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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
3
Intravenous infusion of Ketamine
Xiamen Children's Hospital (Children's Hospital of Fudan University at Xiamen)
Xiamen, Fujian, China
Efficacy of treatment with Esketamine in girls with RTT
Rett Syndrome Behaviour Questionnaire (RSBQ) total score; Minimum value 0; Maximum values 90; higher scores mean a worse outcome.
Time frame: baseline, week 5, and month 6
Incidence of Adverse events
Safety of treatment with Esketamine in girls with RTT
Time frame: baseline, week 5
Co-outcome for efficacy of treatment with Esketamine in girls with RTT
Clinical Global Impressions Scale-improvement (CGI-I) score; Minimum value 0; Maximum values 7; higher scores mean a worse outcome.
Time frame: baseline, week 5, and month 6
Autism Diagnostic Observation Schedule, Second Edition (ADOS-2)
Total score,; Minimum value 0; Maximum values 28; higher scores mean a worse outcome.
Time frame: baseline, week 5, and month 6
Griffiths Scales of Child Development
Development quotient; Minimum value 0; Maximum values 96; higher scores mean a better outcome.
Time frame: baseline, week 5, and month 6
Revised-Motor Behavior Assessment scale (R-MBA)
Subscale score, 0-100, higher sores means a worse outcome
Time frame: baseline, week 5, and month 6
Rett Syndrome Severity Scale (RSSS)
Subscale score, 0-21, higher sores means a worse outcome
Time frame: baseline, week 5, and month 6
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Sleep improvement by Esketamine treatment
Sleep record
Time frame: baseline, week 5, and month 6
Behavior observation
Unusual hand movements duration per hour
Time frame: baseline, week 5, and month 6
Brain image change
Magnetic Resonance Imaging (MRI);brain network connectivity
Time frame: baseline, week 5, and month 6
Brain function change
Electroencephalogram (EEG); brain-wave activity
Time frame: baseline, week 5, and month 6