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Completed

NCT Number: NCT02948244

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

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.

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Key information

Conditions

Facio-Scapulo-Humeral Dystrophy Congenital, Hereditary, and Neonatal Diseases and Abnormalities FSHD1 FSHD2 Genetic Diseases, Inborn Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophy, Facioscapulohumeral Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Age range

5 year–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

The Royal Children's Hospital

Melbourne, Victoria, 3052, Australia

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Creatine Monohydrate

Dietary Supplement

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

Dietary Supplement

Placebo

Primary outcomes

  1. Motor Function Measure for Neuromuscular disease

    Time frame: 3 months

    Composite functional outcome measure

Secondary outcomes

  1. Muscle Magnetic Resonance Imaging

    Time frame: 3 months

  2. Muscle Ultrasound Scan

    Time frame: 3 months

  3. Performance of the Upper Limb Measure

    Time frame: 3 months

  4. ACTIVILIM

    Time frame: 3 Months

  5. PedsQL Neuromuscular

    Time frame: 3 months

  6. FSH-COM

    Time frame: 3 months

    FSHD specific composite measure

  7. FSH-Health Index (Pediatric Version)

    Time frame: 3 months

    Patient reported outcome measure specific for patients with FSHD.

  8. Six Minute Walk Test

    Time frame: 3 months

  9. FSHD Severity Score

    Time frame: 3 months

  10. Quantitative muscle strength testing

    Time frame: 3 months

  11. GPX3 Level

    Time frame: 3 months

    Possible biomarker of disease severity in FSHD

  12. Step Counter

    Time frame: 3 months

    Physical activity measure

  13. Laboratory safety monitoring

    Time frame: 3 months

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

Sponsors and collaborators

Lead sponsor

Murdoch Childrens Research Institute

Other

Registry information

Official study 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

Important dates

Study start
2017
Primary completion
2021
Study completion
2022
First posted
Oct 28, 2016
Registry last updated
Feb 8, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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