Autism Spectrum Disorder
Conditions
Keywords
Magnetic resonance imaging, N-acetyl cysteine, Restricted and repetitive behaviors, Electroencephalography
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.
Interventions
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
DRUGPlacebo
matched placebo
Sponsors
Stanford University
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Masking description
The placebo compound will match the active N-acetylcysteine in appearance, odor, taste, and packaging.
Intervention model description
Double-blind, randomized, placebo-controlled trial 2 arms, N-acetylcysteine and placebo
Eligibility
Sex/Gender
ALL
Age
3 Years to 12 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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 |
Countries
United States
Contacts
CONTACTRamy Chebbo
CONTACTJohn Hegarty, PhD
PRINCIPAL_INVESTIGATORJohn Hegarty, PhD
Stanford University
Outcome results
None listed