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

Autism Spectrum Disorder Clinical Trial

Official title:

Targeting the Neurobiology of Restricted and Repetitive Behaviors in Children With Autism Using N-acetylcysteine: A Randomized, Controlled Trial

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05664789
• Other study ID #: IRB-63262
• Secondary ID: 4R00HD101702-03
• Status: Recruiting
• Phase: Phase 2/Phase 3
• Start date: April 26, 2023
• Est. completion date: January 31, 2026

Study information

• Verified date: June 2024
• Source: Stanford University
• Contact: John Hegarty, PhD
• Phone: (650)723-7845
• Email: autismresearch[@]stanford.edu
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 48
• Est. completion date: January 31, 2026
• Est. primary completion date: January 31, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years to 12 Years

Eligibility

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

Related Conditions & MeSH terms

• Autism Spectrum Disorder
• Autistic Disorder

Intervention

Drug:
• N acetyl cysteine
N-acetylcysteine (NAC) is an over-the-counter dietary supplement that is relatively well tolerated and exhibits minimal side effects, even at high dosages. N
• Placebo
matched placebo

Locations

Country	Name	City	State
United States	Department of Psychiatry and Behavioral Sciences	Palo Alto	California

Sponsors (2)

Lead Sponsor	Collaborator
Stanford University	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in Children's Yale Brown-Obsessive Compulsive Scale for Autism Spectrum Disorder (CYBOCS-ASD) at 12 weeks	Severity of restricted and repetitive behaviors, higher scores indicate more severe behaviors	Screening and week 12
Primary	Change in Glutamatergic neurometabolites at 12 weeks	Glu and Glx measured by magnetic resonance imaging	Baseline and week 12
Secondary	Change in Restricted and Repetitive Behavior Scale Revised (RBS-R) at 12 weeks	Severity of restricted and repetitive behaviors, higher scores indicate more severe behaviors	Baseline and week 12
Secondary	Change in Gamma band activity at 12 weeks	Electrical activity between 30-48 Hz measured by electroencephalography	Baseline and week 12