Esketamine for the Treatment of Rett Syndrome
- Conditions
- Rett Syndrome
- Interventions
- Registration Number
- NCT06199700
- Lead Sponsor
- Children's Hospital of Fudan University
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- Female
- Target Recruitment
- 3
- Classic/typical RTT
- Causing mutation in MECP2 gene
- Stable pattern of seizures, or has had no seizures at least 8 weeks
- Use of other drugs that interact with Esketamine: thyroxine, meglumine diatrizoate, aminophylline, diazepam or midazolam, drugs for antihypertension or central nervous depressants, halogenated general anesthetics (e.g., halothane), tubocurarine, atracurium
- Condition with hypertension, high cerebrospinal fluid pressure, and high intraocular pressure
- Unstable systemic illness other than Rett syndrome: current clinically significant cardiovascular, endocrine (such as hypo- or hyperthyroidism, type 1 diabetes, or uncontrolled type 2 diabetes), renal, hepatic, respiratory, or gastrointestinal disease (such as celiac disease or inflammatory bowel disease), or major surgery planned during the study
- Clinically important variations in medication use
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Esketamine treatment Esketamine hydrochloride Esketamine, diluted with 20ml Saline at a dose of 0.25mg/kg, intravenously for 40 minutes, once a week; 5 weeks in total.
- Primary Outcome Measures
Name Time Method Efficacy of treatment with Esketamine in girls with RTT baseline, week 5, and month 6 Rett Syndrome Behaviour Questionnaire (RSBQ) total score; Minimum value 0; Maximum values 90; higher scores mean a worse outcome.
Co-outcome for efficacy of treatment with Esketamine in girls with RTT baseline, week 5, and month 6 Clinical Global Impressions Scale-improvement (CGI-I) score; Minimum value 0; Maximum values 7; higher scores mean a worse outcome.
Incidence of Adverse events baseline, week 5 Safety of treatment with Esketamine in girls with RTT
- Secondary Outcome Measures
Name Time Method Revised-Motor Behavior Assessment scale (R-MBA) baseline, week 5, and month 6 Subscale score, 0-100, higher sores means a worse outcome
Rett Syndrome Severity Scale (RSSS) baseline, week 5, and month 6 Subscale score, 0-21, higher sores means a worse outcome
Behavior observation baseline, week 5, and month 6 Unusual hand movements duration per hour
Brain image change baseline, week 5, and month 6 Magnetic Resonance Imaging (MRI)；brain network connectivity
Autism Diagnostic Observation Schedule, Second Edition (ADOS-2) baseline, week 5, and month 6 Total score，; Minimum value 0; Maximum values 28; higher scores mean a worse outcome.
Griffiths Scales of Child Development baseline, week 5, and month 6 Development quotient; Minimum value 0; Maximum values 96; higher scores mean a better outcome.
Sleep improvement by Esketamine treatment baseline, week 5, and month 6 Sleep record
Brain function change baseline, week 5, and month 6 Electroencephalogram (EEG); brain-wave activity
Trial Locations
- Locations (1)
Xiamen Children's Hospital (Children's Hospital of Fudan University at Xiamen)
🇨🇳Xiamen, Fujian, China