Arbaclofen in Children and Adolescents With ASD
Condition: Autism Spectrum Disorder
MeSH terms: Autism Spectrum Disorder
Study type: Interventional
Phase: Phase 2

Summary: 
      AIMS-2-CT-01 is a randomized, double-blind, placebo controlled, study to explore the
      efficacy, safety and tolerability of Arbaclofen administered to children and adolescents
      (ages 5-17) for the treatment of social adaptive function in participants with ASD. The
      effects of Arbaclofen on social function in children and adolescents with ASD will be
      evaluated in a randomized, placebo controlled, parallel-group study of 16 weeks duration.
      Subjects who meet protocol criteria will be randomly allocated to receive either Arbaclofen
      or placebo in a 1:1 ratio in the Treatment Period. There will be 7 recruiting sites and
      randomization will be stratified by site. A sample of 130 patients will be recruited.
      Blinding will be maintained by utilizing identical tablets containing either Arbaclofen or
      placebo.
    

Detailed description: 
      Autism Spectrum Disorder (ASD) is a clinically and etiologically heterogeneous
      neurodevelopmental condition affecting approximately 1% of the population. The core symptoms
      of ASD are deficits in social communication and the presence of repetitive and restricted
      behaviours and interests, including sensory anomalies. Currently, there are no effective
      medical treatments for the core symptoms of ASD, and families frequently use costly non
      evidence based interventions. Developing drugs for ASD has been challenging because of a
      limited understanding of its underlying pathophysiology(ies), and difficulties modelling it
      in vitro and in vivo.

      A recent study from EU-AIMS reported, for the first time in ASD, that differences in E-I
      balance can be 'shifted' using a GABA acting drug (riluzole), and that abnormalities in
      functional connectivity can be 'normalised' by targeting E-I, even in adults. This offers
      promise that drugs targeting specific parts of the GABA pathway may improve symptoms.

      The aim of the investigator's project is to conduct a double-blind Randomized Control Trial
      (RCT) focused on GABA/glutamate equilibrium, to assess the efficacy of a drug that targets
      core and/or comorbid symptoms in ASD. Arbaclofen is a selective GABA-B receptor agonist and
      augments GABA-ergic activity, inhibits presynaptic release of glutamate, inhibits
      postsynaptic transmission, and modulates intracellular signalling. Through elevation of
      GABA-ergic inhibitory activity, Arbaclofen may act to alleviate ASD symptoms with social
      anxiety and emotional hyperarousal.

      Hypothesis: Arbaclofen will be superior to placebo in improving social function as measured
      by the Vineland-3 social domain.
    

Intervention name(s): Arbaclofen; Placebo
Intervention type(s): Drug; Drug
Intervention description(s): Arbaclofen tablet.; Placebo tablet.
Other intervention name(s): R-4-amino-3-(4-chlorophenyl) butanoic acid (R-baclofen); STX209; C10H12ClNO2; Placebo (for Arbaclofen)
Intervention MeSH terms: Baclofen
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: Arbaclofen; Placebo-Controlled; Randomized; Double-Blind; Social Function; Efficacy; Safety; Tolerability; Children; Adolescents; ASD