country [2] 252329 0
Australia
Other collaborator category [3] 277682 0
Hospital
Name [3] 277682 0
The Children's Hospital at Westmead
Address [3] 277682 0
Postal address: The Children's Hospital at Westmead, Locked Bag 4001, Westmead 2145

Street Address: Cnr Hawkesbury Rd and Hainsworth St, Westmead, Sydney, NSW, 2145
Country [3] 277682 0
Australia
Other collaborator category [4] 278488 0
Hospital
Name [4] 278488 0
Sydney Children's Hospital
Address [4] 278488 0
High St, Randwick NSW 2031, Australia
Country [4] 278488 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 259040 0
The Royal Children's Hospital Human Research Ethics Committee
Ethics committee address [1] 259040 0
Ethics committee country [1] 259040 0
Australia
Date submitted for ethics approval [1] 259040 0
15/03/2010
Approval date [1] 259040 0
20/10/2010
Ethics approval number [1] 259040 0
30022
Ethics committee name [2] 260081 0
Monash University Human Research Ethics Committee
Ethics committee address [2] 260081 0
Ethics committee country [2] 260081 0
Australia
Date submitted for ethics approval [2] 260081 0
10/11/2010
Approval date [2] 260081 0
17/11/2010
Ethics approval number [2] 260081 0
2010001692
Ethics committee name [3] 272101 0
Royal Children's Hospital Human Research Ethics Committee
Ethics committee address [3] 272101 0
Ethics committee country [3] 272101 0
Australia
Date submitted for ethics approval [3] 272101 0
Approval date [3] 272101 0
02/02/2011
Ethics approval number [3] 272101 0
HREC/11/QRCH/2
Ethics committee name [4] 272102 0
Mater Hospital Human Research Ethics Committee
Ethics committee address [4] 272102 0
Ethics committee country [4] 272102 0
Australia
Date submitted for ethics approval [4] 272102 0
Approval date [4] 272102 0
27/06/2011
Ethics approval number [4] 272102 0
1730C
Ethics committee name [5] 292934 0
Hunter New England Human Research Ethics Committee
Ethics committee address [5] 292934 0
Ethics committee country [5] 292934 0
Australia
Date submitted for ethics approval [5] 292934 0
Approval date [5] 292934 0
11/06/2014
Ethics approval number [5] 292934 0
13/05/15/3.04

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 31207 0
Prof Helen Truby
Address 31207 0
Monash University
Department Nutrition and Dietetics
Level 1
264 Ferntree Gully Road
Notting Hill, VIC, 3168
Country 31207 0
Australia
Phone 31207 0
+61399024261
Fax 31207 0
Email 31207 0
[email protected]
Contact person for public queries
Name 14454 0
Zoe Davidson
Address 14454 0
Monash University
Department Nutrition and Dietetics
Level 1
264 Ferntree Gully Road
Notting Hill, VIC, 3168
Country 14454 0
Australia
Phone 14454 0
+61399024274
Fax 14454 0
Email 14454 0
[email protected]
Contact person for scientific queries
Name 5382 0
Zoe Davidson
Address 5382 0
Monash University
Department Nutrition and Dietetics
Level 1
264 Ferntree Gully Road
Notting Hill, VIC, 3168
Country 5382 0
Australia
Phone 5382 0
+61399024274
Fax 5382 0
Email 5382 0
[email protected]

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.