Randomized, Placebo-Controlled Trial of Creatine in Children With Spinal Muscular Atrophy

ABSTRACT Objective: To determine whether oral creatine produces positive changes in any of 4 outcomes in children with spinal muscular atrophy (SMA). Methods: The authors conducted a randomized, double-blind, placebo-controlled trial on 55 patients aged 2-18 years with SMA. Patients aged younger than 5 years received 2 g/day of creatine/placebo for 6 months. Patients aged 5 years and older received 5 g/day. The primary outcome measure was the Gross Motor Function Measure (GMFM). Secondary outcome measures were Quantitative Muscle Testing (QMT), Parent Questionnaire for the PedsQL™ Neuromuscular Module (QOL), and Pulmonary Function Tests (PFT). Results: Forty of the 55 patients completed the protocol. There was no significant difference in the 4 outcome measures between creatine and placebo groups. Conclusions: Under the experimental conditions of our study, creatine supplementation for 6 months did not improve motor function, muscle strength, pulmonary function, or quality of life in children with SMA,