Clinical TrialAutism Spectrum Disorder (ASD)KetamineKetaminePlaceboCompleted

A Study to Evaluate Ketamine for the Treatment of Rett Syndrome

Double-blind, placebo-controlled crossover trial (n=23) assessing oral ketamine given twice daily for 5 days (cohort doses 0.75 mg/kg and 1.5 mg/kg) vs placebo in females aged 6–12 with Rett syndrome.

Target Enrollment
23 participants
Study Type
Phase II interventional
Design
Randomized, triple Blind

Detailed Description

Sequential, ascending-dose study with up to four cohorts; each participant receives a 5-day BID course of cohort-specified oral ketamine and a 5-day course of matched placebo in randomised, double-blind crossover order with 2-week safety follow-up after each period.

Primary assessments include safety and tolerability (AEs, vitals, physical exam, concomitant medications); exploratory efficacy via physician and caregiver questionnaires and continuous wearable at-home biosensor data.

Study Protocol

Preparation

sessions

Dosing

2 sessions
7200 min each

Integration

sessions

Study Arms & Interventions

Ketamine 0.75 mg/kg

experimental

Oral ketamine 0.75 mg/kg given BID for 5 days (cohort-specific active treatment); crossover with placebo.

Interventions

  • Ketamine0.75 mg/kg
    via Oral• BID for 5 days• 10 doses total

    Cohort-specified active treatment; participants also receive placebo in crossover.

Ketamine 1.5 mg/kg

experimental

Oral ketamine 1.5 mg/kg given BID for 5 days (cohort-specific active treatment); crossover with placebo.

Interventions

  • Ketamine1.5 mg/kg
    via Oral• BID for 5 days• 10 doses total

    Cohort-specified active treatment; participants also receive placebo in crossover.

Placebo

inactive

Matched oral placebo given BID for 5 days; crossover comparator.

Interventions

  • Placebo
    via Oral• BID for 5 days• 10 doses total

    Matched placebo period; each participant receives both placebo and active in crossover.

Participants

Ages
6 – 12
Sexes
female

Inclusion 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

  • 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 Details

Locations

University of Alabama Birmingham School of Medicine — Birmingham, Alabama, United States
Children's Hospital Colorado — Aurora, Colorado, United States
Rush University Medical Center — Chicago, Illinois, United States
Boston Children's Hospital — Boston, Massachusetts, United States
Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
Vanderbilt University Medical Center — Nashville, Tennessee, United States
Texas Children's Hospital — Houston, Texas, United States

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