Long-Term Safety and Efficacy Evaluation of Vatiquinone (EPI-743) in Children With Leigh Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 30
- 试验地点
- 4
- 主要终点
- Disease Severity as Measured by Newcastle Pediatric Mitochondrial Disease Scale (NPMDS) Sections 1-3 Score
研究概览
简要总结
EPI-743 in Leigh syndrome participants that participated in previous EPI743-12-002 (NCT01721733) study.
详细描述
To monitor the long-term safety and neurodevelopmental effects of EPI-743 on children who complete the EPI743-12-002 placebo-controlled trial.
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
- 1 Year 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of Leigh syndrome with genetic confirmation
- •Completion of EPI743-12-002 protocol and initiation of treatment within 14 days of Month 12 visit in EPI743-12-002 study
- •Participant or participant's guardian able to consent and comply with protocol requirements
- •Continued abstention from supplements excluded in EPI743-12-002 study
- •Botox® is allowed if approved by the sponsor
排除标准
- •Allergy to EPI-743 or sesame oil
- •Allergy to vitamin E
- •Clinical history of bleeding or abnormal baseline prothrombin time (PT)/partial thromboplastin time (PTT)
- •Hepatic insufficiency with liver function tests (LFTs) greater than two times upper limit of normal
- •Renal insufficiency requiring dialysis
- •End-stage cardiac failure
- •Fat malabsorption syndromes precluding drug absorption
- •Use of anticoagulant medications
- •Participation in other clinical research studies/taking other experimental agents
- •Participation in elective procedures that required sedation
研究组 & 干预措施
EPI-743
Participants will receive EPI-743 at a dose of 15 milligrams/kilogram (mg/kg) up to a total 200 mg 3 times daily (TID) orally with meal or by using nasogastric (NG) or gastrostomy feeding tubes with liquid food for at least 36 months.
干预措施: EPI-743 (Drug)
结局指标
主要结局
Disease Severity as Measured by Newcastle Pediatric Mitochondrial Disease Scale (NPMDS) Sections 1-3 Score
时间窗: Baseline up to Month 36
NPMDS is a validated scale to assess the mitochondrial disease progression.
Number of Participants With Dose-Limiting Serious Adverse Events (SAEs)
时间窗: Baseline up to Month 36
次要结局
- Gross Motor Function Measure(Baseline up to Month 24)
- Awake Oxygen Saturation Levels(Baseline up to Month 24)
- Barry-Albright Dystonia Scale Score(Baseline up to Month 24)
- Bayley Scales of Infant Development-III Score (Participants Age 0-3)(Baseline up to Month 24)
- Wechsler Scale of Intelligence and Movement Assessment Battery Score for Children (Participants Age 4-18)(Baseline up to Month 24)
- Total Ventilator Days and Total Intensive Care Unit Days(Baseline up to Month 24)
- Number of Participants With Pneumonia Episodes and Tracheostomy(Baseline up to Month 24)
- Total Number of Mortalities, Medical Encounters, Hospitalizations, and Hospital Days(Baseline up to Month 24)
- Health-Related Quality of Life as Measured by NPMDS Section 4 Score(Baseline up to Month 24)
- Glutathione Cycle Biomarkers Level(Baseline up to Month 24)