Journal Article

STX209 (Arbaclofen) for Autism Spectrum Disorders: An 8-Week Open-Label Study

Erickson, Craig A., Walton-Bowen, Karen, Carpenter, Randall L., McCracken, James T., Cherubini, Maryann, Scahill, Lawrence, Veenstra-Vanderweele, Jeremy M., King, Bryan H., Melmed, Raun D., Zarevics, Peter, Bear, Mark F., Wang, Paul P., Ginsberg, Lawrence D., Sikich, Linmarie

2014

Overview

STX209 (arbaclofen), a selective GABA-B agonist, is hypothesized to modulate the balance of excitatory to inhibitory neurotransmission, and has shown preliminary evidence of benefit in fragile X syndrome. We evaluated its safety, tolerability, and efficacy in non-syndromic autism spectrum disorders, in an 8-week open-label trial enrolling 32 children and adolescents with either Autistic Disorder or Pervasive Developmental Disorder—Not Otherwise Specified, and a score ≥17 on the Aberrant Behavior Checklist (ABC)—Irritability subscale. STX209 was generally well-tolerated. The most common adverse events were agitation and irritability, which typically resolved without dose changes, and were often felt to represent spontaneous variation in underlying symptoms. Improvements were observed on several outcome measures in this exploratory trial, including the ABC-Irritability (the primary endpoint) and the Lethargy/Social Withdrawal subscales, the Social Responsiveness Scale, the CY-BOCS-PDD, and clinical global impression scales. Placebo-controlled study of STX209 is warranted.

• Publisher: Springer US,Springer,Springer Nature B.V
• Subject: Aberrant Behavior Checklist / Adolescence / Adolescent / Adolescents / Agitation / Akathisia, Drug-Induced - diagnosis / Antipsychotic drugs / Autism / Autism Spectrum Disorders / Autistic adolescents / Autistic children / Autistic spectrum disorders / Baclofen - adverse effects / Baclofen - analogs & derivatives / Baclofen - therapeutic use / Behavior Problems / Behavior Rating Scales / Behavioral Science and Psychology / Behavioral Sciences / Biological and medical sciences / Brain Hemisphere Functions / Check Lists / Child / Child and School Psychology / Child clinical studies / Child Development Disorders, Pervasive - drug therapy / Complications and side effects / Critical incidents / Developmental disabilities / Developmental disorders / Disorders / Dosage and administration / Drug Therapy / Efficacy / Female / Fragile X syndrome / GABA-B Receptor Agonists - adverse effects / GABA-B Receptor Agonists - therapeutic use / Gamma-aminobutyric acid / Humans / Impressions / Infantile autism / Irritability / Irritable Mood - drug effects / Labeling / Lethargy / Male / Measures / Measures (Individuals) / Medical sciences / Neurosciences / Original Paper / Outcome Measures / Outcomes of Treatment / Patient outcomes / Pediatrics / Pervasive Developmental Disorders / Pharmacology / Psychiatry / Psychological Patterns / Psychology / Psychology. Psychoanalysis. Psychiatry / Psychopathology. Psychiatry / Public Health / Responsiveness / Risk factors / Scores / Social Behavior / Symptoms / Symptoms (Individual Disorders) / Treatment Outcome / Withdrawal (Psychology)