Project Detail
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Office of Autism Research Coordination (OARC) logo

1/4-RUPP autism network: Guanfacine for the treatment of hyperactivity in PDD  

This is a multi-site collaborative research project from The Research Units on Pediatric Psychopharmacology [RUPP] Autism Network (Indiana University, Seattle Children's Research Institute, UCLA, and Yale University). Recent data indicate that as many as 50% of children with pervasive developmental disorders (PDDs) have moderate to severe problems of hyperactivity and impulsiveness. Consensus is lacking on how to treat children with PDD accompanied by hyperactivity. Compared to typically developing children with ADHD, children with PDD often show less benefit and greater side effect burden. Guanfacine is commonly used in this population, but poorly studied. Pilot data indicate that guanfacine is a promising treatment for hyperactivity in children with PDD with a good tolerability profile. In addition, biomarkers have been identified (genetic and neurochemical) that may be associated with positive effects. This study involves an 8-week randomized, double-blind, placebo- controlled trial of guanfacine for 170 children (ages 5-13 years) with PDD accompanied by hyperactivity and impulsiveness. Subjects who show a positive response will be invited to enter an 8-week Extension phase (treatment mask will not be broken). The treatment blind will be broken for children who do not achieve a positive response in the double-blind phase. Children who show no change or deterioration on placebo will be treated with guanfacine in an 8-week Open-label phase. Children who show a partial response to guanfacine will be randomly assigned to a 4-week add-on trial of methylphenidate or placebo to evaluate the potential benefits of combined treatment. Fifty subjects are expected to enter this pilot trial. The role of gene variants and urinary adrenergic/noradrenergic measures as biomarkers in moderating response to guanfacine on primary efficacy measures and adverse effects will also be explored. Project Status


Funder National Institutes of Health
Fiscal Year Funding $366,035.00
Current Award Period 2010-2013
Project Number 1R01MH083739-01A2
Principal Investigator McDougle, Christopher
Received ARRA Funding? No
Strategic Plan Question Question 4: Which Treatments And Interventions Will Help? (Treatments)
Subcategory Medical/Pharmacologic
Strategic Plan Objective Green dot: Objective has greater than or equal to the recommended funding. 4LC. Conduct at least one study to evaluate the safety and effectiveness of medications commonly used in the treatment of co-occurring conditions or specific behavioral issues in people with ASD by 2015. IACC Recommended Budget: $10,000,000 over 5 years.
Federal or Private? Federal
Institution Indiana University-Purdue University Indianapolis
State/Country Indiana
Web Link 1 1/4-RUPP autism network: Guanfacine for the treatment of hyperactivity in PDD (External web link)
Web Link 2 No URL available.
Web Link 3 No URL available.
History/Related Projects N/A