A Phase 2B Randomized, Placebo Controlled, Double Blind Clinical Trial of EPI-743 in Children With Leigh Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 35
- 试验地点
- 4
- 主要终点
- Newcastle Pediatric Mitochondrial Disease Scale (NPMDS) Sections 1-3
研究概览
简要总结
The purpose of this study is to evaluate the effects of EPI-743 in children with Leigh syndrome on disease severity, neuromuscular function, respiratory function, disease morbidity and mortality and disease associated biomarkers.
详细描述
The purpose of this study is to evaluate the effects of EPI-743 in patient with Leigh syndrome on disease severity, neuromuscular function, respiratory function, disease morbidity and mortality and biomarkers associated with the disease.
This study is a six month prospective randomized double-blind, placebo-controlled trial with a six month extension phase of two dose levels of EPI743. The planned enrollment is for approximately 30 children with genetically confirmed Leigh syndrome. After 6 months of treatment, those children that were randomized to the placebo treatment arm will be re-randomized to one of the 2 active treatment arms.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 6 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical and MRI diagnosis of Leigh syndrome
- •Moderate disease severity based on NPMDS score
- •Age under 18 years
- •Documented evidence of disease progression within 12 month of enrollment
- •Availability of MRI that confirms necrotizing encephalopathy
- •Patient or guardian able to consent and comply with protocol requirements
- •Abstention from Coenzyme Q10, Vitamins C & E, lipoic acid and Idebenone
排除标准
- •Allergy to EPI-743, Vitamin E or sesame oil
- •History of bleeding abnormalities or abnormal PT/PTT
- •Diagnosis of concurrent inborn error of metabolism
- •Previous tracheostomy
- •Ventilator dependent or use of noninvasive ventilatory support w/in 1 month of enrollment
- •LFTs greater than 2 times ULN
- •Renal insufficiency
- •End stage cardiac failure
- •Fat malabsorption syndrome
- •Use of anticoagulant medications
- •Abstention from Botox for 6 months prior to enrollment and for duration of study
研究组 & 干预措施
Placebo
Each patient will receive a volume of placebo based on weight
干预措施: Placebo (Drug)
EPI-743 15 mg/kg
Each subjects dose will be based on their weight. 15 mg/kg with a maximum dose of 200 mg per dose, t.i.d., will be administered in this treatment arm.
干预措施: EPI-743 15 mg/kg (Drug)
EPI-743 5 mg/kg
Each subjects dose will be based on their weight. 5 mg/kg with a maximum dose of 100 mg per dose, t.i.d., will be administered in this treatment arm.
干预措施: EPI-743 5 mg/kg (Drug)
结局指标
主要结局
Newcastle Pediatric Mitochondrial Disease Scale (NPMDS) Sections 1-3
时间窗: 6 months
Change from baseline to six months will be compared between subjects in active treatment group and placebo group
次要结局
- Disease morbidity(6 months)
- Mortality(6 months)
- Respiratory function(6 months)
- Neuromuscular function(6 months)
- Glutathione cycle biomarkers(6 months)
- Number of dose limiting serious adverse events(6 months)