Targeting GABA-A for the Treatment of Social Disability in Young Adults With Autism Spectrum Disorders: A Phase II Proof of Mechanism Trial (FAST-AS)
This study is a NIMH-funded multi-site clinical trial that includes UCLA as the coordinating site, with Emory University, Massachusetts General 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, AZ7325, 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, AZ7325, 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. AZ7325 was found to have a very good safety profile and was tolerated by the majority of subjects. AZ7325 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 AZ7325 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.
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
|Official Title:||Targeting GABA-A for the Treatment of Social Disability in Young Adults With Autism Spectrum Disorders: A Phase II Proof of Mechanism Trial|
- EEG [ Time Frame: week 6 ] [ Designated as safety issue: No ]
|Study Start Date:||October 2013|
|Estimated Study Completion Date:||December 2014|
|Estimated Primary Completion Date:||December 2014 (Final data collection date for primary outcome measure)|
Active Comparator: Double-blind (active versus placebo)
AZ7325 versus placebo
Other Name: sugar pill
|Placebo Comparator: Placebo||Drug: Placebo|
Please refer to this study by its ClinicalTrials.gov identifier: NCT01966679
|Contact: Jenny Cowen, PhDfirstname.lastname@example.org|
|United States, California|
|Los Angeles, California, United States, 90095|
|Principal Investigator: James T. McCracken, MD|
|Principal Investigator:||James McCracken, MD||University of California, Los Angeles|