Clinical trial NCT03008889: A Feasibility Study of N-acetylcysteine for Self-injurious Behavior in Children With Autism Spectrum Disorder
Official title: A Feasibility Study of N-acetylcysteine for Self-injurious Behavior in Children With Autism Spectrum Disorder
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Autism Spectrum Disorder
Keywords: Repetitive Self-Injurious Behavior
N-acetylcysteine (NAC)
Lead sponsor: Emory University
Sponsor class: OTHER
First posted: 2017-01-04
Enrollment: 8
Sex: ALL
Minimum age (years): 5
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to demonstrate the feasibility of a 9-week, randomized trial of N-acetylcysteine (NAC) compared to placebo in 14 children (age 5 to 12 years) with Autism Spectrum Disorder (ASD) and a moderate level of repetitive self-injurious behavior (SIB). Additional aims are to evaluate the positive predictive value of a screening method to classify children with automatically maintained self-injurious behavior; to evaluate the preliminary efficacy of NAC for reducing repetitive SIB in children with ASD; and to evaluate biomarkers and possible mechanisms of action of NAC in children with ASD.

Detailed description: Self-injurious behavior (SIB) in children with autism spectrum disorder (ASD) can cause physical harm to the child and interfere with the child's ability to make use of educational programs and helpful treatments such as speech therapy. The turmoil caused by self-injurious behaviors in children with ASD invariably interferes with daily routines because family life often stops during these episodes and family members worry about setting off SIB between episodes. This project will use the detailed assessment methods developed in the field of behavior therapy to evaluate the potential for N-acetylcysteine (NAC) to treat children with ASD and moderate repetitive SIB. NAC is an over-the-counter dietary supplement that may have beneficial effects on the brain through its well-documented antioxidant effects and/or reduced glutamate signaling. In the proposed study, 14 children with ASD and repetitive SIB between the ages of 5 and 12 will be randomly assigned to gradually increasing doses of NAC or placebo for 9 weeks. The research team, parents and children will be blind to the treatment with NAC or placebo. Participants will come to the research site periodically to complete measures and behavioral assessments.

After the 9 weeks of treatment, children randomized to NAC who showed improvement will be encouraged to continue taking the supplement outside the study. Children who were randomly assigned to the placebo and showed no improvement will be offered open-label treatment with NAC. Children who did not improve while taking NAC or those who improved while on the placebo will be advised on next steps by the study team.

The goal of this feasibility study is establish the acceptability viability of study procedures in this vulnerable population, to learn about the potential benefits and adverse effects of NAC. Demonstrating these feasibility aims and the preliminary efficacy and safety of NAC is a prerequisite for planning a larger, more definitive, study.

Eligibility criteria: Inclusion Criteria:

* Confirmed diagnosis of Autism Spectrum Disorder (ASD)
* Confirmed presence of moderate Self Injurious Behavior (SIB)
* Score \> 16 on the parent-rated Aberrant Behavior Checklist Irritability subscale (moderate level of disruptive behavior)
* Classified as having automatically maintained SIB (determined during screening by a detailed functional analysis)

Exclusion Criteria:

* On a stable medication dose for less than 4 weeks
* Planned change in medication during the 9-week trial
* Had one or more seizures in the last 6 months

Primary outcomes:
measure: Percentage of Participants Randomized
timeFrame: 12 months (throughout the duration of the study)
measure: Attrition Rate
timeFrame: 12 months (throughout the duration of the study)
measure: Study Medication Compliance
timeFrame: 12 months (throughout the duration of the study)
measure: Successful Collection of Outcome Measures
timeFrame: 12 months (throughout the duration of the study)
measure: Parent Satisfaction Rating
timeFrame: Week 9 (at the end of the study intervention)
Secondary outcomes:
measure: Positive Predictive Value of Screening Method of Classifying Self-injurious Behavior (SIB) by Type.
timeFrame: 12 months (duration of the study)
measure: Aberrant Behavior Checklist Irritability Subscale Score at Baseline and 9 Weeks Post-intervention
timeFrame: Baseline, Week 9
measure: Number of Self-Injurious Behavior Events
timeFrame: Baseline, Week 9
measure: Change in Clinical Global Impression (CGI-I) Scale at 9 Weeks Post-intervention
timeFrame: Week 9
measure: Change in Biomarkers and Possible Mechanisms of Action of NAC in Children With ASD.
timeFrame: Baseline, Week 9
Other outcomes:
Locations:
city: Atlanta
country: United States
facility: Marcus Autism Center
state: Georgia
Study officials:
affiliation: Emory University
name: Lawrence Scahill, MSN, PhD
role: PRINCIPAL_INVESTIGATOR
References: