Deep Brain Stimulation in Children With Autism

NCT ID: NCT03982888

Last Updated: 2024-02-22

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 6 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-08-14
• Study Completion Date: 2024-01-31

Brief Summary

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.

Detailed Description

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.

Conditions

Autism Spectrum Disorder; Self-Injurious Behavior

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

DBS Treatment

Deep brain stimulation of both limbic and dysfunctional reward processing circuits for treatment of repetitive self injurious behaviours in children with ASD

Group Type: EXPERIMENTAL

Deep Brain Stimulation

Intervention Type: PROCEDURE

Stimulation of the nucleus accumbens via 2 electrodes implanted in the brain per patient to reduce self-injurious behaviours.

DBS

Intervention Type: DEVICE

Use of the Medtronic DBS device. DBS device consists of 2 parts:

1. DBS lead, a thin wire with 2 electrical contacts implanted into pre-determined brain regions, and

2. Implantable pulse generator, a small device containing the batter and computer source placed under the skin of the chest to generate electrical pulses

Interventions

Deep Brain Stimulation

Stimulation of the nucleus accumbens via 2 electrodes implanted in the brain per patient to reduce self-injurious behaviours.

Intervention Type: PROCEDURE

DBS

Use of the Medtronic DBS device. DBS device consists of 2 parts:

1. DBS lead, a thin wire with 2 electrical contacts implanted into pre-determined brain regions, and

2. Implantable pulse generator, a small device containing the batter and computer source placed under the skin of the chest to generate electrical pulses

Intervention Type: DEVICE

Other Intervention Names

Medtronic DBS Therapy (Device # 37601)

Eligibility Criteria

Inclusion Criteria

• 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.

Exclusion Criteria

• 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.

• Minimum Eligible Age: 7 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

The Hospital for Sick Children

OTHER

Sponsor Role: lead

Responsible Party

George Ibrahim

Principal Investigator

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

George Ibrahim, MD

Role: PRINCIPAL_INVESTIGATOR

The Hospital for Sick Children

Locations

The Hospital for Sick Children

Toronto, Ontario, Canada

Countries

Canada

References

Gorodetsky C, Mithani K, Breitbart S, Yan H, Zhang K, Gouveia FV, Warsi N, Suresh H, Wong SM, Huber J, Kerr EN, Kulkarni AV, Taylor MJ, P Hagopian L, Fasano A, Ibrahim GM. Deep Brain Stimulation of the Nucleus Accumbens for Severe Self-Injurious Behavior in Children: A Phase I Pilot Trial. Biol Psychiatry. 2025 Jun 15;97(12):1116-1126. doi: 10.1016/j.biopsych.2024.12.001. Epub 2024 Dec 5.

Reference Type: DERIVED

PMID: 39645140 (View on PubMed)

Yan H, Siegel L, Breitbart S, Gorodetsky C, Fasano A, Rahim A, Loh A, Kulkarni AV, Ibrahim GM. An open-label prospective pilot trial of nucleus accumbens deep brain stimulation for children with autism spectrum disorder and severe, refractory self-injurious behavior: study protocol. Pilot Feasibility Stud. 2022 Feb 2;8(1):24. doi: 10.1186/s40814-022-00988-3.

Reference Type: DERIVED

PMID: 35109924 (View on PubMed)

Other Identifiers

REB1000060282

Identifier Type: -

Identifier Source: org_study_id