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.

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.

De-identified data from the clinical trial will be made available upon reasonable request, subject to institutional approval and, where applicable, the execution of a Data Sharing Agreement. Requests for data access should be directed to the corresponding author.