SMART02 2016.

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, placebo‐controlled, randomised, double‐blind trial
Participants	28 patients with SMA types I and II, age 1 to 7 years
Interventions	Oral valproic acid or placebo, 12.5 mg/kg or 25 mg/kg once a day after supper. Treatment period is 40 weeks
Outcomes	HFMSE, HFMS, motor function, World Health Organization motor milestones
Starting date	January 2016
Contact information	Kayoko Saito, Institute of Medical Genetics, Tokyo Women's Medical University
Notes	None

HFMS(E): Hammersmith Functional Motor Scale (Expanded); SMA: spinal muscular atrophy