Clinical Trial: Behavioral Effects of Kuvan in Children With Mild Phenylketonuria

Study Status: Terminated
Recruit Status: Terminated
Study Type: Observational

Official Title: Behavioral Effects of Kuvan in Children With Mild Phenylketonuria

Brief Summary: The purpose of this study is to determine whether improvements in behavior occur in children with phenylketonuria (PKU) who are taking Kuvan.

Detailed Summary:

Little research has been conducted to examine behavior and cognition in children with mild PKU/hyperphenylalanemia, but there is evidence of reductions in general intelligence (IQ) (Costello, 1994) and impairments in executive abilities (Diamond, 1994; Gassio, 2005) in this population. It is important to note that the phenylalanine levels of children with mild PKU are approximately equivalent to those of children with classical PKU whose phenylalanine levels have been managed through dietary control. In children with diet-treated PKU, impairments in behavior and cognition are well-documented, particularly in relation to executive abilities (Christ, 2006; White, 2001, 2002). Taken together, these findings suggest that children with mild PKU are at risk for behavioral and cognitive impairments, and it is possible that these impairments may be mitigated by lowering phenylalanine levels through treatment with Kuvan.

To investigate this issue, approximately 20 children with mild PKU from 6 to 18 years of age (inclusive) and their parents will participate in the study. The behavior and cognition of children with mild PKU will be assessed using the following methods: (1) Parents will complete inventories to rate the behavior and cognition of their children; (2) Older children will complete self-report inventories to rate their behavior and cognition; (3) Cognitive tasks assessing IQ and executive aspects of attention (i.e., sustained attention and inhibitory control) will be administered to all children.

The primary objectives are two-fold. First, we will determine if behavior and cognition are compromised in children with mild PKU prior to treatment with Kuvan (baseline). To accomplish this objective, we will administer measures of behavior and cognition that include normative data based on age. We hypothesize that children with
Sponsor: Washington University School of Medicine

Current Primary Outcome: Behavior Rating Inventory of Executive Function (BRIEF) [ Time Frame: baseline, 4-week follow-up, 24-week follow-up ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Behavior Assessment System for Children - Second Edition (BASC-2) [ Time Frame: baseline, 4-week follow-up, 24-week follow-up ]
  • Conners 3rd Edition (Conners 3) [ Time Frame: baseline, 4-week follow-up, 24-week follow-up ]
  • Conners Continuous Performance Test II Version 5 (CCPT-II Version 5) [ Time Frame: baseline, 4-week follow-up, 24-week follow-up ]
  • Matrix Reasoning subtest of the Wechsler Abbreviated Scale of Intelligence (WASI) [ Time Frame: baseline, 4-week follow-up, 24-week follow-up ]


Original Secondary Outcome: Same as current

Information By: Washington University School of Medicine

Dates:
Date Received: January 21, 2009
Date Started: January 2009
Date Completion:
Last Updated: December 3, 2013
Last Verified: December 2013