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

Murdoch Childrens Research Institute11 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Facio-Scapulo-Humeral Dystrophy FSHD2 FSHD1

Interventions

Eligibility

Sex: ALLMin age: 5 YearsMax age: 18 Years

Medical Language ↔ Plain English

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.

Outcomes

Primary Outcomes

Motor Function Measure for Neuromuscular disease

Composite functional outcome measure

Time frame: 3 months

Secondary Outcomes

Muscle Magnetic Resonance Imaging

Time frame: 3 months

Muscle Ultrasound Scan

Time frame: 3 months

Performance of the Upper Limb Measure

Time frame: 3 months

ACTIVILIM

Time frame: 3 Months

PedsQL Neuromuscular

Time frame: 3 months

FSH-COM

FSHD specific composite measure

Time frame: 3 months

FSH-Health Index (Pediatric Version)

Patient reported outcome measure specific for patients with FSHD.

Time frame: 3 months

Six Minute Walk Test

Time frame: 3 months

FSHD Severity Score

Time frame: 3 months

Quantitative muscle strength testing

Time frame: 3 months

GPX3 Level

Possible biomarker of disease severity in FSHD

Time frame: 3 months

Step Counter

Physical activity measure

Time frame: 3 months

Laboratory safety monitoring

bloods and urine safety testing (urea and electrolytes, urine plasma creatine:creatinine ratios)

Time frame: 3 months

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

Overview

Conditions

Interventions

Eligibility

Locations (1)

Outcomes

Primary Outcomes

Secondary Outcomes