MedPath

Buspirone in the Treatment of 2-6 Year Old Children With Autistic Disorder

Phase 2
Completed
Conditions
Autistic Disorder
Interventions
Drug: Placebo
Registration Number
NCT00873509
Lead Sponsor
Chugani, Diane C.
Brief Summary

The purpose of this study is to evaluate the effects of twice-daily oral buspirone on core features of autism in autistic children aged 2-6 years as measured by the change from baseline in the Autism Diagnostic Observation Schedule (ADOS) Composite Total scores compared to placebo at 6 months.

Detailed Description

This is a multi-center, randomized, placebo-controlled, double-masked study of 166 evaluable participants taking buspirone twice daily for 6 months. Children aged 2-6 years with autism will be randomized to receive one of three treatments: 2.5mg, 5.0mg, or matched placebo. The placebo controlled trial will be followed by an optional follow-up trial to assess the long term safety of buspirone. In addition, a PET scan of serotonin synthesis and plasma serotonin will be measured at baseline to determine whether these measures are predictors of drug response. This trial is aimed at the core features of autism. The outcome measures for efficacy will be examiner and parent ratings on psychological tests and questionnaires. The outcome measure for the primary objective will be the Autism Diagnostic Observation Scale (ADOS) Composite Total score. The behavioral outcomes for the secondary aims are delineated in the study design.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
166
Inclusion Criteria
  • Participants: must meet the study definition for diagnosis of autistic disorder as determined by clinical diagnosis based upon DSM-IV criteria, the Autism Diagnostic Interview-Revised (ADI-R) and the Autism Diagnostic Observation Schedule (ADOS) performed at baseline 1. ADI-R will be conducted by trained study staff at Baseline 1 Visit. If the participant has had an ADI-R in the past 12 months, this will be accepted provided the person administering and scoring the test is site personnel validated for the study.
  • Age 2 to less than 6 years, male and female.
  • Parent/Legal Guardian/Caregiver must be able to understand , read and speak English
  • Written Informed Consent.
Exclusion Criteria
  • Presence or history of neurological disorders, including seizure disorders (abnormal EEG without seizures will not be excluded), PKU, tuberous sclerosis, Rett syndrome, Fragile X syndrome, Down Syndrome and traumatic brain injury.
  • Other medical or behavioral problems requiring medications which are centrally active.
  • Clinical or laboratory evidence of renal or hepatic disease (SGPT, GGT > 2 x normal value, and serum creatinine > 1.5 x normal value).

Treatment with any medication known to alter the activity of the CYP3A4 enzyme including ketoconazole, itraconazole, grapefruit juice, erythromycin, clarithromycin, cimetidine, verapamil, diltiazem, rifampin, phenytoin, phenobarbital, or carbamazepine within the previous 2 months and for the duration of the study is prohibited.

  • Use of centrally acting drugs during the 6 weeks prior or during the study. These drugs include but are not limited to neuroleptics, benzodiazepines, anticonvulsants and antidepressants. Shorter times may be considered depending on the half life of the drug.
  • Prior treatment for periods longer than two weeks with buspirone or selective serotonin reuptake inhibitors. This includes herbal substances such as St John's Wort which have similar pharmacological actions.
  • Known allergies to study medication.
  • Unable to provide the required blood samples.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
1BuspironeBuspirone 2.5 mg
2BuspironeBuspirone 5.0 mg
3PlaceboPlacebo match
Primary Outcome Measures
NameTimeMethod
To evaluate the effects of twice-daily oral buspirone on core features of autism in autistic children 2-6 years measuring the change from baseline in ADOS (Autism Diagnostic Observation Schedule) Composite Total scores compared to placebo at 6 months.Baseline 1, Week 24
Secondary Outcome Measures
NameTimeMethod
To evaluate the effects of twice-daily oral buspirone on the ADOS Composite calibrated severity score, social behavior, repetitive behavior, language, sensory dysfunction and anxiety.Baseline 1, Week 24 and Week 48
To determine whether there is a difference in the incidence of side effects and long term safety between the buspirone and placebo groups, and between the different dose groups.Duration of the study
To determine whether blood serotonin concentration is a predictor of buspirone effect.Baseline 2
To determine whether there are age group differences in the effects of buspirone on social interaction, repetitive behavior, language, sensory dysfunction and anxiety.Baseline 1, Week 1, Week 24, Week 48
To determine whether the whole brain PET measure of serotonin synthesis capacity is a predictor of buspirone effect.Baseline 2

Trial Locations

Locations (6)

Cleveland Clinic Lerner College of Medicine

🇺🇸

Cleveland, Ohio, United States

Children's Hospital of Michigan Wayne State University

🇺🇸

Detroit, Michigan, United States

University California Davis M.I.N.D. Institute

🇺🇸

Sacramento, California, United States

New York University Langone Medical Center

🇺🇸

New York, New York, United States

Rainbow Babies and Children's Hospital

🇺🇸

Cleveland, Ohio, United States

University of Texas Southwestern

🇺🇸

Dallas, Texas, United States

© Copyright 2025. All Rights Reserved by MedPath