A Phase 2A Randomized, Placebo Controlled Trial of EPI-743 in Children With Rett Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 24
- 试验地点
- 1
- 主要终点
- Rett Syndrome Clinical Severity Sore
研究概览
简要总结
Rett syndrome is a severe neurodevelopmental disorder that primarily affects female children. Rett syndrome is characterized by significant elevation in blood markers of oxidative stress. EPI-743 is a novel therapeutic with demonstrated efficacy and safety in the treatment of disorders characterized by oxidative stress. The purpose of this study is to examine the safety and efficacy of EPI-743 in a population of children with Rett syndrome.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- — 至 18 Years（Child, Adult）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of Rett syndrome with disease stage 1-2
- •Abnormality of at least two disease biomarker levels
- •Confirmed MeCP2 mutation
- •Patient or patient's guardian able to consent and comply with protocol requirements
- •Abstention from use of Coenzyme Q10, vitamin E and Idebenone two weeks prior to enrollment into the study
排除标准
- •Any condition, which in the opinion of the investigator could compromise the subject's safety or adherence to treatment with EPI-
- •Clinically significant allergy or hypersensitivity to EPI-743 or to any of the excipients of with EPI-743 (eg., sesame oil).
- •Clinically significant allergy or hypersensitivity to Vitamin E
- •Lack of confirmation of MeCP2 mutation
- •Clinical history of bleeding or abnormal baseline PT/PTT
- •Diagnosis of any other concurrent inborn error of metabolism
- •Hepatic insufficiency with LFTs greater than 3 times upper limit of normal
- •Renal insufficiency requiring dialysis
- •End stage cardiac failure
- •Fat malabsorption syndromes precluding drug absorption
研究组 & 干预措施
EPI-743 15 mg/kg
Subjects in this arm will receive EPI-743 at a dose of 15 mg/kg three times daily
干预措施: EPI-743 (Drug)
Placebo
Subjects in this arm will receive placebo at a volume equivalent to the volume of EPI-743 they would receive if in active group based on their weight
干预措施: Placebo (Drug)
结局指标
主要结局
Rett Syndrome Clinical Severity Sore
时间窗: Change at six months from baseline
Measure of disease progression
次要结局
- Head circumference(Change at six months from baseline)
- Oxidative Stress Biomarkers(Change at six months from baseline)
- Rett syndrome behavioral questionnaire(Change at six months from baseline)
- PedsQL(Change at six months from baseline)
- Number of Drug-related adverse and serious adverse events(Six months)
- Respiratory Disturbance Index (RDI)(Change at six months from baseline)