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

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.

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Key information

Conditions

Autism Spectrum Disorders Anxiety Anxiety Disorders Autism Spectrum Disorder Child Development Disorders, Pervasive Mental Disorders Neurodevelopmental Disorders

Age range

6 year–17 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Buspirone

Drug

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.

Primary outcomes

Change From Baseline in Pediatric Anxiety Rating Scale (PARS)

Time frame: Baseline to week 8

The PARS is a clinician-rated scale to be used with parents and children. It consists of 57 items and has two sections: a symptom checklist (first 50 items) and severity items (last 7 items). The symptom checklist is used to determine the child's repertoire of symptoms during the past week. The 7 severity items are used to determine severity of symptoms and the PARS total score. The total score for the PARS is derived by summing the 7 severity items; total score ranges from 0 to 35, where higher scores indicate greater severity. The outcome reported reflects the change from baseline in PARS scores. When examining change from baseline, negative scores represent improvement (i.e., decrease in severity from baseline).
Clinician-rated Clinical Global Impression-Anxiety-Improvement (CGI-Anxiety-I) Scores of Improved or Very Much Improved at Study Endpoint

Time frame: Week 8 (study endpoint)

The CGI-Anxiety-I is a clinician rated measure of anxiety improvement. Improvement scores range from 1 (very much improved) to 7 (very much worse). This outcome reports the number of participants who scored ≤2 (improved or very much) at study endpoint.
Treatment Responder

Time frame: Week 8 (study endpoint)

Treatment responder is defined by a Clinician-rated Clinical Global Impression-Anxiety-Improvement (CGI-Anxiety-I) score ≤ 2 (improved or very much improved) and a ≥30% reduction on the Pediatric Anxiety Rating Scale (PARS) from baseline.

Sponsors and collaborators

Lead sponsor

Massachusetts General Hospital

Other

Registry information

Important dates

Study start: 2013
Primary completion: 2024
Study completion: 2024
First posted: May 9, 2013
Registry last updated: Feb 25, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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Completed