Randomized controlled trial of sulforaphane and metabolite discovery in children with Autism Spectrum Disorder.

The study had a relatively small sample size of 45 children, which limits the power to detect small differences. The primary outcome (clinician-rated severity) was null, while positive effects were found on secondary caregiver-reported measures, which are more susceptible to placebo effects. Children with severe ASD were overrepresented in the treatment group, and when these children were excluded from the analysis, the effect sizes for the primary outcome became larger and more positive, though still not significant. The trial design included an open-label phase where all children received the drug, which complicates the interpretation of long-term improvements since there was no control group for that period. The source of sulforaphane was a broccoli seed extract with an enzyme, which may have variable bioavailability compared to pure sulforaphane.

The study was funded by the U.S. Department of Defense. The sulforaphane tablets were provided by Nutramax, a company that sells the product. The authors declared no other specific conflicts, but the funding source and product provision are relevant to the interpretation of the results.

Do not conclude that sulforaphane treats core autism symptoms. The primary measure of autism severity showed no significant difference between the drug and placebo. The positive results were on secondary behavioral measures reported by caregivers, which can be influenced by expectation, and on biological markers that do not necessarily translate to clinical improvement.