Targeting the Neurobiology of Restricted and Repetitive Behaviors in Children With Autism Using N-acetylcysteine: Open Label

• Status: Not yet recruiting
• Trial end date: 2028-09-01
• 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.

• 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:

Inclusion criteria: successful completion of IRB protocols 54931 or 65821.

Inclusion for 54931 include - (1) children between 3 years and 12 years 11 months, (2) 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, (3) at least moderate severity of restricted and repetitive behaviors defined by a Children's Yale-Brown Obsessive Compulsive Scale in ASD score ≥ 11, (4) physical development indicative of prepubescence as defined by criteria for Tanner Stage 1 (5) medically stable, (6) passes MR safety screening (e.g., no metal in the body).

Inclusion for 65821 - (1) same criteria as above for 54931, (2) have stable medication regimens (≥ 30 days) and psychosocial treatments (≥ 60 days) prior to randomization with no anticipated changes during the trial, and (3) successfully completes baseline neuroimaging (MRI or EEG).

Exclusion Criteria:

Exclusion criteria - significant adverse affects that led to withdrawal or early termination from IRB 54931 or 65821.

Exclusion criteria for 54931 and 65821 include - (1) presence of known genetic abnormalities associated with ASD (e.g. Fragile X), (2) current or life-time diagnosis of severe psychiatric disorder (e.g., schizophrenia), (3) presence of significant medical problems, (4) the inability of at least one caregiver to speak and read English to a sufficient level, (5) participants taking glutathione agents/prodrugs, or (6) the inability to drink a dissolved agent.