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

• Status: Not yet recruiting
• Trial end date: 2026-01-31
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Stanford University
• Collaborator: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
• Treatments: Acetylcysteine; N-monoacetylcystine

Criteria

Inclusion Criteria:

• children between 3 years and 12 years 11 months

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

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

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

• medically stable

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

• passes MR safety screening (e.g., no metal in the body)

• successfully completes baseline neuroimaging (MRI or EEG)

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 sufficient level

• participants taking glutathione agents/prodrugs

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