The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU)

This study is enrolling participants by invitation only.
Sponsor:
Collaborator:
BioMarin Pharmaceutical
Information provided by:
University of Missouri-Columbia
ClinicalTrials.gov Identifier:
NCT00964236
First received: August 21, 2009
Last updated: NA
Last verified: August 2009
History: No changes posted

August 21, 2009
August 21, 2009
August 2009
August 2010   (final data collection date for primary outcome measure)
functional magnetic resonance imaging of the brain [ Time Frame: baseline, 4 week, & 6 month follow-up ] [ Designated as safety issue: No ]
Same as current
No Changes Posted
Not Provided
Not Provided
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The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU)
The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU)

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.

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.

Observational
Observational Model: Case Control
Time Perspective: Prospective
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Not Provided
Non-Probability Sample

Primary care clinic for phenylketonuria. Columbia, Missouri, community for control.

Phenylketonuria
Drug: Sapropterin (Kuvan)
20mg/kg/day taken once daily or as otherwise prescribed by physician as standard care.
Other Name: Kuvan
  • Phenylketonuria
    Individuals with phenylketonuria (PKU) who are beginning treatment with Kuvan (sapropterin).
    Intervention: Drug: Sapropterin (Kuvan)
  • Control
    Healthy individuals without phenylketonuria (PKU).

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Enrolling by invitation
20
August 2011
August 2010   (final data collection date for primary outcome measure)

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.
Both
6 Years and older
Yes
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT00964236
PKU/Kuvan/Christ
No
University of Missouri (Shawn Christ, Ph.D., Assistant Professor of Psychological Sciences), University of Missouri-Columbia
University of Missouri-Columbia
BioMarin Pharmaceutical
Principal Investigator: Shawn E Christ, Ph.D. University of Missouri-Columbia
University of Missouri-Columbia
August 2009

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP