A Study to Evaluate Ketamine for the Treatment of Rett Syndrome

Summary

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.

Timeline

Start

2019-03-12

Primary completion

2021-11-08

Completion

2021-11-22

Publications

• Campbell K, Neul JL, Lieberman DN, Berry-Kravis E, Benke TA, Fu C, Percy A, Suter B, Morris D, Carpenter RL, Marsh ED, von Hehn J. A randomized, placebo-controlled, cross-over trial of ketamine in Rett syndrome. J Neurodev Disord. 2025 Jan 24;17(1):4. doi: 10.1186/s11689-025-09591-y.

Drugs

Indications