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Early Pharmacotherapy Aimed at Neuroplasticity in Autism : Safety and Efficacy

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: NCT00166621
Recruitment Status : Completed
First Posted : September 14, 2005
Last Update Posted : July 20, 2011
Information provided by:
Chugani, Diane C.

Tracking Information
First Submitted Date  ICMJE September 9, 2005
First Posted Date  ICMJE September 14, 2005
Last Update Posted Date July 20, 2011
Study Start Date  ICMJE March 2004
Primary Completion Date Not Provided
Current Primary Outcome Measures  ICMJE
 (submitted: April 9, 2007)
  • Safety will be measured by obtaining clinical laboratory tests, vital signs and evaluating probably or definitely related adverse events.
  • Population pharmacokinetics will be conducted to measure plasma concentrations in relation to the drug responses to buspirone.
  • The primary efficacy outcome will be the overall severity score from the Clinical Global Impressions assessment obtained from two raters, (parent and examiner)
  • Comparisons of allele, and genotype frequencies between responders and non-responders will be done for each polymorphism using Fisher's exact test to best predict response to buspirone.
Original Primary Outcome Measures  ICMJE Not Provided
Change History Complete list of historical versions of study NCT00166621 on Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
Descriptive Information
Brief Title  ICMJE Early Pharmacotherapy Aimed at Neuroplasticity in Autism : Safety and Efficacy
Official Title  ICMJE Early Pharmacotherapy Aimed at Neuroplasticity in Autism : Safety and Efficacy
Brief Summary The purpose of this study is to determine the efficacy, safety, and population pharmacokinetics and determinants of drug responses to buspirone in children with autism using a randomized, double blind, cross over study in children ages 2 to 6 years.
Detailed Description

Autism is a neurodevelopmental disorder defined as qualitative impairment in social interaction and communication and restrictive stereotype patterns of behavior, interests and activities. Pharmacological agents are being increasingly used off label in very young autistic children, and there is virtually no data regarding the pharmacokinetics, safety or efficacy of these agents in young children.

The approach in this study differs from pharmacotherapy studies of autism carried out thus far in several ways:

  • the rationale underlying our approach is based upon an attempt to alter synaptic plasticity during postnatal development, focusing on very young children
  • are integrating our drug trial with a PG study evaluating whether buspirone response is related to expression of genes involved in serotoninergic neurotransmission
  • will assess these variables together with in vivo assessment of serotonin synthesis capacity with PET.

This is a prospective, randomized, double blind, crossover study where children will be stratified by age into two groups. Treatment will last for 12 weeks with dosing twice a day. Parent ratings, cognitive tests and blood sampling will occur throughout the study period.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double
Primary Purpose: Treatment
Condition  ICMJE Autism
Intervention  ICMJE Drug: Buspirone
Study Arms  ICMJE Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Completed
Enrollment  ICMJE
 (submitted: September 9, 2005)
Original Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE August 2005
Primary Completion Date Not Provided
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Meet study definition for the diagnosis of autistic disorder
  • Age 2 to 6 (male or female)
  • Informed Consent

Exclusion Criteria:

  • Clinical or lab evidence of renal or hepatic disease
  • 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 3 months
  • Use of centrally acting drugs during the 6 weeks prior or during the study
  • Presence or history of neurological disorders, including seizure disorders
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 2 Years to 6 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
Administrative Information
NCT Number  ICMJE NCT00166621
Other Study ID Numbers  ICMJE PPRU 10659s
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Diane C Chugani, Wayne State University
Study Sponsor  ICMJE Chugani, Diane C.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Chugani, Diane C.
Verification Date July 2011

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP