Behavioral Effects of Kuvan in Children With Mild Phenylketonuria
NCT ID: NCT00827762
Last Updated: 2013-12-05
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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TERMINATED
2 participants
OBSERVATIONAL
2009-01-31
2010-01-31
Brief Summary
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Detailed Description
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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.
Conditions
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Keywords
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Study Design
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CASE_ONLY
PROSPECTIVE
Study Groups
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Phenylketonuria
Individuals with mild phenylketonuria/hyperphenylalanemia who are beginning treatment with Kuvan.
Kuvan
20/mg/kg/day taken once daily or as otherwise prescribed by physician as standard care.
Interventions
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Kuvan
20/mg/kg/day taken once daily or as otherwise prescribed by physician as standard care.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Willing and able to comply with study procedures.
* Between 6 and 18 years of age, inclusive.
* Intention of physician to prescribe Kuvan.
* Phenylalanine levels between 360μmol/L and 600μmol/L, inclusive, when untreated with dietary restrictions.
* Negative pregnancy test if of childbearing potential.
* Willing to use contraception if sexually active.
Exclusion Criteria
* Pregnant, breastfeeding, or planning to become pregnant during study.
* Use of investigational product less than 30 days prior to or during study.
* Concurrent condition that could interfere with participation or safety.
* Any condition creating high risk of poor compliance with study.
* History of major medical disorder unrelated to phenylketonuria.
* Perceived to be unreliable or unavailable for study.
* Use of L-Dopa, methotrexate, or other drugs that inhibit folate metabolism.
* Known hypersensitivity to sapropterin or excipients.
6 Years
18 Years
ALL
No
Sponsors
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BioMarin Pharmaceutical
INDUSTRY
University of Missouri-Columbia
OTHER
Northwestern University
OTHER
Oregon Health and Science University
OTHER
Washington University School of Medicine
OTHER
Responsible Party
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Principal Investigators
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Desiree White, Ph.D.
Role: PRINCIPAL_INVESTIGATOR
Washington University School of Medicine
Dorothy K. Grange, M.D.
Role: PRINCIPAL_INVESTIGATOR
Washington University School of Medicine
Locations
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Northwestern University/Children's Memorial Hospital
Chicago, Illinois, United States
University of Missouri
Columbia, Missouri, United States
Washington University
St Louis, Missouri, United States
Oregon Health & Science University
Portland, Oregon, United States
Countries
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References
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Christ SE, Steiner RD, Grange DK, Abrams RA, White DA. Inhibitory control in children with phenylketonuria. Dev Neuropsychol. 2006;30(3):845-64. doi: 10.1207/s15326942dn3003_5.
White DA, Nortz MJ, Mandernach T, Huntington K, Steiner RD. Age-related working memory impairments in children with prefrontal dysfunction associated with phenylketonuria. J Int Neuropsychol Soc. 2002 Jan;8(1):1-11.
White DA, Nortz MJ, Mandernach T, Huntington K, Steiner RD. Deficits in memory strategy use related to prefrontal dysfunction during early development: evidence from children with phenylketonuria. Neuropsychology. 2001 Apr;15(2):221-9. doi: 10.1037//0894-4105.15.2.221.
Other Identifiers
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MildPKU/Kuvan/White
Identifier Type: -
Identifier Source: org_study_id