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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT02948244
Ethics application status
Date submitted
26/10/2016
Date registered
28/10/2016

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
Treatment: Other - Creatine Monohydrate
Treatment: Other - 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.


Treatment: Other: 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.

Treatment: Other: Placebo
Placebo
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Motor Function Measure for Neuromuscular disease
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
Timepoint [6] 0 0
3 months
Secondary outcome [7] 0 0
FSH-Health Index (Pediatric Version)
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
Timepoint [11] 0 0
3 months
Secondary outcome [12] 0 0
Step Counter
Timepoint [12] 0 0
3 months
Secondary outcome [13] 0 0
Laboratory safety monitoring
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
Sex
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
NA
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
31/10/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/07/2022
Sample size
Target
Accrual to date
Final
11
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
Trial website
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
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

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


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT02948244