An Open-Label Prospective Pilot Trial of Nucleus Accumbens Deep Brain Stimulation for Children with Autism Spectrum Disorder and Severe, Refractory Self-Injurious Behaviour: Study Protocol

Abstract

\n Background\n\nChildren and youth with Autism Spectrum Disorder (ASD) may manifest self-injurious behaviours (SIB) that may become severe and refractory with limited pharmacologic or behavioural treatment options. Here, we present the protocol of a prospective, mixed-methods study to assess the safety and efficacy of deep brain stimulation (DBS) of the nucleus accumbens (NAcc) for children and youth with ASD and severe, refractory SIB.\nMethods\n\nThis is a prospective, single-center, single-cohort, open-label, non-randomized pilot trial of 6 patients. Participants will be recruited through specialized behavioural clinics with persistent severe and refractory SIB following standard and intensive interventions. Following NAcc-DBS, participants will be enrolled in the study for 12 months. The primary objectives of the study are safety and feasibility, assessed by rate of recruitment and identification of factors impacting adherence to follow-up and study protocol. Treatment efficacy will be assessed by changes in the Children’s Yale-Brown Obsessive-Compulsive Scale (CY-BOCS), the Behaviour Problems Index (BPI), the Inventory of Statements about Self-Injury (ISAS) and the Repetitive Behaviour Scale-Revised (RBS-R) questionnaires. Additional clinical outcomes will be assessed, including measures of participant and caregiver quality of life, actigraph measurements, and positron emission tomography (PET) changes following DBS.\nDiscussion\n\nThis study will be the first to evaluate the effect of DBS of the NAcc on a pediatric population in a controlled, prospective trial. Secondary outcomes will improve the understanding of behavioural, neuro-imaging and electrophysiologic changes in children with ASD and SIB treated with DBS. This trial will provide an estimated effect size of NAcc-DBS for severe refractory SIB in children with ASD in preparation for future comparative trials.\nTrial Registration\n\nRegistration on ClinicalTrials.gov was completed on June 12 of 2019 with the Identifier: NCT03982888. https://clinicaltrials.gov/ct2/show/NCT03982888