Trial reportNeuropsychopharmacology : official publication of the American College of Neuropsychopharmacology2017
Arbaclofen in Children and Adolescents with Autism Spectrum Disorder: A Randomized, Controlled, Phase 2 Trial.
Trial report in Neuropsychopharmacology : official publication of the American College of Neuropsychopharmacology, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 77 papers, 6 of them syntheses that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
77 citing papers in PubMed, 6 syntheses or guidelines pooled it, 159 citations in OpenAlex.
- Effects of Probiotic Supplementation on Core Symptoms of Autism Spectrum Disorder in Children.Nutrients · 2026Pooled it
- Navigating agitation in neurodevelopmental disorders: A comparative study of pharmacotherapies via network meta-analysis in children and adults with autism spectrum disorder or intellectual disabilities.Journal of psychopharmacology (Oxford, England) · 2025Pooled it
- Neurometabolite differences in Autism as assessed with Magnetic Resonance Spectroscopy: A systematic review and meta-analysis.Neuroscience and biobehavioral reviews · 2024Pooled it
- Pharmacological intervention for irritability, aggression, and self-injury in autism spectrum disorder (ASD).The Cochrane database of systematic reviews · 2023Pooled it
- Interventions on Microbiota: Where Do We Stand on a Gut-Brain Link in Autism? A Systematic Review.Nutrients · 2022Pooled it
- Placebo response in pharmacological and dietary supplement trials of autism spectrum disorder (ASD): systematic review and meta-regression analysis.Molecular autism · 2020Pooled it
- Differential GABA dynamics across brain functional networks in autism.Communications biology · 2026Trial
- Double-blind, sham-controlled, pilot study of trigeminal nerve stimulation for autism spectrum disorder.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2026Trial
- Anxiety, repetitive and restricted behaviors and interests, and social communication in autistic adults: an exploratory analysis of a phase 3, randomized clinical trial.Scientific reports · 2025Trial
- Retinal GABAergic Alterations in Adults with Autism Spectrum Disorder.The Journal of neuroscience : the official journal of the Society for Neuroscience · 2024Trial
- Exploratory evidence for differences in GABAergic regulation of auditory processing in autism spectrum disorder.Translational psychiatry · 2023Trial
- Safety and target engagement of an oral small-molecule sequestrant in adolescents with autism spectrum disorder: an open-label phase 1b/2a trial.Nature medicine · 2022Trial
- Baclofen as an adjuvant therapy for autism: a randomized, double-blind, placebo-controlled trial.European child & adolescent psychiatry · 2019Trial
- Drug Repurposing for Treating NCOR-Related Intellectual Disability.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- Beyond the Outburst: Charting a New Frontier for Understanding and Treating Irritability in Autistic Adults.Autism research : official journal of the International Society for Autism Research · 2026Review
- Medical treatment of autism spectrum disorder in children: Current evidence, controversies, and clinical challenges.World journal of clinical pediatrics · 2026Article
- Review
- Efficacy, safety, and tolerability of arbaclofen in Autistic children and adolescents, the AIMS-2-TRIALS-CT1: a randomized, double-blind, placebo-controlled phase II trial.EClinicalMedicine · 2026Article
- Mapping Global Research Trends on Autism Spectrum Disorder: A Bibliometric Analysis of Pharmacology and Pharmacy Studies.Pharmaceuticals (Basel, Switzerland) · 2026Article
- Novel therapeutics in autism spectrum disorder.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2026Review
17 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
9 authors at 7 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Several lines of emerging data point to an imbalance between neuronal excitation and inhibition in at least a subgroup of individuals with autism spectrum disorder (ASD), including in those with fragile X syndrome (FXS), one of the most common genetic syndromes within ASD. In animal models of FXS and of ASD, GABA-B agonists have improved both brain and behavioral phenotypes, including social behavior. A phase 2 randomized, placebo-controlled, crossover trial found that the GABA-B agonist arbaclofen improved social avoidance symptoms in FXS. A pilot open-label trial of arbaclofen suggested similar benefits in ASD. We therefore evaluated arbaclofen in a randomized, placebo-controlled, phase 2 study of 150 participants, aged 5-21 years, with ASD. No difference from placebo was detected on the primary outcome measure, the parent-rated Aberrant Behavior Checklist Social Withdrawal/Lethargy subscale. However, a specified secondary analysis found improvement on the clinician-rated Clinical Global Impression of Severity. An exploratory post hoc analysis of participants with a consistent rater across the trial revealed greater improvement in the Vineland Adaptive Behavior Scales II socialization domain in participants receiving arbaclofen. Affect lability (11%) and sedation (9%) were the most common adverse events. In this exploratory study, secondary analyses suggest that arbaclofen may have the potential to improve symptoms in some children with ASD, but further study will be needed to replicate and extend these initial findings.
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What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.