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
Visit trial
Status Recruiting
Results Published No
Start date 15 November 2023
End date 01 May 2025
Phase Phase I
Design Open
Type Interventional
Generation Second
Participants 3
Sex Female
Age 5- 10
Therapy No

Trial Details

The goal of this interventional study is to learn about the efficacy and safety of Esketamine for treating children with Rett syndrome (RTT). The main questions it aims to answer are: - whether Esketamine treatment is effective in improving symptom severity for RTT. - whether Esketamine is safe in the treatment of RTT. Participants will receive a weekly intravenous infusion of Esketamine for five weeks and will be assessed for disease severity and drug safety.

NCT Number NCT06199700

Data attribution

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