Targeting GABA-A for the Treatment of Social Disability in Young Adults With Autism Spectrum Disorders: A Phase II Proof of Mechanism Trial
Condition: Autism Spectrum Disorder
MeSH terms: Autistic Disorder; Autism Spectrum Disorder; Child Development Disorders, Pervasive
Study type: Interventional
Phase: Phase 2

Summary: 
      This study is a NIMH-funded multi-site clinical trial that includes UCLA as the coordinating
      site, with Emory University and Seattle Children's Hospital, as other recruiting sites, and
      the Nathan Kline Institute as the Data Management Center. The purpose of the study is to
      examine the effects of an investigational drug, AZD7325, as a potential treatment for
      high-functioning adults 18 -35 years old with Autism Spectrum Disorders (ASD). The primary
      study measures are effects on brain waves as measured by non-invasive brain wave recordings
      (electroencephalograms or EEGs), assessments of side effects, and measures of attention and
      learning.

      The study drug, AZD7325, is manufactured by Astra Zeneca, and was initially tested as a
      medication for anxiety disorders in over 488 subjects, but was not pursued for marketing due
      to too few benefits for anxiety. AZD7325 was found to have a very good safety profile and was
      tolerated by the majority of subjects. AZD7325 has some similar actions to currently marketed
      anxiety drugs in the benzodiazepine class, but lacks the sedative and negative effects on
      attention of the benzodiazepines. The study drug is designed to target the GABA
      neurotransmitter system which is believed to be abnormal in this population.

      There are 2 study phases. Phase 1 includes the recruitment of 24 healthy volunteers without
      mental disorder (6 per site) in order to establish normal EEG reference ranges. Controls will
      only be seen for one study visit which includes a clinical evaluation, physical exam, routine
      blood tests, and an EEG. Once control recruitment is complete, Phase 2 will begin.

      Phase 2 involves the recruitment of 40 adults (10 per site) 18 - 35 years old with a
      diagnosis of ASD, normal intelligence, and specific EEG patterns compared to control values.
      Screening for eligibility will be performed in one visit, which includes a clinical
      evaluation, tests of learning and intelligence, blood and urine tests, and an EEG. Those
      subjects who are found to be eligible will be enrolled in a 6-week medication study. Subjects
      with ASD who are enrolled will be randomly assigned to receive the study drug AZD7325 or
      placebo in matching capsules. Subjects will be seen weekly by study physicians and clincians
      for the 7 study visits, including 3 additional EEG recordings, and then for a final follow-up
      visit (9 total visits including screening lasting up to 11 weeks to complete). Study
      physicians can adjust the dose of study medication to reduce any side effects.
    


Intervention name(s): AZD7325; Placebo
Intervention type(s): Drug; Drug
Other intervention name(s): sugar pill
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment