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A Controlled Trial of Sertraline in Young Children with ASD


  • Perinatal/Infancy (0-12 months)
  • Early Childhood (3-5 years)


Autism Spectrum Disorder (ASD) is a behaviorally described disorder with many causes including genetic mutations, current estimate of over 1000 ASD related genes, neuroimmune mechanisms and possible exacerbation by environmental toxicity. Behavioral interventions are most effective if given early in development, such as the Early Start Denver Model, and we have seen the same to be true for psychopharmacological intervention. Because there is a deficit of serotonin synthesis in children with ASD who are under 5 years old we began the study of early low dose sertraline treatment in children who are 2 through 5 years old with fragile X syndrome (FXS) funded by HRSA. Both our preliminary retrospective data of sertraline treatment and now an interim analysis of the first 30 children who participated in our controlled trial of sertraline in FXS have demonstrated remarkable benefit from sertraline vs placebo in language, behavior and overall development. The current proposal is expanding the low dose sertraline (2.5 to 5.0 mg a day) treatment to children with idiopathic ASD who are 2 to 5 years old. This is a randomized double-blind placebo controlled trial of sertraline lasting 6 months with primary outcome measures of the Clinical Global Impression Scale (CGI-I) and the Mullen Scale of Early Learning. Secondary measures include the Preschool Language Scale, the ADOS, the Repetitive Behavior Scale- Revised, Preschool Anxiety Scale-Revised, Sensory Processing Scale- Preschool and the Visual Analogue Scale with parent chosen target behaviors. We will also assess biomarkers associated with ASD and response to sertraline including BDNF levels, allelic variants of BDNF, Serotonin Transporter gene (SCL6A4) and the serotonin- synthesizing tryptophan hydroxylase 2 (TPH2) gene in addition to cytokine and chemokine profiles both pre and post treatment. We will study 20 patients per year for a total of 60 patients after 3 years. Our data will not only demonstrate safety and efficacy of low dose sertraline but also the biomarkers that may correlate with sertraline treatment response. All of our patients will be receiving ASD behavioral interventions but we believe that the addition of sertraline will be very beneficial for the development of enhanced neuronal connectivity because sertraline enhances BDNF production and neurogenesis and also has anti-inflammatory effects. This is a critical time to show the benefit of an early sertraline treatment that can work synergistically with behavioral interventions to stem the tide of the growing prevalence of ASD in our children.

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