Status:
WITHDRAWN
Neuroimaging and Neurocognitive Assessment and Response to Sapropterin Dihydrochloride Treatment in Phenylketonuria
Lead Sponsor:
Children's National Research Institute
Collaborating Sponsors:
Georgetown University
Conditions:
PKU
Eligibility:
All Genders
18-40 years
Phase:
NA
Brief Summary
The investigators will use different types of brain imaging (MRI) in patients with Phenylketonuria (PKU) who are currently not on a strict diet to test the hypothesis that there is improvement in brai...
Detailed Description
We plan to enroll 36 subjects with PKU. They will all be counseled to follow the PKU diet. They will be randomized to receive Kuvan. We will plan to enroll 12 in the diet alone group and 24 in the Kuv...
Eligibility Criteria
Inclusion
- Patients with PKU identified on Newborn screening and with phe concentration \>12mg/dl on the newborn screen
- Baseline phe level at study enrollment \> 20 mg/dl (this is the level required for inclusion in the study, regardless of the level used to make diagnosis)
- Age range: 18-45 years
- Able to comply with neuroimaging without requiring sedation (typically requires IQ over 65). The IQ will be checked with the WASI (Weschler Adult scales of intelligence) before determining eligibility
- Able to undergo neuroimaging safely (i.e. without presence of ferromagnetic devices)
- Subject has ability to follow instructions in English
- Female of childbearing age who is sexually active agrees to urine pregnancy test
- Availability to come to Washington, DC to participate in this study
Exclusion
- Age range \<18 or \>45 years
- Inability to comply with neuroimaging without the use of sedation (low IQ or claustrophobic)
- Presence of ferromagnetic device(s) that preclude safe imaging including cardiac pacemaker, neural pacemaker, surgical clips in the brain or blood vessels, surgically implanted metal plates, screws or pins, cochlear implants or metal objects in their body
- Pregnant female or breastfeeding at screening or planning to become pregnant at any time during the study.
- Baseline phe \< 20 mg/dl
- Currently on Kuvan
- History of substance abuse
Key Trial Info
Start Date :
April 1 2011
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 1 2012
Estimated Enrollment :
Patients enrolled
Trial Details
Trial ID
NCT01412437
Start Date
April 1 2011
End Date
December 1 2012
Last Update
December 17 2014
Active Locations (1)
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1
Children's Research Institute
Washington D.C., District of Columbia, United States, 20010