A Study to Evaluate Ketamine for the Treatment of Rett Syndrome

Brief Title

A Study to Evaluate Ketamine for the Treatment of Rett Syndrome

Official Title

A Randomized, Double-blind, Placebo-controlled, Cross-over Study to Assess the Safety, Tolerability and Efficacy of Oral Ketamine for Patients With Rett Syndrome

Brief 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.
    

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 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.
    

Study Phase

Phase 2

Study Type

Interventional

Primary Outcome

Dose-Limiting Adverse Events

Secondary Outcome

 Clinical Global Impression of Improvement

Condition

Rett Syndrome

Intervention

Ketamine

Study Arms / Comparison Groups

 0.75 mg/kg

Description:  ketamine will be dosed orally twice daily for 5 days at 0.75 mg/kg, in addition to 5 days of placebo

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Status

Drug

Estimated Enrollment

24

Start Date

March 12, 2019

Completion Date

November 22, 2021

Primary Completion Date

November 8, 2021

Eligibility 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.
      

Gender

Female

Ages

6 Years - 12 Years

Accepts Healthy Volunteers

No

Contacts

Jeffrey Neul, MD, PhD, , 

Location Countries

United States

Location Countries

United States

NCT ID

NCT03633058

Organization ID

Ket-101-RSRT

Responsible Party

Sponsor

Study Sponsor

Rett Syndrome Research Trust

Collaborators

 Vanderbilt University Medical Center

Study Sponsor

Jeffrey Neul, MD, PhD, Principal Investigator, Vanderbilt University Medical Center

Verification Date

April 2022