Double-blind, placebo-controlled crossover trial (n=23) assessing oral ketamine given twice daily for 5 days (cohort doses 0.75 mg/kg and 1.5 mg/kg) vs placebo in females aged 6–12 with Rett syndrome.
Sequential, ascending-dose study with up to four cohorts; each participant receives a 5-day BID course of cohort-specified oral ketamine and a 5-day course of matched placebo in randomised, double-blind crossover order with 2-week safety follow-up after each period.
Primary assessments include safety and tolerability (AEs, vitals, physical exam, concomitant medications); exploratory efficacy via physician and caregiver questionnaires and continuous wearable at-home biosensor data.
Oral ketamine 0.75 mg/kg given BID for 5 days (cohort-specific active treatment); crossover with placebo.
Cohort-specified active treatment; participants also receive placebo in crossover.
Oral ketamine 1.5 mg/kg given BID for 5 days (cohort-specific active treatment); crossover with placebo.
Cohort-specified active treatment; participants also receive placebo in crossover.
Matched oral placebo given BID for 5 days; crossover comparator.
Matched placebo period; each participant receives both placebo and active in crossover.