A Single Patient Trial of Fluoxetine in KCNC1-related Disorder
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 1
- 试验地点
- 1
- 主要终点
- Motor development
研究概览
简要总结
This is a single patient study of oral powdered fluoxetine to target developmental outcomes in a child with KCNC1-related disorder. This trial will be conducted at Holland Bloorview Kids Rehabilitation Hospital over 32 to 42 weeks, using a quasi experimental ABA phase design (placebo-fluoxetine-placebo) with randomized and blinded active treatment start and stop moments.
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
盲法说明
This quasi experimental design involves masking of transition moments, but the investigator and participant are aware of the order of cross-over.
入排标准
- 年龄范围
- — 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Consent provided by substitute decision maker.
- •In good general health as evidenced by medical history
- •Screening baseline bloodwork (or availability of clinical bloodwork within 3 months of trial start) with values below relevant cut-offs for adequate hepatic and renal function, and baseline electrolytes including potassium within normal range.
- •Ability to take oral medication and be willing to adhere to the daily oral medication regimen
排除标准
- •Current use of monoamine oxidase inhibitors, other selective serotonin reuptake inhibitors, tricyclic antidepressants, or agents that strongly affect metabolism via CYP2D6, CYP2C9 or CYP3A
- •Hypersensitivity to fluoxetine or to any ingredient in the formulation, including any non-medicinal ingredient, or component of the container
- •Treatment with another investigational drug or other medication intervention within 8 weeks of starting the trial.
- •Any condition or diagnosis, that could in the opinion of the Principal Investigator or delegate interfere with the participant's ability to comply with study instructions, might confound the interpretation of the study results, or put the participant at risk.
- •Long QT syndrome including acquired long QT syndrome (e.g., due to concomitant use of a drug that prolongs the QT); a family history of QT prolongation; or other clinical conditions that predispose to arrhythmias.
研究组 & 干预措施
Placebo-Fluoxetine-Placebo
placebo (medical grade cornstarch in a gel capsule, 1 capsule daily), followed by blinded randomized cross-over moment to active fluoxetine (2.5 then 5 mg po daily), followed by blinded randomized cross-over moment back to placebo
干预措施: Fluoxetine (Drug)
结局指标
主要结局
Motor development
时间窗: Weekly from date of randomization to up to 42 weeks
Parent report on the Early Motor Questionnaire
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
时间窗: From date of randomization to up to 42 weeks
Adverse event reporting
次要结局
- Adaptive skills(Week 1, 13, 29 and 37)
- Cognitive skills(Week 1, 13, 29 and 37)
- Family priority outcome targets(Weekly from date of randomization to up to 42 weeks)
- Clinical Global Impression- Improvement Scale (CGI-I)(Every 4 weeks from date of randomization to up to 42 weeks)