A Study to Evaluate Ketamine for the Treatment of Rett Syndrome

Purpose

This 4 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 and up to approximately 48 patients in total are anticipated to participate for approximately 8-10 weeks at approximately 7 US study centers.

Condition

  • Rett Syndrome

Eligibility

Eligible Ages
Between 6 Years and 12 Years
Eligible Genders
Female
Accepts Healthy Volunteers
No

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.

Study Design

Phase
Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Crossover Assignment
Intervention Model Description
Patients will receive placebo and ketamine for 5 days BID each, in a double-blind treatment order, and will be assessed for 2 weeks after each treatment.
Primary Purpose
Treatment
Masking
Triple (Participant, Care Provider, Investigator)
Masking Description
double-blind

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
0.75 mg/kg
ketamine will be dosed orally twice daily for 5 days at 0.75 mg/kg, in addition to 5 days of placebo
  • Drug: Ketamine
    oral ketamine dosed twice daily for 5 days
    Other names:
    • Ketalar
Experimental
1.5 mg/kg
ketamine will be dosed orally twice daily for 5 days at 0.75 mg/kg, in addition to 5 days of placebo
  • Drug: Ketamine
    oral ketamine dosed twice daily for 5 days
    Other names:
    • Ketalar
Experimental
3.0 mg/kg
ketamine will be dosed orally twice daily for 5 days at 3.0 mg/kg, in addition to 5 days of placebo
  • Drug: Ketamine
    oral ketamine dosed twice daily for 5 days
    Other names:
    • Ketalar
Experimental
4.5 mg/kg
ketamine will be dosed orally twice daily for 5 days at 4.5 mg/kg, in addition to 5 days of placebo
  • Drug: Ketamine
    oral ketamine dosed twice daily for 5 days
    Other names:
    • Ketalar

Recruiting Locations

University of Alabama Birmingham School of Medicine
Birmingham, Alabama 35294
Contact:
Meagan Whatley
205-934-2091
mwhatley@uabmc.edu

More Details

Status
Recruiting
Sponsor
Rett Syndrome Research Trust

Study Contact

Jana von Hehn, PhD
203-444-1587
jana@rsrt.org

Detailed Description

This study is designed to assess oral ketamine for the treatment of Rett Syndrome and consists of up to 4 ascending dose cohorts, each assessing 1 dose level of ketamine vs placebo. Patients will receive in either order, a 5-day BID regimen of both placebo and the cohort-specified dose level of oral ketamine. Patients may only participate in 1 cohort. Safety and tolerability will be assessed via patient disposition, vital signs, physical examination, adverse events and concomitant medication use. Efficacy will be assessed via physician and caregiver questionnaires and assessments, and continuous, wearable, at-home biosensor data collection. An independent safety committee will review safety data from each cohort to determine if the subsequent ascending dose cohort is warranted. A total of 12 patients per cohort and up to 48 patients in total is anticipated at approximately 7 sites. The screening period will last between 2 and 4 weeks, the cross-over treatment period will last 4 weeks, and the safety follow-up period will last 2 weeks. Total patient participation is approximately 8-10 weeks.