This double-blind, placebo-controlled crossover trial (n=12) investigated the effects of oral ketamine in a single 5-day BID regimen on patients with Rett Syndrome, compared to a placebo, in a 4-week cross-over design.
Approximately 12 patients per cohort participated for about 8-10 weeks at seven US study centers. Safety, tolerability, and efficacy were assessed via patient disposition, vital signs, physical examination, adverse events, concomitant medication use, physician and caregiver questionnaires, and continuous, wearable, at-home biosensor data collection.
The study aimed to determine the safety, tolerability, and efficacy of oral ketamine for the treatment of Rett Syndrome. The screening period lasted between 2 and 4 weeks, the cross-over treatment period lasted 4 weeks, and the safety follow-up period lasted 2 weeks.
The study was conducted by the Rett Syndrome Research Trust in collaboration with Vanderbilt University Medical Center. Last updated information was posted on March 27, 2024.
Trial Details
This 2 cohort, sequential, ascending dose study will assess the safety, tolerability and efficacy of oral ketamine dosed in a single 5-day BID regimen in addition to placebo, in a 4-week cross-over design in patients with Rett Syndrome. Approximately 12 patients per cohort are anticipated to participate for approximately 8-10 weeks at approximately 7 US study centers.NCT Number NCT03633058
Papers
Entactogen Effects of Ketamine: A Reverse-Translational StudyThis randomized, double-blind, placebo-controlled study (n=68) assesses the prosocial, entactogen effects of ketamine (35mg/70kg) in participants with treatment-resistant depression (TRD). Ketamine increased pleasure from social interactions and helping others, lasting for one week post-treatment. In a rodent experiment, ketamine-treated rats showed increased protective behaviour towards their cage mates, indicating entactogen effects.