Double-Blind, Placebo-Controlled, Crossover Study of Glutathione, Vitamin C and Cysteine in Children With Autism and Severe Behavior Problems
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 24
- 试验地点
- 1
- 主要终点
- Improvement in both developmental skills and behavior with either glutathione or glutathione, Vitamin C and N-acetylcysteine therapy as compared to placebo therapy. Subjects will also be monitored using clinical and laboratory safety parameters.
研究概览
简要总结
This is a double-blind, placebo-controlled crossover study to evaluate the safety and efficacy of glutathione alone or glutathione, vitamin C and NAC treatment in children with autism who also have severe behavior problems. The investigators hypothesis is that children with autism will show improvement in both learning capabilities and behavior with either glutathione, or glutathione, vitamin C and NAC therapy.
详细描述
This is a prospective, single center, double-blind, randomized pilot study in children and adolescents with autism and severe behavior disorders. All subjects will undergo initial screening procedures to determine eligibility. They will then be randomized to either 8 weeks of placebo or 8 weeks of glutathione or glutathione, vitamin C and N-acetylcysteine. They will receive intravenous treatment weekly and will have ongoing behavioral studies during this period. Following 8 weeks of therapy, they will have a week without treatment then will cross-over to the alternate therapy for weekly intravenous infusions and behavioral testing. In addition to baseline hematology and chemistries, baseline oxidized and reduced glutathione will be measured. These parameters will be repeated at the end of each 8 week course of therapy.
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
- 5 Years 至 16 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age 5 to 16 years, inclusive
- •Diagnosed with autism using the ADI-R (Autism Diagnostic Interview-Revised)
- •ABC Irritability/Agitation subscale score (Aberrant Behavior Checklist Subscale) ≥ 18
- •CGI-S score (Clinical Global Impression-Severity Scale) ≥ 4
- •Must be drug naïve or on a stable dose of medication(s) and educational/behavioral interventions for one month prior to participation
- •Parent/legal guardian must provide written consent and required research authorization (i.e. HIPAA) prior to the performance of any study procedures.
排除标准
- •Presence of another disorder on the autism spectrum including PDD-NOS (Pervasive Developmental Disorder Not Otherwise Specified), Asperger's, and Rett's Syndrome
- •Patient with a known cause of autism such as Fragile X
- •Evidence of significant renal dysfunction (e.g. GFR estimated by the Schwartz formula < 50 mL/min or serum creatinine > 2.5 X upper limit of normal for age)
- •Evidence of significant hepatic dysfunction (serum transaminases > 2.5 X the upper limit of normal)
- •Known hypersensitivity to glutathione, vitamin C or NAC
- •Pregnant or lactating female
- •Inability of subject and parent to be able to comply with requirements for study visits and procedures
- •Presence of major mental illness
- •History of antioxidant supplementation.
研究组 & 干预措施
Placebo
Randomized to placebo or treatment (glutathione or glutathione/vit C/NAC)
干预措施: placebo (Drug)
Glutathione
Randomized to placebo or treatment (glutathione or glutathione/vit C/NAC)
干预措施: glutathione (Drug)
Glutathione, Vit C and NAC
Randomized to placebo or treatment (glutathione or glutathione/vit C/NAC)
干预措施: glutathione, vit C and NAC (Drug)
结局指标
主要结局
Improvement in both developmental skills and behavior with either glutathione or glutathione, Vitamin C and N-acetylcysteine therapy as compared to placebo therapy. Subjects will also be monitored using clinical and laboratory safety parameters.
时间窗: 4 months
Improvement in both developmental skills and behavior measured using validated scales in subjects receiving either glutathione, glutathione/Vitamin C/N-acetylcysteine or placebo measured at baseline, 9 weeks and at the end of study in a double-blind cross-over design.
次要结局
- Response to glutathione (changes in behavior) will correlate with the glutathione level (GSH) and GSH:GSSG ratio(18 weeks)