TY - JOUR
T1 - STX209 (Arbaclofen) for autism spectrum disorders
T2 - An 8-week open-label study
AU - Erickson, Craig A.
AU - Veenstra-Vanderweele, Jeremy M.
AU - Melmed, Raun D.
AU - McCracken, James T.
AU - Ginsberg, Lawrence D.
AU - Sikich, Linmarie
AU - Scahill, Lawrence
AU - Cherubini, Maryann
AU - Zarevics, Peter
AU - Walton-Bowen, Karen
AU - Carpenter, Randall L.
AU - Bear, Mark F.
AU - Wang, Paul P.
AU - King, Bryan H.
PY - 2014/4
Y1 - 2014/4
N2 - 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.
AB - 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.
KW - Arbaclofen
KW - Autism spectrum disorder
KW - Clinical trial
KW - Gamma-aminobutyric acid (GABA)
KW - STX209
UR - http://www.scopus.com/inward/record.url?scp=84896405056&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84896405056&partnerID=8YFLogxK
U2 - 10.1007/s10803-013-1963-z
DO - 10.1007/s10803-013-1963-z
M3 - Article
C2 - 24272415
AN - SCOPUS:84896405056
VL - 44
SP - 958
EP - 964
JO - Journal of Autism and Developmental Disorders
JF - Journal of Autism and Developmental Disorders
SN - 0162-3257
IS - 4
ER -