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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02948244




Registration number
NCT02948244
Ethics application status
Date submitted
26/10/2016
Date registered
28/10/2016
Date last updated
26/08/2019

Titles & IDs
Public title
Effect of Creatine Monohydrate on Functional Muscle Strength in Children With FSHD
Scientific title
Effect of Creatine Monohydrate on Functional Muscle Strength and Muscle Mass in Children With FSHD: a Multi-centre, Randomised, Double-blind Placebo-controlled Crossover Trial
Secondary ID [1] 0 0
36298
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Facio-Scapulo-Humeral Dystrophy 0 0
FSHD2 0 0
FSHD1 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Neurological 0 0 0 0
Other neurological disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Other interventions - Creatine Monohydrate
Other interventions - Placebo

Active Comparator: Group A - Active/Placebo - Participants will receive 3 months of creatine monohydrate followed by a 6 week washout period followed by 3 months of placebo.

Active Comparator: Group B - Placebo/Active - Participants will receive 3 months of placebo followed by a 6 week washout period followed by 3 months of creatine monohydrate.


Other interventions: Creatine Monohydrate
Synthetically produced dietary supplement Creatine Monohydrate will be used in powder form reconstituted to a drink. The dosage will be 100mg/kg/day up to a maximum of 10 grams daily.

Other interventions: Placebo
Placebo

Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Motor Function Measure for Neuromuscular disease - Composite functional outcome measure
Timepoint [1] 0 0
3 months
Secondary outcome [1] 0 0
Muscle Magnetic Resonance Imaging
Timepoint [1] 0 0
3 months
Secondary outcome [2] 0 0
Muscle Ultrasound Scan
Timepoint [2] 0 0
3 months
Secondary outcome [3] 0 0
Performance of the Upper Limb Measure
Timepoint [3] 0 0
3 months
Secondary outcome [4] 0 0
ACTIVILIM
Timepoint [4] 0 0
3 Months
Secondary outcome [5] 0 0
PedsQL Neuromuscular
Timepoint [5] 0 0
3 months
Secondary outcome [6] 0 0
FSH-COM - FSHD specific composite measure
Timepoint [6] 0 0
3 months
Secondary outcome [7] 0 0
FSH-Health Index (Pediatric Version) - Patient reported outcome measure specific for patients with FSHD.
Timepoint [7] 0 0
3 months
Secondary outcome [8] 0 0
Six Minute Walk Test
Timepoint [8] 0 0
3 months
Secondary outcome [9] 0 0
FSHD Severity Score
Timepoint [9] 0 0
3 months
Secondary outcome [10] 0 0
Quantitative muscle strength testing
Timepoint [10] 0 0
3 months
Secondary outcome [11] 0 0
GPX3 Level - Possible biomarker of disease severity in FSHD
Timepoint [11] 0 0
3 months
Secondary outcome [12] 0 0
Step Counter - Physical activity measure
Timepoint [12] 0 0
3 months
Secondary outcome [13] 0 0
Laboratory safety monitoring - bloods and urine safety testing (urea and electrolytes, urine plasma creatine:creatinine ratios)
Timepoint [13] 0 0
3 months

Eligibility
Key inclusion criteria
- Is between the ages of 5 and 18 years inclusive at the time of randomisation;

- Has a confirmed genetic diagnosis of Facioscapulohumeral Muscular Dystrophy (FSHD)
types 1 or 2;

- Has a legally acceptable representative capable of understanding the informed consent
document and providing consent on the participant's behalf.
Minimum age
5 Years
Maximum age
18 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Has clinically significant elevation in plasma creatinine level or unexplained
hypertension at screening;

- Has a prior diagnosis of chronic renal failure;

- Has a known hypersensitivity to creatine monohydrate of maltodextrin placebo;

- Patients already taking any medications to increase muscle bulk or strength or
concomitant use of regular sodium valproate, corticosteroids of alpha agonists such as
salbutamol.

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)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Crossover
Other design features
Phase
Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
The Royal Children's Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
3052 - Melbourne

Funding & Sponsors
Primary sponsor type
Other
Name
Murdoch Childrens Research Institute
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This multi-centre, randomised, double-blind, placebo-controlled crossover trial will compare
changes in strength-related motor function following treatment with creatine monohydrate to
treatment with placebo, as measured by the Motor Function Measure, from baseline to 12 weeks.
Eligible subjects will undergo baseline assessments then will be randomised to either
creatine monohydrate therapy or placebo for three months, followed by a six week wash-out
period, then crossover to a further three months of therapy with either placebo or creatine.
Subjects will undergo clinical assessments and study safety assessments at the beginning and
end of each treatment period. The study will begin recruitment in early 2017.
Trial website
https://clinicaltrials.gov/show/NCT02948244
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Ian R Woodcock, MBBS
Address 0 0
Murdoch Children Research Institute/Royal Children Hospital, Melbourne
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Ian R Woodcock, MBBS
Address 0 0
Country 0 0
Phone 0 0
+61 3 9345 5661
Fax 0 0
Email 0 0
ian.woodcock@rch.org.au
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT02948244