The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU)
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Purpose
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.
| Condition | Intervention |
|---|---|
|
Phenylketonuria |
Drug: Sapropterin (Kuvan) |
| Study Type: | Observational |
| Study Design: | Observational Model: Case Control Time Perspective: Prospective |
| Official Title: | The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU) |
- functional magnetic resonance imaging of the brain [ Time Frame: baseline, 4 week, & 6 month follow-up ] [ Designated as safety issue: No ]
| Estimated Enrollment: | 20 |
| Study Start Date: | August 2009 |
| Estimated Study Completion Date: | August 2011 |
| Estimated Primary Completion Date: | August 2010 (Final data collection date for primary outcome measure) |
| Groups/Cohorts | Assigned Interventions |
|---|---|
|
Phenylketonuria
Individuals with phenylketonuria (PKU) who are beginning treatment with Kuvan (sapropterin).
|
Drug: Sapropterin (Kuvan)
20mg/kg/day taken once daily or as otherwise prescribed by physician as standard care.
Other Name: Kuvan
|
|
Control
Healthy individuals without phenylketonuria (PKU).
|
Detailed Description:
The proposed study is designed to evaluate the hypothesis that functional connectivity may represent an early marker for neurocognitive improvements related to Kuvan treatment. As a first step in the study, 20 patients with PKU who are ≥ 6 years of age will receive baseline functional connectivity magnetic resonance imaging (fcMRI) evaluations before being treated with Kuvan™. Response to Kuvan™ will be monitored for 4 weeks. At the end of 4 weeks, response to Kuvan™ will be reviewed and all 20 patients will receive follow-up fcMRI evaluations. Fifteen patients with a reduction of ≥ 20% in blood phenylalanine will continue in the study and receive a third fcMRI evaluation at the end of 6 months of treatment with Kuvan™. For comparison purposes and to control for possible practice effects in repeated testing, a matched control group of 20 healthy individuals without PKU will used. All 20 of the control subjects will receive baseline and 4-week fcMRI evaluations; fifteen will receive a 6-month evaluation.
Eligibility| Ages Eligible for Study: | 6 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | Yes |
| Sampling Method: | Non-Probability Sample |
Primary care clinic for phenylketonuria. Columbia, Missouri, community for control.
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.
Contacts and LocationsPlease refer to this study by its ClinicalTrials.gov identifier: NCT00964236
| United States, Missouri | |
| University of Missouri | |
| Columbia, Missouri, United States, 65211 | |
| Principal Investigator: | Shawn E Christ, Ph.D. | University of Missouri-Columbia |
More Information
Additional Information:
Publications:
| Responsible Party: | University of Missouri (Shawn Christ, Ph.D., Assistant Professor of Psychological Sciences), University of Missouri-Columbia |
| ClinicalTrials.gov Identifier: | NCT00964236 History of Changes |
| Other Study ID Numbers: | PKU/Kuvan/Christ |
| Study First Received: | August 21, 2009 |
| Last Updated: | August 21, 2009 |
| Health Authority: | United States: Institutional Review Board |
Keywords provided by University of Missouri-Columbia:
|
sapropterin Kuvan cognition brain |
Additional relevant MeSH terms:
|
Phenylketonurias Amino Acid Metabolism, Inborn Errors Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn |
Central Nervous System Diseases Genetic Diseases, Inborn Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases |
ClinicalTrials.gov processed this record on March 03, 2015