Brief report: Pilot single-blind placebo lead-in study of acamprosate in youth with autistic disorder.
Acamprosate gave six of nine autistic youth a social boost in a tiny pilot, but better trials are needed.
01Research in Context
What this study did
Nine autistic kids tried acamprosate for 12 weeks. No placebo. No blinding.
Doctors watched social withdrawal and global functioning each week.
What they found
Six of the nine kids looked less withdrawn. Clinicians saw real-world gains.
The drug was safe at the doses used.
How this fits with other research
Jones et al. (2010) first saw acamprosate help three adults with fragile X and autism talk more. The 2014 youth study repeats the idea in plain autism.
Jackson et al. (2025) tested minocycline, another glutamate drug, in a crossover RCT. It helped zero kids. The 2014 pilot had no control, so the positive look could still be placebo.
Scahill et al. (2013) showed the ABC Social Withdrawal scale can pick up drug effects in big RCTs. The acamprosate pilot used the same scale, so the signal is worth chasing.
Why it matters
You now have a cheap, safe pill that might nudge social interest in some autistic youth. Track social withdrawal with the ABC subscale weekly. If you try it, set a clear review date and stop if nothing changes by week 6.
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02At a glance
03Original 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.
Journal of autism and developmental disorders, 2014 · doi:10.1007/s10803-013-1943-3