The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU)
1 other identifier
observational
20
1 country
1
Brief Summary
The objective of the study is to determine whether Kuvan™ (sapropterin) improves the strength of the functional connectivity between brain regions in individuals with PKU.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for all trials
Started Aug 2009
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
August 1, 2009
CompletedFirst Submitted
Initial submission to the registry
August 21, 2009
CompletedFirst Posted
Study publicly available on registry
August 24, 2009
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 1, 2010
CompletedStudy Completion
Last participant's last visit for all outcomes
August 1, 2011
CompletedJanuary 4, 2018
January 1, 2018
1 year
August 21, 2009
January 2, 2018
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
functional magnetic resonance imaging of the brain
baseline, 4 week, & 6 month follow-up
Study Arms (2)
Sapropterin
Individuals with phenylketonuria (PKU) who are beginning treatment with Kuvan (sapropterin).
Control
Healthy individuals without phenylketonuria (PKU).
Interventions
Sapropterin (Kuvan) 20mg/kg/day taken once daily or as otherwise prescribed by physician as standard care.
Eligibility Criteria
Primary care clinic for phenylketonuria. Columbia, Missouri, community for control.
You may qualify if:
- Willing and able to provide informed consent or assent.
- Willing and able to comply with study procedures.
- Greater than or equal to 6 years of age.
- For phenylketonuria, intention of physician to prescribe sapropterin.
- For phenylketonuria, phenylalanine level greater than or equal to 300μmol/L.
- For phenylketonuria, negative pregnancy test if of childbearing potential.
- For phenylketonuria, willing to use contraception if sexually active.
You may not qualify if:
- 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.
- Perceived to be unreliable or unavailable for study.
- Use of L-Dopa, methotrexate, or other drugs that inhibit folate metabolism.
- For phenylketonuria, known hypersensitivity to sapropterin or excipients.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- University of Missouri-Columbialead
- BioMarin Pharmaceuticalcollaborator
Study Sites (1)
University of Missouri
Columbia, Missouri, 65211, United States
Related Publications (2)
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.
PMID: 17083296BACKGROUNDAraujo GC, Christ SE, Steiner RD, Grange DK, Nardos B, McKinstry RC, White DA. Response monitoring in children with phenylketonuria. Neuropsychology. 2009 Jan;23(1):130-4. doi: 10.1037/a0013488.
PMID: 19210041BACKGROUND
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Shawn E Christ, Ph.D.
University of Missouri-Columbia
Study Design
- Study Type
- observational
- Observational Model
- CASE CONTROL
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Prof Assoc, Psychological Sciences
Study Record Dates
First Submitted
August 21, 2009
First Posted
August 24, 2009
Study Start
August 1, 2009
Primary Completion
August 1, 2010
Study Completion
August 1, 2011
Last Updated
January 4, 2018
Record last verified: 2018-01