Targeting the Neurobiology of RRB in Autism Using N-acetylcysteine: Trial

Last updated: July 23, 2024
Sponsor: Stanford University
Overall Status: Active - Recruiting

Phase

2/3

Condition

Asperger's Disorder

Autism

Williams Syndrome

Treatment

Placebo

N acetyl cysteine

Clinical Study ID

NCT05664789
IRB-63262
4R00HD101702-03
  • Ages 3-12
  • All Genders

Study Summary

The goal of this study is to target the neurobiology of restricted and repetitive behaviors in children with autism spectrum disorder using N-acetylcysteine (NAC), a well-tolerated nutritional supplement that has shown promise for reducing symptom severity in recent small-scale trials. The findings from this research will shed light on the mechanisms of action underlying the clinical benefits of NAC and the effects of NAC on altering restricted and repetitive behavior symptom severity in children with autism spectrum disorder. This is a 12-week double-blind, randomized, placebo-controlled trial of NAC.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • children between 3 years and 12 years 11 months

  • diagnosis of autism spectrum disorder confirmed with the Autism DiagnosticInterview- Revised and the Autism Diagnostic Observation Schedule-2, BriefObservation of Symptoms of Autism, or Childhood Autism Rating Scale

  • at least moderate severity of restricted and repetitive behaviors defined by aChildren's Yale-Brown Obsessive Compulsive Scale for Autism Spectrum Disorder score ≥ 11

  • physical development indicative of prepubescence as defined by criteria for TannerStage 1

  • if home address is within 300 miles of the primary study site, participants mustpass MR safety screening (e.g., no metal in the body) and attempt baselineneuroimaging (MRI or EEG), otherwise baseline neuroimaging is not required forparticipation

  • have stable medication regimens (≥ 30 days) and psychosocial treatments (≥ 60 days)prior to randomization with no anticipated changes during the trial

Exclusion

Exclusion Criteria:

  • presence of known genetic abnormalities associated with ASD (e.g. Fragile X)

  • current or life-time diagnosis of severe psychiatric disorder (e.g., schizophrenia)

  • presence of significant medical problems

  • the inability of at least one caregiver to speak and read English to a sufficientlevel

  • participants taking glutathione agents/prodrugs

  • history of any adverse effects to glutathione agents/prodrugs

  • the inability to drink a sample study compound dissolved in liquid

Study Design

Total Participants: 48
Treatment Group(s): 2
Primary Treatment: Placebo
Phase: 2/3
Study Start date:
April 26, 2023
Estimated Completion Date:
January 31, 2026

Connect with a study center

  • Department of Psychiatry and Behavioral Sciences

    Palo Alto, California 94304
    United States

    Active - Recruiting

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