Esketamine for the Treatment of Rett Syndrome

This interventional trial (n=3) will assess the efficacy and safety of esketamine for the treatment of Rett Syndrome (RTT) in children aged 5 to 10 years.

Participants will receive a weekly intravenous infusion of esketamine for five weeks. The primary goal is to determine if Esketamine can improve symptom severity for RTT and if it is safe for use in this population.

The trial, conducted by the Children’s Hospital of Fudan University in China, started in November 2023 and is expected to be completed by May 2025.

The study will use various outcome measures, including the Rett Syndrome Behaviour Questionnaire (RSBQ) total score, Clinical Global Impressions Scale-improvement (CGI-I) score, Autism Diagnostic Observation Schedule, Second Edition (ADOS-2) total score, Griffiths Scales of Child Development, Revised-Motor Behavior Assessment scale (R-MBA), Rett Syndrome Severity Scale (RSSS), sleep improvement records, behavioural observations, magnetic resonance imaging (MRI), and electroencephalogram (EEG).

Compound Ketamine
Country China
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