Effect of creatine monohydrate on motor function in children with facioscapulohumeral muscular dystrophy: A multicenter, randomized, double-blind placebo-controlled crossover trial
Woodcock IR, de Valle K, Cairns A, Davidson ZE, Kean M, Varma N, Grobler A, Metz D, Carroll K, Dilek N, Heatwole C, Ryan MM, Delatycki MB, Yiu EM
Pharmacotherapy · 4 citations
Review labels
Neutral facts our review recorded about how this study was done. They describe method, never whether we like the result.
How it was studied
- Design
- Randomized controlled trial (indexed by PubMed)
- Studied in
- People
- Main outcome
- Health markers and function
Who paid for it
- Funding
- Independent funding
- Nonprofit
- FSHD Global Research Foundation
- Authors
- At least one author declares a financial tie to industry
Based on 1 listed funder(s) and full-text disclosure statement.
Publication
- Published
- 2025-05-14 · Pharmacotherapy · vol. 45 · issue 6 · pp. 341–351
- Publisher
- Wiley
- Cited
- 4 citations · more than 79% of similar papers · 1.4× the field average
- References
- 62 works
- Access
- Open access (hybrid journal) · CC-BY-NC
- Research areas
- Muscle Physiology and Disorders · Nutrition and Health in Aging · Muscle metabolism and nutrition
- Keywords
- Facioscapulohumeral muscular dystrophy, Crossover study, Medicine, Placebo, Muscular dystrophy, Physical therapy, Confidence interval, Randomized controlled trial, Creatine, Internal medicine, Pathology
- MeSH
- humans, muscular dystrophy, facioscapulohumeral, creatine, magnetic resonance imaging, treatment outcome, cross-over studies, double-blind method, adolescent, child, female, male, muscle strength
14 authors
From AU, US
- Ian R. Woodcock · correspondingRoyal Children's Hospital; The University of Melbourne; Murdoch Children's Research Institute
- Katy de ValleRoyal Children's Hospital; The University of Melbourne; Murdoch Children's Research Institute
- Anita G. CairnsChildren's Health Queensland Hospital and Health Service
- Zoe E. DavidsonRoyal Children's Hospital; Murdoch Children's Research Institute; Monash University
- Michael J. KeanRoyal Children's Hospital
- Nisha VarmaRoyal Children's Hospital
Abstract
Background
Facioscapulohumeral muscular dystrophy (FSHD) is a rare, progressive muscle disease with no available disease-modifying therapy. Creatine monohydrate (CrM) has been shown to improve muscle strength in individuals with muscular dystrophies but has not been tested in young people with FSHD. This study aimed to explore the efficacy of CrM on motor function in children with FSHD.
Methods
In a randomized placebo-controlled double-blind crossover trial, powdered CrM at a dose of 100 mg/kg/day (maximum 10 g daily) was compared with placebo in two 12-week treatment periods with a 6-week washout between crossover arms. The primary outcome measure was the Motor Function Measure for Neuromuscular Disease (MFM-32) with secondary outcomes assessing safety, endurance, strength, patient-reported outcome measures, and muscle morphology measurements as assessed by whole-body magnetic resonance imaging (MRI).
Results
Thirteen children were enrolled (mean (standard deviation, SD) 12.2 (2.67) years of age) and 11 patients completed both trial treatment periods. In an intention-to-treat analysis, no clinically meaningful difference was seen between treatment groups as measured by the mean difference in MFM-32 (0.19, 95% confidence interval (CI) -0.71 to 1.08). However, there was an improvement in 6-minute walk distance of 27.74 m (95% CI -1.41 to 56.88) and trends to improvement in the FSHD-Composite Outcome Measure for Pediatrics (FSHD-COM Peds), 10 meter walk/run, and in MRI measures. There were no serious adverse events. Serum creatinine increased by a mean 12.63 μmol/L (95% CI 1.14 to 24.12) post-CrM treatment, though this was presumed to reflect increased creatinine production. No participants discontinued CrM due to adverse events.
Conclusion
CrM is safe and well tolerated in children with FSHD. Although CrM had no effect on motor function as measured by the MFM-32 compared with placebo, there were trends toward improvement in the 6-minute walk distance and other secondary outcome measures. This study confirms the feasibility of conducting clinical trials in children with FSHD. Further assessment of the efficacy of CrM in pediatric FSHD is warranted in a larger randomized controlled clinical trial. Future studies may benefit from stratifying population cohorts according to functional ability or by MRI fat infiltration measurements.
Abstract via Europe PMC. Copyright remains with the authors or publisher (CC BY-NC).
Community trust
Loading…
How much do you trust this study's findings?
Comments
Sign in to rate, comment on or flag this study.Sign inSomething wrong here?
Flag this study if its information, labels or funding look wrong. An editor reviews every flag.
Sign in to rate, comment on or flag this study.Sign inEducational information about published research. Not medical advice, and not a recommendation to start or stop anything.