Deep Brain Stimulation for the Treatment of Refractory Repetitive Self-Injurious Behaviour in Children With Autism Spectrum Disorder: A Pilot Project
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 6
- 试验地点
- 2
- 主要终点
- Changes in abberant behaviour
研究概览
简要总结
The purpose of this study is to evaluate the safety and possible effectiveness of deep brain stimulation (DBS) of the nucleus accumbens in children with autism spectrum disorder and treatment-refractory, repetitive self-injurious behavior. Six (6) patients will be recruited and enrolled in this pilot study and study duration for each patient will be one (1) year. All will undergo surgical implantation of the Medtronic DBS system and will receive stimulation of the nucleus accumbens (2 electrodes per patient).This will be an open, non-blinded, non-randomized, pilot, phase I trial.Expected study duration is 36 months.
详细描述
This is a phase I, non-blinded, non-randomized, pilot trial for safety and efficacy of deep brain stimulation for medically-refractory, repetitive self-injurious behaviours in children with ASD (i.e. secondary stereotypies). The trial will be conducted in compliance with the protocol, GCP and the applicable regulatory requirement(s).
Patients who meet inclusion and exclusion criteria will be identified and recruited the Neurosurgical Clinic at The Hospital for Sick Children. The study will proceed according to the schedule laid out below, and both patients and treating team will be aware of all treatment parameters at all times. Six (6) subjects will be enrolled in this study and study duration for each patient will be one (1) year. Previous phase I trials of DBS in psychiatric and Alzheimer's Disease populations have utilized 6 subjects per surgical target. Such a number is sufficient to demonstrate initial safety, as well as feasibility and clinical effectiveness.
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
- 7 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Female or Male patients between age 7-18
- •Diagnosis of Autism Spectrum Disorder (as defined by the DSM-5).
- •Failure or non eligibility of medical therapy with ongoing repetitive self-injurious behaviours, at 6 months or more after instigation of therapy. Failure is defined as a lack of improvement in self-injurious behaviours, as documented by objective evidence, including caregiver logs or clinician assessment, if the clinician has documented a baseline status prior to instigation of the medical therapy.
- •Diagnosis of secondary stereotypies, based on clinical assessment of the treating physicians with evidence of self-injury, documented in the patient records.
- •Parents or legal guardians, including caregivers, informed and able to give written consent.
- •Able to comply with all testing, follow-ups and study appointments and protocols for 12 months following the end of the duration of the study.
排除标准
- •Substance dependence or abuse in the last 6 months, excluding caffeine and nicotine
- •Any contraindication to MRI or PET scanning
- •Likely to relocate away from the study site or move during the study's one year duration
- •Presence of cardiac arrhythmias, or other cardiac, respiratory, renal or endocrine conditions that will result in significant risk from a surgical procedure.
- •Pregnancy
- •Unable to communicate adequately in English in order to complete the baseline and follow-up questionnaires.
研究组 & 干预措施
DBS Treatment
Deep brain stimulation of both limbic and dysfunctional reward processing circuits for treatment of repetitive self injurious behaviours in children with ASD
干预措施: Deep Brain Stimulation (Procedure)
DBS Treatment
Deep brain stimulation of both limbic and dysfunctional reward processing circuits for treatment of repetitive self injurious behaviours in children with ASD
干预措施: DBS (Device)
结局指标
主要结局
Changes in abberant behaviour
时间窗: 1 year
The Aberrant Behavior Checklist (Aman, Singh, Stewart \& Field, 1985) will be used to measure changes in the extent that abberant behaviour is a problem pre and post intervention. The scale is scored from 0-3 as follows: 0= not at all a problem 1. the behaviour is a problem but slight in degree 2. the problem is moderately serious 3. the problem is severe in degree Where the higher number indicates the behaviour is problematic. Questions are scored and totaled. Measure will be completed at baseline (pre-surgery), weekly from weeks 2-6 post-surgery, bi-weekly from weeks 7-10 post surgery, monthly from weeks 11-51 post-surgery, and 1 year post surgery.
Changes in self-injurious behaviour
时间窗: 1 year
The Inventory of Statements about Self-Injury (ISAS; Klonsky \& Olino, 2008) will be used to measure changes in self-injurious behaviour pre and post intervention. The scale is scored from 0-2 as follows: 0= not relevant 1. somewhat relevant 2. very relavant Where a higher number indicates increased self-injurious behaviour. Questions are scored and totaled. Measure will be completed at baseline (pre-surgery), weekly from weeks 2-6 post-surgery, bi-weekly from weeks 7-10 post surgery, monthly from weeks 11-51 post-surgery, and 1 year post surgery.
Changes in obsessive-compulsive thoughts
时间窗: 1 year
The Yale-Brown Obsessive Compulsive Scale (Y-BOCS; Goodman, Price, Rasmussen,1989) will be used to measure changes in the amount of interference of unwanted ideas, images, or impulses pre and post intervention. The scale is scored from 0-4, where 0 represents minimal interference and 4 represents substantial interference. Questions are scored and totaled. Measure will be completed at baseline (pre-surgery), weekly from weeks 2-6 post-surgery, bi-weekly from weeks 7-10 post surgery, monthly from weeks 11-51 post-surgery, and 1 year post surgery.
Changes in repetitive behaviour
时间窗: 1 year
The Repetitive Behavior Scale-Revised (RBS-R; Bodfish, Symons, Parker \& Lewis, 2000) will be used to measure changes in repetitive behaviour pre and post intervention. The following sub-scales will be used: * Steretyped behaviour subscale * Self-injurious behaviour subscale * Compulsive behaviour subscale * Ritualistic behaviour subscale * Sameness behaviour subscale * Restricted behaviour subscale All subscales are scored from 0-3 as follows: 0 = behaviour does not occur 1. behaviour occurs and is a mild problem 2. behaviour occurs and is a moderate problem 3. behaviour occurs and is a severe problem Where a higher number indicates increased repetitive behaviour. Subscores are totaled and then an overall score is calculated. Measure will be completed at baseline (pre-surgery), weekly from weeks 2-6 post-surgery, bi-weekly from weeks 7-10 post surgery, monthly from weeks 11-51 post-surgery, and 1 year post surgery.
Changes in quality of life
时间窗: 1 year
The Paediatric Quality of Life Inventory (PedsQL v. 4.0) will be used to measure changes in self-reported quality of life pre and post intervention. The statements are scored from 0-4 as follows: 0= it is never a problem 1 = it is almost never a problem 2= it is sometimes a problem 3 = it is often a problem 4= it is almost always a problem Where a higher score indicates the statement is a problem. All items are scored and totaled. Measure will be completed at baseline (pre-surgery), weekly from weeks 2-6 post-surgery, bi-weekly from weeks 7-10 post surgery, monthly from weeks 11-51 post-surgery, and 1 year post surgery.
次要结局
- Changes in physical brain abnormalities(1 year)
- Changes in metabolic brain abnormalities(1 year)
- Type of adverse events reported in caregiver logs(1 year)
- Changes in activity(1 year)
- Changes in number of complications pre and post intervention will be compared.(1 year)
研究者
George Ibrahim
Principal Investigator
The Hospital for Sick Children