Behavioral Effects of Kuvan in Children With Mild Phenylketonuria
The purpose of this study is to determine whether improvements in behavior occur in children with phenylketonuria (PKU) who are taking Kuvan.
|Study Design:||Observational Model: Case-Only
Time Perspective: Prospective
|Official Title:||Behavioral Effects of Kuvan in Children With Mild Phenylketonuria|
- Behavior Rating Inventory of Executive Function (BRIEF) [ Time Frame: baseline, 4-week follow-up, 24-week follow-up ] [ Designated as safety issue: No ]
- Behavior Assessment System for Children - Second Edition (BASC-2) [ Time Frame: baseline, 4-week follow-up, 24-week follow-up ] [ Designated as safety issue: No ]
- Conners 3rd Edition (Conners 3) [ Time Frame: baseline, 4-week follow-up, 24-week follow-up ] [ Designated as safety issue: No ]
- Conners Continuous Performance Test II Version 5 (CCPT-II Version 5) [ Time Frame: baseline, 4-week follow-up, 24-week follow-up ] [ Designated as safety issue: No ]
- Matrix Reasoning subtest of the Wechsler Abbreviated Scale of Intelligence (WASI) [ Time Frame: baseline, 4-week follow-up, 24-week follow-up ] [ Designated as safety issue: No ]
|Study Start Date:||January 2009|
|Study Completion Date:||January 2010|
|Primary Completion Date:||January 2010 (Final data collection date for primary outcome measure)|
Individuals with mild phenylketonuria/hyperphenylalanemia who are beginning treatment with Kuvan.
20/mg/kg/day taken once daily or as otherwise prescribed by physician as standard care.
Other Name: Sapropterin
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 mild PKU will have ratings and scores that are ≥ 1 standard deviation from the normative mean. Second, we will determine if behavior and cognition improve in children with mild PKU following treatment with Kuvan. To accomplish this objective, we will administer the same measures of behavior and cognition after 4 and 24 weeks of treatment with Kuvan(4-week and 24-week follow-ups, respectively). We hypothesize that the follow-up ratings and scores of children with mild PKU will improve by ≥ 0.5 standard deviation relative to their baseline ratings and scores.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00827762
|United States, Illinois|
|Northwestern University/Children's Memorial Hospital|
|Chicago, Illinois, United States, 60614|
|United States, Missouri|
|University of Missouri|
|Columbia, Missouri, United States, 65211|
|St. Louis, Missouri, United States, 63130|
|United States, Oregon|
|Oregon Health & Science University|
|Portland, Oregon, United States, 97239|
|Principal Investigator:||Desiree White, Ph.D.||Washington University School of Medicine|
|Principal Investigator:||Dorothy K. Grange, M.D.||Washington University School of Medicine|