An Exploratory Trial of Esketamine for the Treatment of Rett Syndrome
试验速览
- 阶段
- 早期 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 3
- 试验地点
- 1
- 主要终点
- Efficacy of treatment with Esketamine in girls with RTT
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 5 Years 至 10 Years（Child）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •Classic/typical RTT
- •Causing mutation in MECP2 gene
- •Stable pattern of seizures, or has had no seizures at least 8 weeks
排除标准
- •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
研究组 & 干预措施
Esketamine treatment
Esketamine, diluted with 20ml Saline at a dose of 0.25mg/kg, intravenously for 40 minutes, once a week; 5 weeks in total.
干预措施: Esketamine hydrochloride (Drug)
结局指标
主要结局
Efficacy of treatment with Esketamine in girls with RTT
时间窗: 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
时间窗: 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
时间窗: baseline, week 5
Safety of treatment with Esketamine in girls with RTT
次要结局
- Revised-Motor Behavior Assessment scale (R-MBA)(baseline, week 5, and month 6)
- Rett Syndrome Severity Scale (RSSS)(baseline, week 5, and month 6)
- Behavior observation(baseline, week 5, and month 6)
- Brain image change(baseline, week 5, and month 6)
- Autism Diagnostic Observation Schedule, Second Edition (ADOS-2)(baseline, week 5, and month 6)
- Griffiths Scales of Child Development(baseline, week 5, and month 6)
- Sleep improvement by Esketamine treatment(baseline, week 5, and month 6)
- Brain function change(baseline, week 5, and month 6)