Double-blind , Randomized, Placebo Controlled Study of N-Acetyl Cysteine in Autism.
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 43
- 试验地点
- 1
- 主要终点
- Total Number of Subjects With Reported Side Effects as Assessed by Dosage Record and Treatment Emergent Symptom Scale (DOTES)
研究概览
简要总结
The purpose of the study is to test the tolerability and efficacy of N-Acetyl Cysteine (NAC) in children with Autism. NAC is a compound that increases the levels of Glutathione, the body's main antioxidant. Glutathione is a compound in the blood that is part of a natural defense system (the antioxidant system). Anti-oxidants protect the body from damage caused by internal toxins called "free radicals." It is possible that children with Autism tend to have lower levels of glutathione, an important compound in our bodies that helps combat the effects of toxic free radicals.
We hope that by studying the antioxidant system in more detail, we will increase our understanding of the reasons why people develop Autism so that we can design better ways to treat individuals with this condition. This study is meant to test the safety tolerability of NAC and its effectiveness in the treatment of behavioral difficulties in children with autism. It will also examine the possible benefit of this agent in improving the core deficits in autism such as social deficits.
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
- 3 Years 至 12 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Outpatients between 3.0 and 12.11 years of age inclusive
- •Males and females who are physically healthy
- •diagnosis of autism based Diagnostic and Statistical Manual (DSM-IV-TR) criteria, the Autism Diagnostic Interview-Revised, and expert clinical evaluation
- •Clinical Global Impression Severity rating of 4
- •Care provider who can reliably bring subject to clinic visits, can provide trustworthy ratings, and interacts with subject on a regular basis
- •Ability of subject to swallow the compound
- •Stable concomitant medications for at least 2 weeks
- •No planned changes in psychosocial interventions during the open-label N-Acetyl Cysteine trial
排除标准
- •DSM-IV-TR diagnosis of schizophrenia, schizoaffective disorder, or psychotic disorder not otherwise specified
- •Prior adequate trial of N-Acetyl Cysteine
- •Active medical problems: unstable seizures, significant physical illness (e.g., serious liver or renal pathology)
- •Pregnancy or sexually active females
- •Subjects taking antioxidant agents and glutathione prodrugs will be excluded from the study except if they have been off these compounds for at least 4 weeks
研究组 & 干预措施
N-Acetyl Cysteine
active compound N-Acetyl Cysteine
干预措施: N-Acetyl Cysteine (Drug)
Sugar pill
Placebo or sugar pill
干预措施: Placebo - sugar pill (Other)
结局指标
主要结局
Total Number of Subjects With Reported Side Effects as Assessed by Dosage Record and Treatment Emergent Symptom Scale (DOTES)
时间窗: 4, 8, and 12 weeks
The Dosage Record and Treatment Emergent Symptom Scale (DOTES) provides information on the presence, frequency, and severity of side effects reported during the course of the trial.
The Clinical Global Rating Scale (CGRS) Improvement Subscale Score
时间窗: 12 weeks
Score range 1-7 (lower score mean more improvement compared to baseline)
Glutathione (GSH) Levels in Peripheral Blood, Measured by State-of-the-art High-performance Liquid Chromatography (HPLC)
时间窗: 12 weeks
Data not collected. The laboratory was not able to measure Glutathione levels.
Irritability Subscale of the Aberrant Behavior Checklist (ABC)
时间窗: baseline and 12 weeks
Aberrant Behavior Checklist (ABC) Irritability Subscale Score (range 0-45); higher scores mean higher irritability
次要结局
- The Aberrant Behavior Checklist Total Score (ABC)(4, 8, and 12 weeks)
- Sensory Profile Questionnaire (SPQ)(12 weeks)
- Glutathione (GSH) Metabolism Intermediates in Peripheral Blood(12 weeks)
- Social Responsiveness Scale (SRS)(12 weeks)
研究者
Antonio Hardan
Professor
Stanford University