NCT02226458撤回2 期
An Exploratory Open-Label Phase 2 Study of EPI-743 (Vincerinone TM) in Children With Autism Spectrum Disorder
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 主要终点
- Primary Efficacy
研究概览
简要总结
The investigators hypothesize that EPI-743 may provide clinical benefit to children with Autism Spectrum Disorder.
详细描述
An Exploratory Open Label Phase 2 Study of EPI-743 (Vincerinone TM) in Children with Autism Spectrum Disorder
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
- 3 Years 至 14 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of Autism Spectrum Disorder as defined by the DSM-V criteria for ASD and a gold-standard diagnostic evaluation for ASD such as the Autism Diagnostic Observation Schedule (ADOS) and/or the Autism Diagnostic Interview - Revised (ADI-R)
- •Male or female, 3 years to 14 years of age
- •Abnormal glutathione cycle biomarkers (GSH/GSSG in plasma<8.0)
- •Language impairment (as defined by the CELF-2 or CELF-4 screener)
- •Ability to complete language assessment (using either CELF or PLS)
- •Subject or subject's guardian able to consent and comply with protocol requirements
- •Abstention from use of Coenzyme Q10, vitamin E, lipoic acid, folinic acid, other forms of folic acid above recommended daily allowance (RDA), and idebenone 2 months prior to treatment with EPI-743 and for duration of study
- •Stable regimen of medication and supplements for 2 months prior to enrollment and duration of the study
排除标准
- •Allergy to EPI-743 or sesame oil
- •Allergy to vitamin E
- •Clinical history of bleeding or abnormal baseline PT/PTT
- •Use of anticoagulant medications
- •Participation in any other interventional study within 90 days of treatment.
- •Use of antipsychotic medications
- •Moderate to severe positive response on ABC irritability subscale on questions: Injures self on purpose, is aggressive to other children or adults (verbally or physically), deliberately hurts himself/ herself, and/or does physical violence to self
- •Severe impairment as defined as a Vineland Adaptive Behavioral Scales composite standard score of <40
- •Patients with genetic disease that gives rise to ASD (e.g. Rett syndrome, Down syndrome, fragile x syndrome)
- •Abnormal liver function tests two times the upper limit of normal or renal insufficiency with creatinine levels two times the upper limit of normal
研究组 & 干预措施
EPI-743
Experimental
15 mg/kg oral solution three times per day, maximum of 200 mg per dose
干预措施: EPI-743 (Drug)
结局指标
主要结局
Primary Efficacy
时间窗: 6 months
Change in plasma levels of reduced and oxidized glutathione from baseline to six months
次要结局
- Secondary Endpoint(6 months)
- Safety Endpoint(8 months)
研究者
相似试验
已完成
2 期
EPI-743 in Cobalamin C Defect: Effects on Visual and Neurological ImpairmentMethylmalonic Aciduria and Homocystinuria,Cblc TypeRetinopathyGenetic DiseaseNCT01793090Bambino Gesù Hospital and Research Institute30
招募中
2 期
A Study of Esomeprazole in Children With AutismAutism Spectrum DisorderAutismNCT03866668Stanford University25
已完成
不适用
ABC-CT Pre-School Feasibility StudyAutism Spectrum DisorderNCT06413316Yale University50
已完成
不适用
Trial of Vitamin D3 Supplementation in Paediatric AutismAutismNCT02508922University of Dublin, Trinity College42
已完成
2 期
Safety and Efficacy Study of EPI-743 in Children With Leigh SyndromeLeigh SyndromeNCT01721733PTC Therapeutics35