An Open-Label Trial of Buspirone for the Treatment of Anxiety in Youth With Autism Spectrum Disorders

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2016 by Massachusetts General Hospital
Information provided by (Responsible Party):
Gagan Joshi, MD, Massachusetts General Hospital Identifier:
First received: April 22, 2013
Last updated: February 26, 2016
Last verified: February 2016
The main objective of this exploratory 8-week pilot study is to evaluate the safety and efficacy of buspirone for the treatment of anxiety in youth (ages 6-17 years) with autism spectrum disorders. The study results will be used to generate hypothesis for a larger randomized controlled clinical trials with explicit hypotheses and sufficient statistical power.

Condition Intervention
Autism Spectrum Disorders
Drug: Buspirone

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open-label Trial of Buspirone for the Treatment of Anxiety in Youth With Autism Spectrum Disorders

Resource links provided by NLM:

Further study details as provided by Massachusetts General Hospital:

Primary Outcome Measures:
  • Reduction in Pediatric Anxiety Rating Scale (PARS) score [ Time Frame: Baseline to 8 weeks ] [ Designated as safety issue: No ]
    Primary outcome measure of efficacy will be assessed by reduction in anxiety symptom severity as measured by change from baseline. Responders are defined as >/=30% reduction in PARS score.

  • Clinical Global Impression-Anxiety (CGI-Anxiety) Improvement Score [ Time Frame: Baseline to 8 weeks ] [ Designated as safety issue: No ]
    Primary outcome measure of efficacy will be assessed by reduction in anxiety symptom severity as measured by Clinical Global Impression-Anxiety (CGI-Anxiety). Responders are defined as a score of </=2 on the improvement sub scale.

Estimated Enrollment: 40
Study Start Date: July 2013
Estimated Study Completion Date: April 2017
Estimated Primary Completion Date: April 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Buspirone
Buspirone administered in tablets twice daily titrated to a maximum daily dose of 60mg for 8 weeks.
Drug: Buspirone
Children with autism spectrum disorders will receive buspirone treatment for eight weeks. Buspirone will be titrated to the maximum daily dose during the first four weeks of the trial (dose titration phase). Week 4 onwards, subjects will be maintained on maximum achieved dose until the end of the trial (dose maintenance pahe). During the titration phase, total dose will be increased by 10mg at each visit and by 5mg on the 4th day after each visit.


Ages Eligible for Study:   6 Years to 17 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Male or female participants between 6 and 17 years of age
  • Fulfills diagnosis of autism spectrum disorders by meeting DSM-IV-TR PDD diagnostic criteria of autistic disorder, Asperger's disorder, or PDD-NOS as established by clinical diagnostic interview
  • Participants with a score of ≥13 on the Pediatric Anxiety Rating Scale (PARS)
  • Participants with a score of ≥60 or more on the Anxiety/Depression subscale of CBCL and CGI-Anxiety severity of ≥ 4
  • Subjects can be on psychotropic drugs if they have been on the medication for at least 4 weeks prior to initiating trial treatment and if they are stable, provided the medication is not listed in the Concomitant Medications section of the protocol.
  • Subjects with disruptive behavior disorders, mood, or psychosis will be allowed to participate in the study provided they do not meet any exclusionary criteria

Exclusion Criteria:

  • I.Q. < 70
  • DSM-IV-TR PDD diagnoses of Rett's disorder, and childhood disintegrative disorder
  • History of active seizure disorder (EEG suggestive of seizure activity and/or history of seizure in last 1 month)
  • Subjects with a medical condition or treatment that will either jeopardize subject safety or affect the scientific merit of the study, including:
  • Pregnant or nursing females
  • Organic brain disorders
  • Uncorrected hypothyroidism or hyperthyroidism
  • Clinically significant abnormalities on ECG (e.g., QT prolongation, arrhythmia)
  • History of renal or hepatic impairment
  • Clinically unstable psychiatric conditions or judged to be at serious suicidal risk
  • Current diagnosis of schizophrenia
  • History of substance use (except nicotine or caffeine) within past 3 months or urine drug screen positive for substances of abuse
  • Current treatment with medication with primary central nervous system activity (as specified in the Concomitant Medication section of the protocol)
  • A non-responder or history of intolerance to buspirone, after treatment at an adequate dose and duration as determined by the clinician
  • Subjects currently taking monoamine oxidase inhibitors (MAOI) and/or CYP3A4 inducers or inhibitors including nefazodone, diltiazem, verapamil, erythromaycin, itraconazole, or rifampin.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01850355

Contact: Ryan Kilcullen 617-724-7301
Contact: Leah Feinberg, BS 617-726-4651

United States, Massachusetts
Massachusetts General Hospital Recruiting
Boston, Massachusetts, United States, 02114
Contact: Ryan Kilcullen    617-724-7301   
Contact: Leah Feinberg, BS    617-726-4651   
Sponsors and Collaborators
Massachusetts General Hospital
Principal Investigator: Gagan Joshi, MD Massachusetts General Hospital
  More Information

Responsible Party: Gagan Joshi, MD, Principal Investigator, Massachusetts General Hospital Identifier: NCT01850355     History of Changes
Other Study ID Numbers: 2013-P-000661 
Study First Received: April 22, 2013
Last Updated: February 26, 2016
Health Authority: United States: Food and Drug Administration

Keywords provided by Massachusetts General Hospital:
Autism Spectrum Disorders
Pervasive Developmental Disorders

Additional relevant MeSH terms:
Anxiety Disorders
Autism Spectrum Disorder
Autistic Disorder
Child Development Disorders, Pervasive
Mental Disorders
Neurodevelopmental Disorders
Pathologic Processes
Anti-Anxiety Agents
Central Nervous System Depressants
Molecular Mechanisms of Pharmacological Action
Neurotransmitter Agents
Physiological Effects of Drugs
Psychotropic Drugs
Serotonin Agents
Serotonin Receptor Agonists
Tranquilizing Agents processed this record on May 26, 2016