Clinical trial NCT05664789: Targeting the Neurobiology of RRB in Autism Using N-acetylcysteine: Trial
Official title: Targeting the Neurobiology of Restricted and Repetitive Behaviors in Children With Autism Using N-acetylcysteine: A Randomized, Controlled Trial
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
PHASE3
Conditions: Autism Spectrum Disorder
Keywords: Magnetic resonance imaging
N-acetyl cysteine
Restricted and repetitive behaviors
Electroencephalography
Lead sponsor: Stanford University
Sponsor class: OTHER
Collaborators: name: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
First posted: 2022-12-27
Enrollment: 48
Sex: ALL
Minimum age (years): 3
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Double-blind, randomized, placebo-controlled trial 2 arms, N-acetylcysteine and placebo
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Change in Children's Yale Brown-Obsessive Compulsive Scale for Autism Spectrum Disorder (CYBOCS-ASD) at 12 weeks
timeFrame: Screening and week 12
measure: Change in Glutamatergic neurometabolites at 12 weeks
timeFrame: Baseline and week 12
Secondary outcomes:
measure: Change in Restricted and Repetitive Behavior Scale Revised (RBS-R) at 12 weeks
timeFrame: Baseline and week 12
measure: Change in Gamma band activity at 12 weeks
timeFrame: Baseline and week 12
Other outcomes:
Locations:
city: Palo Alto
country: United States
facility: Department of Psychiatry and Behavioral Sciences
state: California
Study officials:
affiliation: Stanford University
name: John Hegarty, PhD
role: PRINCIPAL_INVESTIGATOR
References: