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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.

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.

Condition or disease Intervention/treatment Phase
Autism Drug: Buspirone Phase 1 Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Enrollment : 20 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double
Primary Purpose: Treatment
Official Title: Early Pharmacotherapy Aimed at Neuroplasticity in Autism : Safety and Efficacy
Study Start Date : March 2004
Actual Study Completion Date : August 2005

Resource links provided by the National Library of Medicine

Primary Outcome Measures :
  1. Safety will be measured by obtaining clinical laboratory tests, vital signs and evaluating probably or definitely related adverse events.
  2. Population pharmacokinetics will be conducted to measure plasma concentrations in relation to the drug responses to buspirone.
  3. The primary efficacy outcome will be the overall severity score from the Clinical Global Impressions assessment obtained from two raters, (parent and examiner)
  4. 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.

Information from the National Library of Medicine

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, Learn About Clinical Studies.

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Ages Eligible for Study:   2 Years to 6 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00166621

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United States, Michigan
PET Center/Children's Hospital of Michigan
Detroit, Michigan, United States, 48201
Sponsors and Collaborators
Chugani, Diane C.

Additional Information:
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Responsible Party: Diane C Chugani, Wayne State University Identifier: NCT00166621    
Other Study ID Numbers: PPRU 10659s
First Posted: September 14, 2005    Key Record Dates
Last Update Posted: July 20, 2011
Last Verified: July 2011
Keywords provided by Chugani, Diane C.:
Additional relevant MeSH terms:
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Autistic Disorder
Autism Spectrum Disorder
Child Development Disorders, Pervasive
Neurodevelopmental Disorders
Mental Disorders
Anti-Anxiety Agents
Tranquilizing Agents
Central Nervous System Depressants
Physiological Effects of Drugs
Psychotropic Drugs
Serotonin Receptor Agonists
Serotonin Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action