- Craig A. Erickson1,
- Jeremy M. Veenstra-Vanderweele2,
- Raun D. Melmed3,
- James T. McCracken4,
- Lawrence D. Ginsberg5,
- Linmarie Sikich6,
- Lawrence Scahill7,
- Maryann Cherubini8,
- Peter Zarevics8,
- Karen Walton-Bowen8,
- Randall L. Carpenter8,
- Mark F. Bear9,
- Paul P. Wang8 &
- …
- Bryan H. King10
2210Accesses
6Altmetric
1Mention
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.
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Acknowledgments
This study was funded by Seaside Therapeutics, Inc. We are grateful to the families who volunteered to participate in this study. Several of the authors are full-time employees of Seaside Therapeutics, Inc., and some hold equity in Seaside Therapeutics, Inc., the sponsor of the study. The remaining authors all served as investigators in the study, and were compensated for their study efforts by Seaside Therapeutics, Inc. Some of these authors also have served as paid consultants to Seaside Therapeutics, Inc.
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Authors and Affiliations
Division of Child and Adolescent Psychiatry, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH, USA
Craig A. Erickson
Department of Psychiatry, Vanderbilt University School of Medicine, Nashville, TN, USA
Jeremy M. Veenstra-Vanderweele
Southwest Autism Research and Resource Center, Scottsdale, AZ, USA
Raun D. Melmed
NPI-Semel Institute, UCLA, 760 Westwood Plaza, Los Angeles, CA, USA
James T. McCracken
Red Oak Psychiatry Associates, Houston, TX, USA
Lawrence D. Ginsberg
Department of Psychiatry, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA
Linmarie Sikich
Marcus Autism Center, Emory University, Atlanta, GA, USA
Lawrence Scahill
Seaside Therapeutics, Inc., 840 Memorial Drive, Cambridge, MA, 02139, USA
Maryann Cherubini, Peter Zarevics, Karen Walton-Bowen, Randall L. Carpenter & Paul P. Wang
Howard Hughes Medical Institute, Picower Institute for Learning and Memory, Department of Brain and Cognitive Sciences, Massachusetts Institute of Technology, Cambridge, MA, USA
Mark F. Bear
Department of Psychiatry and Behavioral Sciences, Seattle Children’s Hospital, University of Washington, Seattle, WA, USA
Bryan H. King
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Erickson, C.A., Veenstra-Vanderweele, J.M., Melmed, R.D.et al. STX209 (Arbaclofen) for Autism Spectrum Disorders: An 8-Week Open-Label Study.J Autism Dev Disord44, 958–964 (2014). https://doi.org/10.1007/s10803-013-1963-z
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