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

Sponsor

Stanford University (Other)

Overall Status

Not yet recruiting

CT.gov ID

NCT05664789

Collaborator

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

Enrollment

Arms

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. This is a 12-week double-blind, randomized, placebo-controlled trial of NAC.

Condition or Disease	Intervention/Treatment	Phase
Autism Spectrum Disorder	Drug: N acetyl cysteine Drug: Placebo	Phase 2/Phase 3

Study Design

Study Type:

Interventional

Anticipated Enrollment :

48 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Intervention Model Description:

Double-blind, randomized, placebo-controlled trial 2 arms, N-acetylcysteine and placeboDouble-blind, randomized, placebo-controlled trial 2 arms, N-acetylcysteine and placebo

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Masking Description:

The placebo compound will match the active N-acetylcysteine in appearance, odor, taste, and packaging.

Primary Purpose:

Treatment

Official Title:

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

Anticipated Study Start Date :

Feb 1, 2023

Anticipated Primary Completion Date :

Jan 31, 2026

Anticipated Study Completion Date :

Jan 31, 2026

Arms and Interventions

Arm	Intervention/Treatment
Active Comparator: N-acetylcysteine 12 week administration of active study compound	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 Comparator: Placebo 12 week administration of matched placebo	Drug: Placebo matched placebo

Arm

Intervention/Treatment

Active Comparator: N-acetylcysteine

12 week administration of active study compound

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 Comparator: Placebo

12 week administration of matched placebo

Drug: Placebo

matched placebo

Outcome Measures

Primary Outcome Measures

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

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
Change in Gamma band activity at 12 weeks [Baseline and week 12]
Electrical activity between 30-48 Hz measured by electroencephalography

Eligibility Criteria

Criteria

Ages Eligible for Study:

3 Years to 12 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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
medically stable
have stable medication regimens (≥ 30 days) and psychosocial treatments (≥ 60 days) prior to randomization with no anticipated changes during the trial
passes MR safety screening (e.g., no metal in the body)
successfully completes baseline neuroimaging (MRI or EEG)

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
the inability to drink a sample study compound dissolved in liquid