Brief report: Pilot single-blind placebo lead-in study of acamprosate in youth with autistic disorder.
BACKGROUND
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).


OBJECTIVE
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.


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.


CONCLUSIONS
Future larger-scale dose finding studies of acamprosate in ASD may be warranted given this preliminary indication of benefit.
Department of Psychiatry, Indiana University School of Medicine, Indianapolis, IN, USA, craig.erickson@cchmc.org.
Craig A
CA
Erickson
Logan K
LK
Wink
Maureen C
MC
Early
Elizabeth
E
Stiegelmeyer
Lauren
L
Mathieu-Frasier
Vanessa
V
Patrick
Christopher J
CJ
McDougle
D000077443:Acamprosate; D000293:Adolescent; D001321:Autistic Disorder / Q000188:drug therapy*; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D008297:Male; D010865:Pilot Projects; D016037:Single-Blind Method; D012919:Social Behavior; D013654:Taurine / Q000031:analogs & derivatives* / Q000627:therapeutic use; D016896:Treatment Outcome
D013654:Taurine; D000077443:Acamprosate