Effect of Creatine Monohydrate on Functional Muscle Strength in Children With FSHD

Not Applicable

Completed

• Conditions: Facio-Scapulo-Humeral Dystrophy; FSHD1; FSHD2
• Interventions: Dietary Supplement: Creatine Monohydrate; Dietary Supplement: Placebo

• Registration Number: NCT02948244
• Lead Sponsor: Murdoch Childrens Research Institute

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2016-10-26
• Study First Submitted QC: 2016-10-27
• Study First Posted: 2016-10-28
• Study Start: 2017-10-31
• Primary Completion: 2021-01-31
• Study Completion: 2022-07-01
• Status Verified: 2023-02
• Last Update Submitted: 2023-02-04
• Last Update Posted: 2023-02-08

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 11

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
Group B - Placebo/Active	Creatine Monohydrate	Participants will receive 3 months of placebo followed by a 6 week washout period followed by 3 months of creatine monohydrate.
Group A - Active/Placebo	Creatine Monohydrate	Participants will receive 3 months of creatine monohydrate followed by a 6 week washout period followed by 3 months of placebo.
Group A - Active/Placebo	Placebo	Participants will receive 3 months of creatine monohydrate followed by a 6 week washout period followed by 3 months of placebo.
Group B - Placebo/Active	Placebo	Participants will receive 3 months of placebo followed by a 6 week washout period followed by 3 months of creatine monohydrate.

Primary Outcome Measures

Name	Time	Method
Motor Function Measure for Neuromuscular disease	3 months	Composite functional outcome measure

Secondary Outcome Measures

Name	Time	Method
FSHD Severity Score	3 months
Muscle Ultrasound Scan	3 months
PedsQL Neuromuscular	3 months
Quantitative muscle strength testing	3 months
GPX3 Level	3 months	Possible biomarker of disease severity in FSHD
FSH-COM	3 months	FSHD specific composite measure
FSH-Health Index (Pediatric Version)	3 months	Patient reported outcome measure specific for patients with FSHD.
Laboratory safety monitoring	3 months	bloods and urine safety testing (urea and electrolytes, urine plasma creatine:creatinine ratios)
Six Minute Walk Test	3 months
Step Counter	3 months	Physical activity measure
Muscle Magnetic Resonance Imaging	3 months
Performance of the Upper Limb Measure	3 months
ACTIVILIM	3 Months

Trial Locations

Locations (1)

The Royal Children's Hospital; 🇦🇺 Melbourne, Victoria, Australia