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

Phase 2

Recruiting

• Conditions: Autism Spectrum Disorder
• Interventions: Drug: N acetyl cysteine; Drug: Placebo

• Registration Number: NCT05664789
• Lead Sponsor: Stanford University

Brief 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2022-12-07
• Study First Submitted QC: 2022-12-15
• Study First Posted: 2022-12-27
• Study Start: 2023-04-26
• Status Verified: 2024-07
• Last Update Submitted: 2024-07-23
• Last Update Posted: 2024-07-24
• Primary Completion: 2026-01-31
• Study Completion: 2026-01-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 48

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
• if home address is within 300 miles of the primary study site, participants must pass MR safety screening (e.g., no metal in the body) and attempt baseline neuroimaging (MRI or EEG), otherwise baseline neuroimaging is not required for participation
• have stable medication regimens (≥ 30 days) and psychosocial treatments (≥ 60 days) prior to randomization with no anticipated changes during the trial

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
• history of any adverse effects to glutathione agents/prodrugs
• the inability to drink a sample study compound dissolved in liquid

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
N-acetylcysteine	N acetyl cysteine	12 week administration of active study compound
Placebo	Placebo	12 week administration of matched placebo

Primary Outcome Measures

Name	Time	Method
Change in Children's Yale Brown-Obsessive Compulsive Scale for Autism Spectrum Disorder (CYBOCS-ASD) at 12 weeks	Screening and week 12	Severity of restricted and repetitive behaviors, higher scores indicate more severe behaviors
Change in Glutamatergic neurometabolites at 12 weeks	Baseline and week 12	Glu and Glx measured by magnetic resonance imaging

Secondary Outcome Measures

Name	Time	Method
Change in Gamma band activity at 12 weeks	Baseline and week 12	Electrical activity between 30-48 Hz measured by electroencephalography
Change in Restricted and Repetitive Behavior Scale Revised (RBS-R) at 12 weeks	Baseline and week 12	Severity of restricted and repetitive behaviors, higher scores indicate more severe behaviors

Trial Locations

Locations (1)

Department of Psychiatry and Behavioral Sciences; 🇺🇸 Palo Alto, California, United States