A Feasibility Study of N-acetylcysteine for Self-injurious Behavior in Children With Autism Spectrum Disorder
Condition: Autism Spectrum Disorder
MeSH terms: Autistic Disorder; Autism Spectrum Disorder; Child Development Disorders, Pervasive; Self-Injurious Behavior
Study type: Interventional
Phase: Phase 2

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.
    

Intervention name(s): N-acetylcysteine; Placebo
Intervention type(s): Drug; Drug
Intervention description(s): Participants will start with taking 900mg of NAC once per day for one week (study days 1-7). At Day 7, parents (or primary caregiver) and subjects will return to the study site to review adverse events.
In the absence of dose limiting adverse events attributable to the study drug, the dose will be gradually increased to 900 mg twice per day for study days 8-28.
If this dose is well-tolerated, the dose will be increased to 900 three times per day for study days 29-63.; Participants will start with taking 900mg of the placebo once per day for one week (study days 1-7). At Day 7, parents (or primary caregiver) and subjects will return to the study site to review adverse events. The dosing for the placebo will increase in the same fashion as the active treatment.
In the absence of dose limiting adverse events attributable to the study drug, the dose will be gradually increased to 900 mg twice per day for study days 8-28.
If this dose is well-tolerated, the dose will be increased to 900 three times per day for study days 29-63.
Other intervention name(s): NAC
Intervention MeSH terms: Acetylcysteine; N-monoacetylcystine
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: Repetitive Self-Injurious Behavior; N-acetylcysteine (NAC)