Results of a trial of creatine monohydrate in a paediatric population with FSHD

A randomised, double-blind, placebo-controlled trial was conducted in 13 children with facioscapulohumeral muscular dystrophy (FSHD) who received either creatine monohydrate or a placeboLe placebo est un produit dont la présentation est identique à celle d’un médicament, mais qui ne contient pas de principe actif. Dans un essai clinique, un placebo est utilisé pour mesurer l’action réelle du médicament testé, en comparant les effets du médicament testé contenant le principe actif et ceux du placebo. in a crossover design over two 12-week periods, separated by a 6-week treatment-free period.

• Eleven participants completed the trial.
• No significant difference between creatine monohydrate and placebo was observed on the MFM-32 score.
• An improvement in the 6-minute walk distance (by 27.74 m) and a trend towards improvement in the paediatric composite score (FSHD-COM Peds), 10 m walk/run and MRI measurements were reported.
• Creatine monohydrate was well tolerated by participants.

However, this study confirms that clinical trials can be conducted in the paediatric FSHD population.

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 et al. Pharmacotherapy. 2025 Jun.