A Feasibility Study of N-acetylcysteine for Self-injurious Behavior in Children With Autism Spectrum Disorder
| Status: | Recruiting |
|---|---|
| Conditions: | Neurology, Psychiatric, Psychiatric, Psychiatric, Autism |
| Therapuetic Areas: | Neurology, Psychiatry / Psychology |
| Healthy: | No |
| Age Range: | 5 - 12 |
| Updated: | 7/11/2018 |
| Start Date: | July 5, 2018 |
| End Date: | December 31, 2019 |
| Contact: | Lawrence Scahill, MSN, PhD |
| Email: | lawrence.scahill@emory.edu |
| Phone: | 404-785-9494 |
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.
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.
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.
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.
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
We found this trial at
1
site
Atlanta, Georgia 30329
Principal Investigator: Lawrence Scahill, MSN, PhD
Phone: 404-785-9467
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