NCT01671384.

Trial name or title	Randomised placebo‐controlled trial of valproic acid and levocarnitine in children with spinal muscular atrophy aged 2–15 years
Methods	Randomised, double‐blind, placebo‐controlled trial
Participants	60 participants with SMA types II or III, aged 2–15 years at time of inclusion Diagnosis of SMA has been made by the presence of exon7 deletion of SMNT gene OR by normal/mildly elevated creatine phosphokinase with electrodiagnostic characteristics suggestive of neurogenic weakness, normal motor and sensory nerve conduction velocities and muscle biopsy showing neurogenic atrophy or evidence of reinnervation (or both)
Interventions	Valproic acid (dose unknown) and levocarnitine 50 mg/kg/day (maximum 1000 mg) divided over 2 doses combined with physiotherapy or placebo combined with physiotherapy
Outcomes	Change in muscle strength (MMT on a 5‐point scale), change in modified HFMS, change in FVC, adverse effects, changes in haematology, liver function and valproic acid levels
Starting date	August 2013 Estimated completion date December 2016
Contact information	G Sheffali, MD, Additional Professor Department of Pediatrics, All India Institute of Medical Sciences, New Delhi, India
Notes	Study ongoing and recruiting participants