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
Intervention: N-acetylcysteine; Placebo
Intervention description: 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.
Intervention MeSH terms: Acetylcysteine; N-monoacetylcystine
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 8.0
Sex: All
Minimum age: 5 Years
Maximum age: 12 Years
Healthy volunteers: Accepts Healthy Volunteers
Keywords: Repetitive Self-Injurious Behavior; N-acetylcysteine (NAC)

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.
    

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