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

Sponsor

Stanford University (Other)

Overall Status

Not yet recruiting

CT.gov ID

NCT05494398

Collaborator

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (NIH)

Enrollment

Arm

Anticipated Duration (Months)

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Autism Spectrum Disorder	Drug: N-acetylcysteine	Phase 2/Phase 3

Study Design

Study Type:

Interventional

Anticipated Enrollment :

48 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Anticipated Study Start Date :

Sep 1, 2022

Anticipated Primary Completion Date :

Sep 1, 2027

Anticipated Study Completion Date :

Sep 1, 2028

Arms and Interventions

Arm	Intervention/Treatment
Experimental: N-Acetylcysteine	Drug: N-acetylcysteine 12 week administration of N-Acetylcysteine to target restricted repetitive behavior severity.

Arm

Intervention/Treatment

Experimental: N-Acetylcysteine

Drug: N-acetylcysteine

12 week administration of N-Acetylcysteine to target restricted repetitive behavior severity.

Outcome Measures

Primary Outcome Measures

Change in glutamatergic neurometabolites (Glx) measured by proton spectroscopy Magnetic Resonance Imaging (MRI) [Baseline and Week 12 of treatment]
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]

Secondary Outcome Measures

Change in Gamma band amplitude and synchronization measured by electroencephalography (EEG) [Baseline, Week 12]
Change in restricted repetitive behavior subtypes as measured by the Restricted Behavior Scale - Revised (RBS-R) [Baseline, week 8, week 12]

Eligibility Criteria

Criteria

Ages Eligible for Study:

3 Years to 12 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.