A Pilot Study 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 hypotheses for a larger randomized-controlled trial with explicit hypotheses and sufficient statistical power.

Study Overview

• Status: Not yet recruiting
• Conditions: Anxiety; Autism Spectrum Disorder
• Intervention / Treatment: Drug: Buspirone

• Study Type: Interventional
• Enrollment (Estimated): 15
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Chloe Hutt Vater, BA; Phone Number: 617-724-7301; Email: chuttvater@mgh.harvard.edu

Study Locations

• United States
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Massachusetts General Hospital
      • Contact: Chloe Hutt Vater, BA; Phone Number: 617-724-7301; Email: chuttvater@mgh.harvard.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years to 17 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Male or female participants between 6 and 17 years of age
• DSM-5 ASD diagnostic criteria as established by clinical diagnostic interview
• Participants with a score of ≥60 or more on the Anxiety/Depression subscale of CBCL
• Subjects can be taking psychotropic medications if they have been on the medication for at least 4 weeks prior to initiating study treatment and if they are on a stable dose, provided the medication is not listed in the Concomitant Medications section of the protocol.

Exclusion Criteria:

• 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 or bipolar disorder
• 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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: Buspirone; Buspirone tablets will be administered twice daily, and will be 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. During the titration phase, total dose will be increased by 10mg at each visit and by 5mg on the 4th day after each visit.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Reduction in Child and Adolescent Symptom Inventory-5-Anxiety (CASI-Anx) Score	The primary outcome measure of efficacy will be assessed by the reduction in anxiety symptom severity as measured by a change from baseline on the Child and Adolescent Symptom Inventory-5-Anxiety (CASI-Anx) scores. Responders are defined as those who demonstrate a >30% reduction on the CASI-Anx.	Baseline to 8 Weeks

Collaborators and Investigators

• Sponsor: Massachusetts General Hospital
• Investigators: Principal Investigator: Atilla Ceranoglu, MD, Massachusetts General Hospital

Study record dates

Study Major Dates

• Study Start (Estimated): December 1, 2024
• Primary Completion (Estimated): August 1, 2025
• Study Completion (Estimated): August 1, 2025

Study Registration Dates

• First Submitted: February 6, 2018
• First Submitted That Met QC Criteria: February 7, 2018
• First Posted (Actual): February 13, 2018

Study Record Updates

• Last Update Posted (Actual): March 13, 2024
• Last Update Submitted That Met QC Criteria: March 11, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Keywords: Anxiety; Autism Spectrum Disorders; Pervasive Developmental Disorders; Buspirone; Buspar
• Additional Relevant MeSH Terms: Mental Disorders; Neurodevelopmental Disorders; Anxiety Disorders; Autistic Disorder; Autism Spectrum Disorder; Child Development Disorders, Pervasive; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Central Nervous System Depressants; Tranquilizing Agents; Psychotropic Drugs; Serotonin Agents; Serotonin Receptor Agonists; Anti-Anxiety Agents; Buspirone
• Other Study ID Numbers: 2017-P-002731

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes