Journal of Autism and Developmental Disorders

, Volume 44, Issue 4, pp 958–964

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

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

DOI: 10.1007/s10803-013-1963-z

Cite this article as:
Erickson, C.A., Veenstra-Vanderweele, J.M., Melmed, R.D. et al. J Autism Dev Disord (2014) 44: 958. doi:10.1007/s10803-013-1963-z

Abstract

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.

Keywords

STX209ArbaclofenGamma-aminobutyric acid (GABA)Autism spectrum disorderClinical trial

Copyright information

© Springer Science+Business Media New York 2013

Authors and Affiliations

  • Craig A. Erickson
    • 1
  • Jeremy M. Veenstra-Vanderweele
    • 2
  • Raun D. Melmed
    • 3
  • James T. McCracken
    • 4
  • Lawrence D. Ginsberg
    • 5
  • Linmarie Sikich
    • 6
  • Lawrence Scahill
    • 7
  • Maryann Cherubini
    • 8
  • Peter Zarevics
    • 8
  • Karen Walton-Bowen
    • 8
  • Randall L. Carpenter
    • 8
  • Mark F. Bear
    • 9
  • Paul P. Wang
    • 8
  • Bryan H. King
    • 10
  1. 1.Division of Child and Adolescent PsychiatryCincinnati Children’s Hospital Medical CenterCincinnatiUSA
  2. 2.Department of PsychiatryVanderbilt University School of MedicineNashvilleUSA
  3. 3.Southwest Autism Research and Resource CenterScottsdaleUSA
  4. 4.NPI-Semel InstituteUCLALos AngelesUSA
  5. 5.Red Oak Psychiatry AssociatesHoustonUSA
  6. 6.Department of PsychiatryUniversity of North Carolina at Chapel HillChapel HillUSA
  7. 7.Marcus Autism CenterEmory UniversityAtlantaUSA
  8. 8.Seaside Therapeutics, Inc.CambridgeUSA
  9. 9.Howard Hughes Medical Institute, Picower Institute for Learning and Memory, Department of Brain and Cognitive SciencesMassachusetts Institute of TechnologyCambridgeUSA
  10. 10.Department of Psychiatry and Behavioral Sciences, Seattle Children’s HospitalUniversity of WashingtonSeattleUSA