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

Facio-Scapulo-Humeral Dystrophy Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02948244
• Other study ID #: 36298
• Status: Completed
• Phase: N/A
• Start date: October 31, 2017
• Est. completion date: July 1, 2022

Study information

• Verified date: February 2023
• Source: Murdoch Childrens Research Institute
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 11
• Est. completion date: July 1, 2022
• Est. primary completion date: January 31, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 5 Years to 18 Years

Eligibility

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.

Related Conditions & MeSH terms

• Facio-Scapulo-Humeral Dystrophy
• FSHD1
• FSHD2
• Muscular Dystrophy, Facioscapulohumeral

Intervention

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.
• Placebo
Placebo

Locations

Country	Name	City	State
Australia	The Royal Children's Hospital	Melbourne	Victoria

Sponsors (1)

Lead Sponsor	Collaborator
Murdoch Childrens Research Institute

Country where clinical trial is conducted

Australia, 

Outcome

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