Rett Syndrome Clinical Trial
Official title:
An Exploratory Trial of Esketamine for the Treatment of Rett Syndrome
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.
Status | Recruiting |
Enrollment | 3 |
Est. completion date | May 1, 2025 |
Est. primary completion date | October 31, 2024 |
Accepts healthy volunteers | No |
Gender | Female |
Age group | 5 Years to 10 Years |
Eligibility | Inclusion Criteria: - Classic/typical RTT - Causing mutation in MECP2 gene - Stable pattern of seizures, or has had no seizures at least 8 weeks Exclusion Criteria: - 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 |
Country | Name | City | State |
---|---|---|---|
China | Xiamen Children's Hospital (Children's Hospital of Fudan University at Xiamen) | Xiamen | Fujian |
Lead Sponsor | Collaborator |
---|---|
Children's Hospital of Fudan University |
China,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Efficacy of treatment with Esketamine in girls with RTT | Rett Syndrome Behaviour Questionnaire (RSBQ) total score | baseline, week 5, and month 6 | |
Primary | Incidence of Adverse events | Safety of treatment with Esketamine in girls with RTT | baseline, week 5 | |
Primary | Co-outcome for efficacy of treatment with Esketamine in girls with RTT | Clinical Global Impressions Scale-improvement (CGI-I) score | baseline, week 5, and month 6 | |
Secondary | Autism Diagnostic Observation Schedule, Second Edition (ADOS-2) | Total score | baseline, week 5, and month 6 | |
Secondary | Griffiths Scales of Child Development | Development quotient | baseline, week 5, and month 6 | |
Secondary | Revised-Motor Behavior Assessment scale (R-MBA) | Subscale score, 0-100, higher sores means a worse outcome | baseline, week 5, and month 6 | |
Secondary | Rett Syndrome Severity Scale (RSSS) | Subscale score, 0-21, higher sores means a worse outcome | baseline, week 5, and month 6 | |
Secondary | Sleep improvement by Esketamine treatment | Sleep record | baseline, week 5, and month 6 | |
Secondary | Behavior observation | Unusual hand movements duration per hour | baseline, week 5, and month 6 | |
Secondary | Magnetic Resonance Imaging (MRI) | Brain network connectivity | baseline, week 5, and month 6 | |
Secondary | Electroencephalogram (EEG) | Abnormal brain-wave activity | baseline, week 5, and month 6 |
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