Effect of Creatine Monohydrate on Functional Muscle Strength in Children With FSHD
Primary Purpose
Facio-Scapulo-Humeral Dystrophy, FSHD2, FSHD1
Status
Completed
Phase
Not Applicable
Locations
Australia
Study Type
Interventional
Intervention
Creatine Monohydrate
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Facio-Scapulo-Humeral Dystrophy focused on measuring fshd, creatine monohydrate, children, pediatrics, Facio-Scapulo-Humeral Dystrophy
Eligibility Criteria
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.
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.
Sites / Locations
- The Royal Children's Hospital
Arms of the Study
Arm 1
Arm 2
Arm Type
Active Comparator
Active Comparator
Arm Label
Group A - Active/Placebo
Group B - Placebo/Active
Arm Description
Participants will receive 3 months of creatine monohydrate followed by a 6 week washout period followed by 3 months of placebo.
Participants will receive 3 months of placebo followed by a 6 week washout period followed by 3 months of creatine monohydrate.
Outcomes
Primary Outcome Measures
Motor Function Measure for Neuromuscular disease
Composite functional outcome measure
Secondary Outcome Measures
Muscle Magnetic Resonance Imaging
Muscle Ultrasound Scan
Performance of the Upper Limb Measure
ACTIVILIM
PedsQL Neuromuscular
FSH-COM
FSHD specific composite measure
FSH-Health Index (Pediatric Version)
Patient reported outcome measure specific for patients with FSHD.
Six Minute Walk Test
FSHD Severity Score
Quantitative muscle strength testing
GPX3 Level
Possible biomarker of disease severity in FSHD
Step Counter
Physical activity measure
Laboratory safety monitoring
bloods and urine safety testing (urea and electrolytes, urine plasma creatine:creatinine ratios)
Full Information
NCT ID
NCT02948244
First Posted
October 26, 2016
Last Updated
February 4, 2023
Sponsor
Murdoch Childrens Research Institute
1. Study Identification
Unique Protocol Identification Number
NCT02948244
Brief Title
Effect of Creatine Monohydrate on Functional Muscle Strength in Children With FSHD
Official 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
Study Type
Interventional
2. Study Status
Record Verification Date
February 2023
Overall Recruitment Status
Completed
Study Start Date
October 31, 2017 (Actual)
Primary Completion Date
January 31, 2021 (Actual)
Study Completion Date
July 1, 2022 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Murdoch Childrens Research Institute
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Facio-Scapulo-Humeral Dystrophy, FSHD2, FSHD1
Keywords
fshd, creatine monohydrate, children, pediatrics, Facio-Scapulo-Humeral Dystrophy
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Crossover Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
11 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Group A - Active/Placebo
Arm Type
Active Comparator
Arm Description
Participants will receive 3 months of creatine monohydrate followed by a 6 week washout period followed by 3 months of placebo.
Arm Title
Group B - Placebo/Active
Arm Type
Active Comparator
Arm Description
Participants will receive 3 months of placebo followed by a 6 week washout period followed by 3 months of creatine monohydrate.
Intervention Type
Dietary Supplement
Intervention Name(s)
Creatine Monohydrate
Intervention Description
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.
Intervention Type
Dietary Supplement
Intervention Name(s)
Placebo
Intervention Description
Placebo
Primary Outcome Measure Information:
Title
Motor Function Measure for Neuromuscular disease
Description
Composite functional outcome measure
Time Frame
3 months
Secondary Outcome Measure Information:
Title
Muscle Magnetic Resonance Imaging
Time Frame
3 months
Title
Muscle Ultrasound Scan
Time Frame
3 months
Title
Performance of the Upper Limb Measure
Time Frame
3 months
Title
ACTIVILIM
Time Frame
3 Months
Title
PedsQL Neuromuscular
Time Frame
3 months
Title
FSH-COM
Description
FSHD specific composite measure
Time Frame
3 months
Title
FSH-Health Index (Pediatric Version)
Description
Patient reported outcome measure specific for patients with FSHD.
Time Frame
3 months
Title
Six Minute Walk Test
Time Frame
3 months
Title
FSHD Severity Score
Time Frame
3 months
Title
Quantitative muscle strength testing
Time Frame
3 months
Title
GPX3 Level
Description
Possible biomarker of disease severity in FSHD
Time Frame
3 months
Title
Step Counter
Description
Physical activity measure
Time Frame
3 months
Title
Laboratory safety monitoring
Description
bloods and urine safety testing (urea and electrolytes, urine plasma creatine:creatinine ratios)
Time Frame
3 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
5 Years
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Ian R Woodcock, MBBS
Organizational Affiliation
Murdoch Children Research Institute/Royal Children Hospital, Melbourne
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Monique M Ryan
Organizational Affiliation
Murdoch Children Research Institute/Royal Children Hospital, Melbourne
Official's Role
Principal Investigator
Facility Information:
Facility Name
The Royal Children's Hospital
City
Melbourne
State/Province
Victoria
ZIP/Postal Code
3052
Country
Australia
12. IPD Sharing Statement
Plan to Share IPD
No
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Effect of Creatine Monohydrate on Functional Muscle Strength in Children With FSHD
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