The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU)
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ClinicalTrials.gov Identifier: NCT00964236 |
Recruitment Status
:
Completed
First Posted
: August 24, 2009
Last Update Posted
: January 4, 2018
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Condition or disease | Intervention/treatment |
---|---|
Phenylketonuria | Drug: Sapropterin |
Study Type : | Observational |
Actual Enrollment : | 20 participants |
Observational Model: | Case-Control |
Time Perspective: | Prospective |
Official Title: | The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU) |
Study Start Date : | August 2009 |
Actual Primary Completion Date : | August 2010 |
Actual Study Completion Date : | August 2011 |

Group/Cohort | Intervention/treatment |
---|---|
Sapropterin
Individuals with phenylketonuria (PKU) who are beginning treatment with Kuvan (sapropterin).
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Drug: Sapropterin
Sapropterin (Kuvan) 20mg/kg/day taken once daily or as otherwise prescribed by physician as standard care.
Other Name: Kuvan
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Control
Healthy individuals without phenylketonuria (PKU).
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- functional magnetic resonance imaging of the brain [ Time Frame: baseline, 4 week, & 6 month follow-up ]

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Ages Eligible for Study: | 6 Years and older (Child, Adult, Senior) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | Yes |
Sampling Method: | Non-Probability Sample |
Inclusion Criteria:
- 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.
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.
- 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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00964236
United States, Missouri | |
University of Missouri | |
Columbia, Missouri, United States, 65211 |
Principal Investigator: | Shawn E Christ, Ph.D. | University of Missouri-Columbia |
Additional Information:
Publications:
Responsible Party: | Shawn Christ, Prof Assoc, Psychological Sciences, University of Missouri-Columbia |
ClinicalTrials.gov Identifier: | NCT00964236 History of Changes |
Other Study ID Numbers: |
PKU/Kuvan/Christ |
First Posted: | August 24, 2009 Key Record Dates |
Last Update Posted: | January 4, 2018 |
Last Verified: | January 2018 |
Keywords provided by Shawn Christ, University of Missouri-Columbia:
sapropterin Kuvan cognition brain |
Additional relevant MeSH terms:
Phenylketonurias Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases Amino Acid Metabolism, Inborn Errors Metabolism, Inborn Errors |
Genetic Diseases, Inborn Metabolic Diseases Verapamil Anti-Arrhythmia Agents Calcium Channel Blockers Membrane Transport Modulators Molecular Mechanisms of Pharmacological Action Vasodilator Agents |