A randomized placebo-controlled pilot study of N-acetylcysteine in youth with autism spectrum disorder
| Autor: | Christopher J. McDougle, Ryan Adams, David J. Posey, Logan K. Wink, James E. Klaunig, Zemin Wang, Craig A. Erickson, Martin H. Plawecki |
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| Rok vydání: | 2016 |
| Předmět: |
Male
medicine.medical_specialty Neurology Autism Spectrum Disorder Autism Administration Oral Pilot Projects Social impairment Placebo Drug Administration Schedule Acetylcysteine 03 medical and health sciences 0302 clinical medicine Double-Blind Method Developmental Neuroscience Internal medicine medicine Humans Child Social Behavior Adverse effect Homocysteine Molecular Biology Research Free Radical Scavengers Venous blood Placebo Effect medicine.disease Glutathione N-acetylcysteine 030227 psychiatry Oxidative Stress Psychiatry and Mental health Tolerability Autism spectrum disorder Child Preschool Female Comet Assay Psychology Oxidation-Reduction 030217 neurology & neurosurgery DNA Damage Follow-Up Studies Developmental Biology medicine.drug Clinical psychology |
| Zdroj: | Molecular Autism |
| ISSN: | 2040-2392 |
| Popis: | Background Social impairment is a defining feature of autism spectrum disorder (ASD) with no demonstrated effective pharmacologic treatments. The goal of this study was to evaluate efficacy, safety, and tolerability of oral N-acetylcysteine (NAC), an antioxidant whose function overlaps with proposed mechanisms of ASD pathophysiology, targeting core social impairment in youth with ASD. Methods This study was a 12-week randomized, double-blind, placebo-controlled trial of oral NAC in youth with ASD. Study participants were medically healthy youth age 4 to 12 years with ASD, weighing ≥15 kg, and judged to be moderately ill based on the Clinical Global Impressions Severity scale. The participants were randomized via computer to active drug or placebo in a 1:1 ratio, with the target dose of NAC being 60 mg/kg/day in three divided doses. The primary outcome measure of efficacy was the Clinical Global Impressions Improvement (CGI-I) scale anchored to core social impairment. To investigate the impact of NAC on oxidative stress markers in peripheral blood, venous blood samples were collected at screen and week 12. Results Thirty-one patients were enrolled (NAC = 16, placebo = 15). Three participants were lost to follow-up, and three left the trial due to adverse effects. The average daily dose of NAC at week 12 was 56.2 mg/kg (SD = 9.7) with dose ranging from 33.6 to 64.3 mg/kg. The frequency of adverse events was so low that comparisons between groups could not be conducted. At week 12, there was no statistically significant difference between the NAC and placebo groups on the CGI-I (p > 0.69) but the glutathione (GSH) level in blood was significantly higher in the NAC group (p 0.16). Conclusions The results of this trial indicate that NAC treatment was well tolerated, had the expected effect of boosting GSH production, but had no significant impact on social impairment in youth with ASD. Trial registration Clinicaltrails.gov NCT00453180 |
| Databáze: | OpenAIRE |
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