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Brief Report: Pilot Single-Blind Placebo Lead-In Study of Acamprosate in Youth with Autistic Disorder

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Abstract

Rationale

An excitatory/inhibitory (E:I) imbalance marked by enhanced glutamate and deficient gamma-aminobutyric acid (GABA) neurotransmission may contribute to the pathophysiology of autism spectrum disorders (ASD).

Objectives

We report on the first single-blind placebo lead-in trial of acamprosate, a drug with putative mechanisms restoring E:I imbalance, in twelve youth with ASD.

Materials and methods

We conducted a 12-week single-blind, placebo lead-in study of acamprosate in youth age 5–17 years with autistic disorder.

Results

Six of nine subjects who received active drug treatment were deemed treatment responders (defined by a score at final visit of “very much improved” or “much improved” on the Clinical Global Impressions Improvement scale) and ≥25 % improvement on the Aberrant Behavior Checklist Social Withdrawal subscale.

Conclusion

Future larger-scale dose finding studies of acamprosate in ASD may be warranted given this preliminary indication of benefit.

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Acknowledgments

This work is supported by an American Academy of Child and Adolescent Psychiatry (AACAP) Young Investigator Award (Dr. Erickson). The work is also supported in part by The Division of Disability and Rehabilitative Services, Indiana Family and Social Services Administration (Drs. Erickson, Wink); National Institute of Health Grant KL2 UL1 RR025761 Indiana University Clinical and Translational Sciences Institute Career Development Award (Dr. Erickson) and NIMH Grants R01 MH072964, R01 MH077600, R01 MH083739 (Dr. McDougle). The authors would also like to thank J.T Diener, Jennifer Mullett, and Arlene Kohn for their assistance with this project.

Conflict of interest

Dr. Erickson is the inventor on intellectual property held by Indiana University related to use of acamprosate in FXS and autism. Dr. Erickson holds equity in Confluence Pharmaceuticals, a company that has licensed this intellectual property from Indiana University.

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Author notes

  1. Craig A. Erickson, Logan K. Wink & Lauren Mathieu-Frasier

    Present address: Division of Child and Adolescent Psychiatry, Cincinnati Children’s Hospital Medical Center, 3333 Burnet Avenue, MLC 4002, Cincinnati, OH, 45215, USA

  2. Christopher J. McDougle

    Present address: Lurie Center for Autism, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA

Authors and Affiliations

  1. Department of Psychiatry, Indiana University School of Medicine, Indianapolis, IN, USA

    Craig A. Erickson, Logan K. Wink, Maureen C. Early, Elizabeth Stiegelmeyer, Lauren Mathieu-Frasier, Vanessa Patrick & Christopher J. McDougle

  2. Christian Sarkine Autism Treatment Center, James Whitcomb Riley Hospital for Children, Indianapolis, IN, USA

    Craig A. Erickson, Logan K. Wink, Maureen C. Early, Elizabeth Stiegelmeyer, Lauren Mathieu-Frasier, Vanessa Patrick & Christopher J. McDougle

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  1. Craig A. Erickson
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Correspondence to Craig A. Erickson.

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Erickson, C.A., Wink, L.K., Early, M.C. et al. Brief Report: Pilot Single-Blind Placebo Lead-In Study of Acamprosate in Youth with Autistic Disorder. J Autism Dev Disord 44, 981–987 (2014). https://doi.org/10.1007/s10803-013-1943-3

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  • DOI: https://doi.org/10.1007/s10803-013-1943-3

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