(PI Lawrence Scahill)
YDCC is the data coordination center for the Research Units on Pediatric Psychopharmacology (RUPP) Autism Network NIMH study titled “Guanfacine for the Treatment of Hyperactivity in Pervasive Development Disorder”. Pervasive developmental disorders (PDDs) such as autism, Asperger‘s disorder and PDD-Not Otherwise Specified (NOS) are chronic disorders of early childhood onset that are associated with substantial disability. Autism, the more severe form of PDD, is defined by significant delay in social interaction, delayed communication, and repetitive behaviors. PDD-NOS and Asperger‘s are often described as milder variants of autism. These disorders occur in all racial, ethnic, and social groups. Recent epidemiological studies indicate that autism and other PDDs are more common than previously believed. Empirical support for many pharmacological interventions in autism, however, is less than adequate due to small sample sizes evaluated in single site studies. Nonetheless, medication use is widespread in children with PDD. Recognizing this gap in evidence based medicine, the overall aim of this trial is to promote the development of an evidenced-based algorithm for the treatment of hyperactivity, impulsiveness and distractibility in children with PDD. To address this overall aim, this multicenter randomized, double blind, placebo controlled trial will evaluate the efficacy and safety of flexibly dosed guanfacine compared to placebo on parent-rated hyperactivity. Additionally we are evaluating stability of response to guanfacine in an extension phase and conducting a pilot trial of combined treatment with guanfacine plus methylphenidate in children partially responding to guanfacine.
The YDCC provides overall coordination of the study including management of regulatory matters (protocol history, protocol amendments, IRB approvals, DSMB correspondence), data
management and biostatistical services. Data management services include setting up data entry screens for remote data entry, training coordinators on data entry system and on the use of the subject calendar to track individual subjects and data quality within each site. The YDCC also prepares the quarterly Data and Safety Monitoring Board reports and study data for analysis. YDCC biostatisticians collaborate with the clinical leadership to carryout statistical analysis. The YDCC also conducts and documents the twice monthly coordinator teleconferences and the annual site visits.