A randomized, placebo-controlled, cross-over trial of ketamine in Rett syndrome.
Journal of neurodevelopmental disorders January 24, 2025 Kathleen Campbell, Jeffrey L Neul, David N Lieberman et al. 5 citations
In a double-blind, placebo-controlled crossover trial, low-dose oral ketamine was safe and well tolerated over five days in girls aged 6–12 with Rett syndrome. Twenty-three participants enrolled; one was excluded and one withdrew due to vomiting. No clinical improvement in Rett symptoms was observed with ketamine compared to placebo, despite electroencephalography showing expected increases in high-frequency brain activity, confirming the drug engaged its target. The trial was stopped after two dose cohorts (0.75 and 1.5 mg/kg twice daily) because of pandemic-related enrollment difficulties. Further research with higher doses or longer treatment is needed.