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

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

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.

Study Overview

• Status: Completed
• Conditions: Facio-Scapulo-Humeral Dystrophy; FSHD2; FSHD1
• Intervention / Treatment: Dietary supplement: Placebo; Dietary supplement: Creatine Monohydrate

• Study Type: Interventional
• Enrollment (Actual): 11
• Phase: Not Applicable

Contacts and Locations

Study Locations

• Australia
  • Victoria
    • Melbourne, Victoria, Australia, 3052
      • The Royal Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years to 18 years (ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: CROSSOVER
• Masking: QUADRUPLE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
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.	Dietary supplement: Placebo; Placebo; Dietary supplement: 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.
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.	Dietary supplement: Placebo; Placebo; Dietary supplement: 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Motor Function Measure for Neuromuscular disease	Composite functional outcome measure	3 months

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Murdoch Childrens Research Institute

Study record dates

Study Major Dates

• Study Start (ACTUAL): October 31, 2017
• Primary Completion (ACTUAL): January 31, 2021
• Study Completion (ACTUAL): July 1, 2022

Study Registration Dates

• First Submitted: October 26, 2016
• First Submitted That Met QC Criteria: October 27, 2016
• First Posted (ESTIMATE): October 28, 2016

Study Record Updates

• Last Update Posted (ACTUAL): February 8, 2023
• Last Update Submitted That Met QC Criteria: February 4, 2023
• Last Verified: February 1, 2023

More Information

Terms related to this study

• Keywords: pediatrics; children; fshd; creatine monohydrate; Facio-Scapulo-Humeral Dystrophy
• Additional Relevant MeSH Terms: Nervous System Diseases; Genetic Diseases, Inborn; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Muscular Disorders, Atrophic; Muscular Dystrophies; Muscular Dystrophy, Facioscapulohumeral
• Other Study ID Numbers: 36298

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• 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