Clinical trial NCT02948244: 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
Overall status: COMPLETED
Phase: NA
Conditions: Facio-Scapulo-Humeral Dystrophy
FSHD2
FSHD1
Keywords: fshd
creatine monohydrate
children
pediatrics
Facio-Scapulo-Humeral Dystrophy
Lead sponsor: Murdoch Childrens Research Institute
Sponsor class: OTHER
First posted: 2016-10-28
Enrollment: 11
Sex: ALL
Minimum age (years): 5
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: Motor Function Measure for Neuromuscular disease
timeFrame: 3 months
Secondary outcomes:
measure: Muscle Magnetic Resonance Imaging
timeFrame: 3 months
measure: Muscle Ultrasound Scan
timeFrame: 3 months
measure: Performance of the Upper Limb Measure
timeFrame: 3 months
measure: ACTIVILIM
timeFrame: 3 Months
measure: PedsQL Neuromuscular
timeFrame: 3 months
measure: FSH-COM
timeFrame: 3 months
measure: FSH-Health Index (Pediatric Version)
timeFrame: 3 months
measure: Six Minute Walk Test
timeFrame: 3 months
measure: FSHD Severity Score
timeFrame: 3 months
measure: Quantitative muscle strength testing
timeFrame: 3 months
measure: GPX3 Level
timeFrame: 3 months
measure: Step Counter
timeFrame: 3 months
measure: Laboratory safety monitoring
timeFrame: 3 months
Other outcomes:
Locations:
city: Melbourne
country: Australia
facility: The Royal Children's Hospital
state: Victoria
Study officials:
affiliation: Murdoch Children Research Institute/Royal Children Hospital, Melbourne
name: Ian R Woodcock, MBBS
role: PRINCIPAL_INVESTIGATOR
affiliation: Murdoch Children Research Institute/Royal Children Hospital, Melbourne
name: Monique M Ryan
role: PRINCIPAL_INVESTIGATOR
References: