SMART02.

Trial name or title	Multicenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophy: confirmatory trial (SMART02)
Methods	Phase IIB, randomised, double‐blind, placebo‐controlled trial
Participants	28 participants with SMA types I and II, aged 1–7 years
Interventions	Oral valproic acid or placebo, 12.5 mg/kg or 25 mg/kg once a day after supper. Treatment period: 40 weeks
Outcomes	HFMSE, HFMS, motor function, WHO motor milestones
Starting date	January 2016
Contact information	Kayoko Saito, Institute of Medical Genetics, Tokyo Women's Medical University, Japan
Notes	Study ongoing and recruiting participants