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Sponsors and Collaborators: |
Washington University School of Medicine BioMarin Pharmaceutical University of Missouri-Columbia Northwestern University Oregon Health and Science University |
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Information provided by: | Washington University School of Medicine |
ClinicalTrials.gov Identifier: | NCT00827762 |
The purpose of this study is to determine whether improvements in behavior occur in children with phenylketonuria (PKU) who are taking Kuvan.
Condition | Intervention |
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Phenylketonuria |
Drug: Kuvan |
Study Type: | Observational |
Study Design: | Case-Only, Prospective |
Official Title: | Behavioral Effects of Kuvan in Children With Mild Phenylketonuria |
Estimated Enrollment: | 20 |
Study Start Date: | January 2009 |
Estimated Study Completion Date: | January 2010 |
Estimated Primary Completion Date: | January 2010 (Final data collection date for primary outcome measure) |
Groups/Cohorts | Assigned Interventions |
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Phenylketonuria
Individuals with mild phenylketonuria/hyperphenylalanemia who are beginning treatment with Kuvan.
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Drug: Kuvan
20/mg/kg/day taken once daily or as otherwise prescribed by physician as standard care.
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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.
Ages Eligible for Study: | 6 Years to 18 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Sampling Method: | Non-Probability Sample |
Primary care clinic for phenylketonuria.
Inclusion Criteria:
Exclusion Criteria:
United States, Illinois | |
Northwestern University/Children's Memorial Hospital | |
Chicago, Illinois, United States, 60614 | |
United States, Missouri | |
Washington University | |
St. Louis, Missouri, United States, 63130 | |
University of Missouri | |
Columbia, Missouri, United States, 65211 | |
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 |
Responsible Party: | Washington University ( Desiree A. White, Ph.D., Associate Professor of Psychology ) |
Study ID Numbers: | MildPKU/Kuvan/White |
Study First Received: | January 21, 2009 |
Last Updated: | January 21, 2009 |
ClinicalTrials.gov Identifier: | NCT00827762 History of Changes |
Health Authority: | United States: Institutional Review Board |
phenylketonuria Kuvan sapropterin behavior |
cognition executive abilities attention |
Metabolism, Inborn Errors Metabolic Diseases Genetic Diseases, Inborn Inborn Amino Acid Metabolism Disorder Amino Acid Metabolism, Inborn Errors Central Nervous System Diseases |
Brain Diseases, Metabolic, Inborn Phenylketonurias Brain Diseases Metabolic Disorder Phenylketonuria Brain Diseases, Metabolic |
Metabolism, Inborn Errors Metabolic Diseases Genetic Diseases, Inborn Amino Acid Metabolism, Inborn Errors Nervous System Diseases |
Central Nervous System Diseases Brain Diseases, Metabolic, Inborn Phenylketonurias Brain Diseases Brain Diseases, Metabolic |