Deep Brain Stimulation for Severe Self-Injurious Behaviour in Children

The Hospital for Sick Children25 enrolled

Overview

Deep Brain Stimulation for the Treatment of Severe Refractory Self-Injurious Behaviour in Children with Autism Spectrum Disorder: A Randomized Trial To evaluate the effectiveness of deep brain stimulation (DBS) of the nucleus accumbens for the treatment of severe refractory, repetitive self-injurious behavior (SIB) in children with Autism Spectrum Disorder. Secondary objectives are to examine the effects of DBS on subtypes of SIB through functional analysis.

The proposed study is a single-center, double-blinded, block-randomized crossover trial. Twenty-five (25) participants will be recruited and enrolled in this trial to undergo bilateral nucleus accumbens DBS. After recovery, participants will be randomized to a 3 month block of active stimulation or the DBS will remain off. After a two week washout period, the participants will cross over to the other group. At the end of the second block, all patients will have their DBS devices activated during an open label period. All participants will have baseline assessments that will be repeated at the end of each block. Expected study duration is 24 months.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

Conditions

Self-Injurious Behavior Autism Spectrum Disorder

Eligibility

Sex: ALLMin age: 5 YearsMax age: 17 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Age 5-17 at the time of enrollment * DSM-5 diagnosis of Autism Spectrum Disorder * History of repetitive self-injurious behaviour, as reported by parents and documented on clinical assessment, either at the time of enrollment into the study or in prior medical records. The definition of self-injury is contextual, but requires ongoing, intermittent or continuous manifestation of self-mediated physical injury to the child. * Foreseeable risk of serious future self-harm. * Screening by study team for presence automatically reinforced self-injurious behaviour (ASIB) subtype 2 or subtype 3 based on caregiver history. * Failure or non-eligibility of medical therapy with ongoing repetitive self-injurious behaviours, at 6 months or more after initiation of therapy. * Parents or legal guardians, including caregivers, informed and able to provide written consent. * Able to comply with all testing, follow-up visits, and study appointments and protocols for 12 months following the end of the duration of the study. Exclusion Criteria: * Substance dependence or abuse in the last 6 months, excluding caffeine and nicotine. * Any contraindication to MRI scanning. * Presence of cardiac arrhythmias, or other cardiac, respiratory, renal or endocrine conditions that may incur significant risk from a surgical procedure. * Pregnancy.

Outcomes

Primary Outcomes

Repetitive Behaviors Scale-Revised (RBS-R)

A 44-item questionnaire that asks parents/caregivers how often a particular behaviour is seen occurring in the participant on a scale of 0-3, as well as a summary measure of how often the repetitive behaviours occur overall on a scale of 1-100. Higher scores indicate greater severity.

Time frame: Baseline, 4 weeks, 16 weeks, 30 weeks, 52 weeks

Secondary Outcomes

Self-injurious behaviour log

Parent/caregiver-reported frequency of repetitive self-injurious events over a given time period (i.e. number of events per day)

Time frame: Baseline (3 months), weekly during trial

Repetitive Behaviour Questionnaire-2 (RBQ-2)

A 20-item parent/caregiver questionnaire that measures the frequency of a child's repetitive, restricted, and sensory behaviours. Questionnaire scores can be added to give a Total Repetitive Behaviours Score, within a minimum score of 20 and a maximum score of 60 (with responses '3' and '4' combined). Higher scores indicate greater severity.

Time frame: Baseline, 4 weeks, 16 weeks, 30 weeks, 52 weeks

Self-Injurious Behavior Questionnaire (SIB-Q)

A 25-item scale with Likert-style response options that measures self-injurious behavior among those with developmental disabilities. Scores range from 0 to 100, with higher scores indicating greater severity.

Time frame: Baseline, 4 weeks, 16 weeks, 30 weeks, 52 weeks

Pediatric Quality of Life Inventory (PedsQL) Questionnaire

A parent questionnaire designed to evaluate health-related quality of life in children. Scores range from 0 to 92, with higher scores indicating poorer quality of life.

Time frame: Baseline, 4 weeks, 16 weeks, 30 weeks, 52 weeks

Autism Diagnostic Observation Schedule (ADOS) Assessment questionnaire

A standardized, activity-based assessment designed to evaluate communication skills, social interaction, and imaginative use of materials in individuals who are suspected to have autism spectrum disorder. Classifications: * Non-spectrum: communication \<1, social interaction \<3, communication + social \<6 * Autism spectrum: communication \>2, AND social interaction \>4, AND communication + social 7-9 * Autism: communication \>3, AND social interaction \>6, AND communication + social \>10 Higher scores indicate a greater presence of behaviours associated with autism spectrum disorder.

Time frame: Baseline, 16 weeks, 30 weeks

Modified Overt Aggression Scale (MOAS) questionnaire

A brief survey of overt aggression. Scores range from 0 to 40, with higher scores indicating greater severity.

Time frame: Baseline, 4 weeks, 16 weeks, 30 weeks, 52 weeks

The Burden Scale for Family Caregivers - Short Version (BSFC-S) questionnaire

A brief survey of parental burden of care. Scores range from 0 to 30, with higher scores indicating a greater burden.

Time frame: Baseline, 4 weeks, 16 weeks, 30 weeks, 52 weeks

Functional analysis (FA) assessment

An observation-based behavioural assessment that requires observing and quantifying how self-injurious behaviour (SIB) changes across conditions of a standardized and replicable assessment.

Time frame: Baseline, 16 weeks, 30 weeks

Deep Brain Stimulation for Severe Self-Injurious Behaviour in Children

Overview

Conditions

Interventions

Eligibility

Locations (1)

Outcomes

Primary Outcomes

Secondary Outcomes

Central Contacts