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Brief Report: An Open-Label Study of the Neurosteroid Pregnenolone in Adults with Autism Spectrum Disorder

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Abstract

The objective of this study was to assess the tolerability and efficacy of pregnenolone in reducing irritability in adults with autism spectrum disorder (ASD). This was a pilot, open-label, 12-week trial that included twelve subjects with a mean age of 22.5 ± 5.8 years. Two participants dropped out of the study due to reasons unrelated to adverse effects. Pregnenolone yielded a statistically significant improvement in the primary measure, Aberrant Behavior Checklist (ABC)-Irritability [from 17.4 ± 7.4 at baseline to 11.2 ± 7.0 at 12 weeks (p = 0.028)]. Secondary measures were not statistically significant with the exception of ABC-lethargy (p = 0.046) and total Short Sensory Profile score (p = 0.009). No significant vital sign changes occurred during this study. Pregnenolone was not associated with any severe side effects. Single episodes of tiredness, diarrhea and depressive affect that could be related to pregnenolone were reported. Overall, pregnenolone was modestly effective and well-tolerated in individuals with ASD.

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Acknowledgments

This study was supported by a grant from the Escher Family Fund at the Silicon Valley Community Foundation to AYH and Mosbacher Fund to LKF. LKF is a recipient of the Ruth L. Kirschstein Individual Postdoctoral National Research Service Award. The authors would like to thank Pure Encapsulations (Sudbury, MA, USA) for donating the pregnenolone capsules.

Conflict of interest

Over the last 3 years, Dr. A.Y. Hardan has received research support and honorarium for consulting from the following companies: Bristol-Myers Squibb, Roche, Forest, and IntegraGen. Drs. L. K. Fung, J. Phillips, F. Haddad and Ms. R. A. Libove did not report any conflict.

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Authors and Affiliations

  1. Division of Child and Adolescent Psychiatry, Department of Psychiatry and Behavioral Sciences, Stanford University, 401 Quarry Road, Stanford, CA, 94305, USA

    Lawrence K. Fung, Robin A. Libove, Jennifer Phillips & Antonio Y. Hardan

  2. Division of Cardiovascular Medicine, Department of Medicine, Stanford University, 300 Pasteur Drive, Stanford, CA, 94305, USA

    Francois Haddad

Authors
  1. Lawrence K. Fung
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  2. Robin A. Libove
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  3. Jennifer Phillips
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  4. Francois Haddad
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  5. Antonio Y. Hardan
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Correspondence to Lawrence K. Fung.

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Fung, L.K., Libove, R.A., Phillips, J. et al. Brief Report: An Open-Label Study of the Neurosteroid Pregnenolone in Adults with Autism Spectrum Disorder. J Autism Dev Disord 44, 2971–2977 (2014). https://doi.org/10.1007/s10803-014-2144-4

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