A Study to Evaluate Ketamine for the Treatment of Rett Syndrome

• Status: Active, not recruiting
• Trial end date: 2021-11-01
• Target enrollment: 0
• Participant gender: Female

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Rett Syndrome Research Trust
• Collaborators: Vanderbilt University; Vanderbilt University Medical Center
• Treatments: Ketamine

Criteria

Inclusion Criteria:

• Female Rett Syndrome patients diagnosed with Rett Syndrome with a confirmed MECP2 mutation

• between the ages of 6 and 12, inclusive, who have not achieved menarche

• ability to take oral medications

• are generally healthy.

Exclusion Criteria:

• Patients not on stable medication regimens/other types of behavioral, educational, or dietary interventions for at least 4 weeks,

• are taking medications that may interact with ketamine,

• have a condition where increased blood pressure, spinal fluid pressure, or ocular pressure may put the patient at increased risk.