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

Study Overview
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Brief Summary
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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.

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

Conditions
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Autism Spectrum Disorder

Intervention / Treatment
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* Drug: N acetyl cysteine
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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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 passes MR safety screening (e.g., no metal in the body) and attempts baseline neuroimaging (MRI or EEG) 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

Ages Eligible for Study
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Minimum Age: 3 Years
Maximum Age: 12 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Interventional Model Description: Double-blind, randomized, placebo-controlled trial 2 arms, N-acetylcysteine and placebo
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: N-acetylcysteine<br>12 week administration of active study compound | Drug: N acetyl cysteine<br>* 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<br>|
| Placebo Comparator: Placebo<br>12 week administration of matched placebo | Drug: Placebo<br>* matched placebo<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 
| Change in Glutamatergic neurometabolites at 12 weeks | Glu and Glx measured by magnetic resonance imaging | Baseline and week 12 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 
| Change in Gamma band activity at 12 weeks | Electrical activity between 30-48 Hz measured by electroencephalography | Baseline and week 12 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Magnetic resonance imaging, N-acetyl cysteine, Restricted and repetitive behaviors, Electroencephalography