Neuronal Nicotinic Receptor Modulation in the Treatment of Autism: A Pilot Trial of Mecamylamine
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- The primary measure will be a global composite derived by calculating the z scores on each measure other than CGI from published norms (or from the baseline descriptives, then averaging the z scores of the various outcome measures for each individual.
研究概览
简要总结
The purpose of this study is to examine the safety and efficacy of mecamylamine for the core symptoms of autism.
详细描述
This is a placebo-controlled pilot clinical trial of a nicotinic agent based on evidence that a disturbance occurs in nicotinic cholinergic transmission in the brain of autistic children. This pharmacotherapeutic trial will be conducted as a direct result of findings of a nicotinic receptor abnormality in autopsied brain tissue from individuals who suffered from autism. A pharmacological agent, mecamylamine, with specificity for the nicotinic receptors implicated in autism, will be tested in children with autism spectrum disorder. Twenty children who meet inclusion/exclusion criteria will be randomized in a 2:3 ratio to 14 weeks of either matched placebo (n=8) or mecamylamine (n=12). Children randomized to placebo who do not respond will be given the opportunity to enroll in a ten week open label extension.
The trial has been completed and published in J. of Child & Adolescent Psychopharmacology.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Factorial
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 4 Years 至 12 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Autistic Disorder or Pervasive Developmental Disorder-Not Otherwise Specified
- •General good health
- •IQ of >=36 or mental age of >=18 months
- •Parent/caregiver willingness to accompany child to clinic and monitor for side effects
排除标准
- •Unstable Seizure Disorder
- •Psychoactive medication in the process of adjustment
- •Antipsychotic medication in previous 3 months before baseline
- •Systemic corticoids (inhalers allowed)
- •Planned beginning of major behavioral intervention in 3 months of study or the 2 months prior to the study.
研究组 & 干预措施
Active Mecamylamine
There will be 12 children in this arm. These children will receive the active medication (mecamylamine).
干预措施: mecamylamine (Drug)
Placebo
There will be 8 children in this arm. These children will receive placebo instead of the active medication.
干预措施: placebo (Drug)
结局指标
主要结局
The primary measure will be a global composite derived by calculating the z scores on each measure other than CGI from published norms (or from the baseline descriptives, then averaging the z scores of the various outcome measures for each individual.
时间窗: Baseline, weeks 2, 4, 6, 8, 10,12, and 14.
次要结局
- Aberrant Behavior Checklist(Baseline, week 2, 4, 6, 8, 10, 12, and 14.)
- Expressive Vocabulary test-Second Edition(Baseline and week 14)
- Social Responsiveness Scale(Baseline, weeks 2, 4, 6, 8, 10, 12, and 14.)
- Repetitive Behavior Questionnaire(Baseline, week 2, 4, 6, 8, 10, 12, and 14)
- Ohio Autism Clinical Impressions Scale(Baseline, weeks 1, 2, 4, 6, 7, 8, 9, 10, 12, and 14.)
- Cognitive Battery(baseline, weeks 6, 8, and 14)
- OSU Autism Rating Scale-DSM-IV(Baseline, week 6, 8, and 14.)
- Adverse Experience checklist and AE log(Baseline, weeks 1, 2, 4, 6, 7, 8, 9, 10, 12, and 14.)
研究者
L. Eugene Arnold
Interim Director of Nisonger Center
Ohio State University