A pilot open label, single dose trial of fenobam in adults with fragile X syndrome.

Journal of Medical Genetics
Elizabeth M Berry-KravisRandi J Hagerman

Abstract

A pilot open label, single dose trial of fenobam, an mGluR5 antagonist, was conducted to provide an initial evaluation of safety and pharmacokinetics in adult males and females with fragile X syndrome (FXS). Twelve subjects, recruited from two fragile X clinics, received a single oral dose of 50-150 mg of fenobam. Blood for pharmacokinetic testing, vital signs and side effect screening was obtained at baseline and numerous time points for 6 h after dosing. Outcome measures included prepulse inhibition (PPI) and a continuous performance test (CPT) obtained before and after dosing to explore the effects of fenobam on core phenotypic measures of sensory gating, attention and inhibition. There were no significant adverse reactions to fenobam administration. Pharmacokinetic analysis showed that fenobam concentrations were dose dependent but variable, with mean (SEM) peak values of 39.7 (18.4) ng/ml at 180 min after the 150 mg dose. PPI met a response criterion of an improvement of at least 20% over baseline in 6 of 12 individuals (4/6 males and 2/6 females). The CPT did not display improvement with treatment due to ceiling effects. Clinically significant adverse effects were not identified in this study of single dose fenobam across...Continue Reading

References

May 29, 2002·Proceedings of the National Academy of Sciences of the United States of America·Kimberly M HuberMark F Bear
Mar 3, 2004·Mental Retardation and Developmental Disabilities Research Reviews·Andrea Beckel-Mitchener, William T Greenough
Mar 19, 2004·The Journal of Neuroscience : the Official Journal of the Society for Neuroscience·Laura N AntarGary J Bassell
Jun 29, 2004·Trends in Neurosciences·Mark F BearStephen T Warren
Nov 19, 2004·Proceedings of the National Academy of Sciences of the United States of America·Ivan Jeanne WeilerWilliam T Greenough
Feb 3, 2005·Proceedings of the National Academy of Sciences of the United States of America·Armaz AschrafiPeter W Vanderklish
Sep 2, 2005·The Journal of Neuroscience : the Official Journal of the Society for Neuroscience·Shih-Chieh ChuangRobert K S Wong
Jul 11, 2006·The Journal of Neuroscience : the Official Journal of the Society for Neuroscience·Aaron W GrossmanWilliam T Greenough
Oct 31, 2006·Journal of Child and Adolescent Psychopharmacology·Elizabeth Berry-KravisRandi Hagerman
Dec 22, 2006·Journal of Intellectual Disability Research : JIDR·Kelly SullivanDeborah Hatton
Sep 21, 2007·Proceedings of the National Academy of Sciences of the United States of America·Mika NakamotoStephen T Warren
Dec 21, 2007·Neuron·Gül DölenMark F Bear
Mar 29, 2008·Journal of Autism and Developmental Disorders·Elizabeth Berry-KravisJoanne Wuu
Apr 17, 2008·Journal of Medical Genetics·Paul J Hagerman
Jun 24, 2008·Neurobiology of Disease·Femke M S de VrijRob Willemsen

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Citations

Jul 13, 2011·Journal of Neurology·Anne Gallagher, Brian Hallahan
Jan 18, 2013·Journal of Molecular Modeling·Yoganjaneyulu Kasetti, Prasad V Bharatam
Jul 18, 2009·Journal of Autism and Developmental Disorders·Craig A EricksonChristopher J McDougle
Mar 10, 2010·Journal of Autism and Developmental Disorders·Craig A EricksonChristopher J McDougle
Jan 27, 2011·Journal of Autism and Developmental Disorders·Faraz FarzinDavid Hessl
Oct 6, 2011·Journal of Autism and Developmental Disorders·Stephanie M SansoneDavid Hessl
Sep 29, 2009·Neuroscience Bulletin·Zhao-Hui Xu, Ming-Gao Zhao
Dec 7, 2010·Biological Psychiatry·Michael J GandalSteven J Siegel
Jul 21, 2010·Neurotherapeutics : the Journal of the American Society for Experimental NeuroTherapeutics·Lulu W WangRandi J Hagerman
Aug 4, 2012·ACS Chemical Neuroscience·Aileen HealyTimothy Ocain
Apr 27, 2011·Molecular Psychiatry·P W Kalivas, N D Volkow
Apr 11, 2012·Molecular Psychiatry·J L RapoportN Gogtay
Apr 9, 2010·Nature Medicine·Nicola Jones
Aug 8, 2009·Nature Medicine·Dan Ehninger, Alcino J Silva
Apr 16, 2013·Nature Neuroscience·Jennifer C Darnell, Eric Klann
Dec 25, 2009·Neuropsychopharmacology : Official Publication of the American College of Neuropsychopharmacology·Jill L SilvermanJacqueline N Crawley
Jul 29, 2011·Neuropsychopharmacology : Official Publication of the American College of Neuropsychopharmacology·Christina GrossGary J Bassell
Aug 18, 2010·Proceedings of the National Academy of Sciences of the United States of America·Daniel Hoyer
Dec 27, 2011·Journal of Child and Adolescent Psychopharmacology·Presenter Swapna Deshpande, Discussant Barbara J Coffey
Apr 24, 2009·Cerebral Cortex·Jay E Brenman
Sep 4, 2010·Journal of Developmental and Behavioral Pediatrics : JDBP·Daniel Z Wetmore, Craig C Garner
Nov 9, 2011·Journal of Developmental and Behavioral Pediatrics : JDBP·Donald B BaileyElizabeth Berry-Kravis
May 15, 2013·Journal of Developmental and Behavioral Pediatrics : JDBP·Elizabeth Berry-KravisLeonard Abbeduto
Apr 11, 2013·Journal of Developmental and Behavioral Pediatrics : JDBP·Mary Jacena S LeighRandi J Hagerman
Sep 18, 2013·Journal of Developmental and Behavioral Pediatrics : JDBP·Elizabeth Berry-KravisUNKNOWN Outcome Measures Working Groups
Jun 16, 2010·Genetics in Medicine : Official Journal of the American College of Medical Genetics·Melissa K HillSylvia A Metcalfe
Aug 21, 2009·Epilepsy Currents·Paul J Hagerman, Carl E Stafstrom
Mar 30, 2010·Annual Review of Neuroscience·C ShilyanskyA J Silva
Dec 4, 2012·The Journal of Clinical Investigation·Claudia BagniRandi Hagerman
Oct 14, 2009·BMC Neurology·Jose-Ramon RuedaMaria-Isabel Tejada
Sep 23, 2010·Molecular Autism·Randi HagermanPaul Hagerman
May 28, 2013·Molecular Autism·Talakad G LohithRobert B Innis
Aug 5, 2010·Disease Models & Mechanisms·Balpreet Bhogal, Thomas A Jongens

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Methods Mentioned

BETA
pregnancy test
blood drawing
blood draws
sedation

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