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

Phase 2

• Conditions: Autism Spectrum Disorder
• Interventions: Drug: N-acetylcysteine

• Registration Number: NCT05494398
• 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2022-08-08
• Study First Submitted QC: 2022-08-08
• Study First Posted: 2022-08-10
• Study Start: 2024-03-12
• Status Verified: 2024-08
• Last Update Submitted: 2024-08-22
• Last Update Posted: 2024-08-26
• Primary Completion: 2027-09
• Study Completion: 2028-09

Recruitment & Eligibility

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

Inclusion Criteria

• participation in an associated single-dose (IRB-54931, NCT04278898) or randomized, controlled trial of NAC (IRB-63262, NCT05664789).
• stable medication regimens (≥ 30 days) with no anticipated changes during the trial.

Exclusion criteria:

• the presence of significant medical problems that reduce medical stability.

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
N-Acetylcysteine	N-acetylcysteine	-

Primary Outcome Measures

Name	Time	Method
Change in restricted and repetitive behaviors as measured by Children's Yale Brown Obsessive Compulsive Scale - Autism Spectrum Disorder (CYBOCS-ASD)	Screening, Week 8, week 12	Higher scores on the Children's Yale Brown Obsessive Compulsive Scale - Autism Spectrum Disorder mean higher levels of restricted and repetitive behaviors (Raw Score Total Range: 0-20)
Change in glutamatergic neurometabolites (Glx) measured by proton spectroscopy Magnetic Resonance Imaging (MRI)	Baseline and Week 12 of treatment

Secondary Outcome Measures

Name	Time	Method
Change in restricted repetitive behavior subtypes as measured by the Restricted Behavior Scale - Revised (RBS-R)	Baseline, week 8, week 12	Higher scores on the Repetitive Behavior Scale- Revised mean higher levels of repetitive and restricted behaviors. (Raw Score Total Range: 0 - 129)
Change in Gamma band amplitude and synchronization measured by electroencephalography (EEG)	Baseline, Week 12

Trial Locations

Locations (1)

Stanford University School of Medicine; 🇺🇸 Stanford, California, United States