A randomised, double-blind, placebo-controlled trial was conducted in 13 children with facioscapulohumeral muscular dystrophy (FSHD) who received either creatine monohydrate or a 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.
