Early Pharmacotherapy Aimed at Neuroplasticity in Autism : Safety and Efficacy
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 20
- 试验地点
- 2
- 主要终点
- Safety will be measured by obtaining clinical laboratory tests, vital signs and evaluating probably or definitely related adverse events.
研究概览
简要总结
The purpose of this study is to determine the efficacy, safety, and population pharmacokinetics and determinants of drug responses to buspirone in children with autism using a randomized, double blind, cross over study in children ages 2 to 6 years.
详细描述
Autism is a neurodevelopmental disorder defined as qualitative impairment in social interaction and communication and restrictive stereotype patterns of behavior, interests and activities. Pharmacological agents are being increasingly used off label in very young autistic children, and there is virtually no data regarding the pharmacokinetics, safety or efficacy of these agents in young children.
The approach in this study differs from pharmacotherapy studies of autism carried out thus far in several ways:
- the rationale underlying our approach is based upon an attempt to alter synaptic plasticity during postnatal development, focusing on very young children
- are integrating our drug trial with a PG study evaluating whether buspirone response is related to expression of genes involved in serotoninergic neurotransmission
- will assess these variables together with in vivo assessment of serotonin synthesis capacity with PET.
This is a prospective, randomized, double blind, crossover study where children will be stratified by age into two groups. Treatment will last for 12 weeks with dosing twice a day. Parent ratings, cognitive tests and blood sampling will occur throughout the study period.
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
- Double
入排标准
- 年龄范围
- 2 Years 至 6 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Meet study definition for the diagnosis of autistic disorder
- •Age 2 to 6 (male or female)
- •Informed Consent
排除标准
- •Clinical or lab evidence of renal or hepatic disease
- •Treatment with any medication known to alter the activity of the CYP3A4 enzyme including ketoconazole, itraconazole, grapefruit juice, erythromycin, clarithromycin, cimetidine, verapamil, diltiazem, rifampin, phenytoin, phenobarbital, or carbamazepine within the previous 3 months
- •Use of centrally acting drugs during the 6 weeks prior or during the study
- •Presence or history of neurological disorders, including seizure disorders
结局指标
主要结局
Safety will be measured by obtaining clinical laboratory tests, vital signs and evaluating probably or definitely related adverse events.
Population pharmacokinetics will be conducted to measure plasma concentrations in relation to the drug responses to buspirone.
The primary efficacy outcome will be the overall severity score from the Clinical Global Impressions assessment obtained from two raters, (parent and examiner)
Comparisons of allele, and genotype frequencies between responders and non-responders will be done for each polymorphism using Fisher's exact test to best predict response to buspirone.
次要结局
未报告次要终点