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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)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01966679
Recruitment Status : Completed
First Posted : October 21, 2013
Last Update Posted : October 22, 2015
Information provided by (Responsible Party):
James McCracken, University of California, Los Angeles

Brief 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.

Condition or disease Intervention/treatment Phase
Autism Spectrum Disorder Drug: AZD7325 Drug: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 40 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, 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
Study Start Date : October 2013
Actual Primary Completion Date : July 2015
Actual Study Completion Date : July 2015

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Active Comparator: Double-blind (active versus placebo)
AZD7325 versus placebo
Drug: AZD7325
Other Name: sugar pill

Placebo Comparator: Placebo Drug: Placebo

Primary Outcome Measures :
  1. EEG [ Time Frame: week 6 ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years to 35 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes

Inclusion Criteria:

  • Subjects with a diagnosis of ASD as defined by DSM-5, confirmed by clinical evaluation and supported by the Autism Diagnostic Observation Schedule (ADOS)
  • Ages 18- 35 years inclusive
  • IQ estimate of >80
  • Aberrant Behavior Checklist (ABC)-Social Withdrawal Score >10 (>40% over population mean for developmentally disabled adults)
  • Existing allowed concomitant medication treatment stable for the 8 weeks prior to study entry, and no anticipated changes
  • Ability to comply with all protocol procedures and assessments
  • Availability of a reliable parent or caregiver willing to provide information regarding subject behavior and health status
  • Evidence of EEG biomarker deficit as defined below.

Exclusion Criteria:

  • Evidence of current drug or alcohol abuse or dependence
  • Prior history of drug or alcohol abuse or dependence in prior 12 months
  • History of seizure disorder (except febrile seizures)
  • Clinically significant aggressive, disruptive, or suicidal behavior in the 3 months prior to study enrollment
  • Presence of a chronic medical condition or prohibited medication (see list in Human Subjects section) which would potentially interfere with the assessment of treatment effects, or interact with study medications (eg. hepatic, neurologic, renal disease) to increase risk to the subject
  • History of paradoxical reactions to benzodiazepines
  • Clinically significant deviation from the reference range in clinical laboratory test results at screening, as judged by the investigator
  • ALT or AST greater than the upper limit of the laboratory standard reference range at screening
  • EKG abnormalities considered to be clinically significant as determined by the investigator and confirmed by an experienced cardiologist
  • Fredericia-corrected QT (QTcF) interval of >450 msec
  • Clinical judgment of the study physician of inability to perform the requirements of the study
  • For sexually active female and male subjects, refusal to agree to maintain a double-barrier birth control method during protocol participation

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01966679

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United States, California
Los Angeles, California, United States, 90095
United States, Georgia
Emory University
Atlanta, Georgia, United States
United States, Washington
Seattle Children's Research Institute
Seattle, Washington, United States, 98121
Sponsors and Collaborators
University of California, Los Angeles
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Principal Investigator: James McCracken, MD University of California, Los Angeles
Additional Information:
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Responsible Party: James McCracken, Department Chair, University of California, Los Angeles Identifier: NCT01966679    
Other Study ID Numbers: HHS-NIH-DA-12-241
First Posted: October 21, 2013    Key Record Dates
Last Update Posted: October 22, 2015
Last Verified: October 2015
Additional relevant MeSH terms:
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Autistic Disorder
Autism Spectrum Disorder
Child Development Disorders, Pervasive
Neurodevelopmental Disorders
Mental Disorders