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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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19 October 2017 |
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Main ID: |
NCT00986973 |
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Date of registration:
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28/09/2009 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Fluorodeoxyglucose Positron Emission Tomography (FDG PET) Findings in Patients With Phenylketonuria Before and After KUVAN Therapy
PKU |
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Scientific title:
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A Pilot Study of FDG PET Findings in Patients With Phenylketonuria Before and After BH4 Supplementation |
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Date of first enrolment:
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March 2010 |
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Target sample size:
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6 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT00986973 |
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Study type:
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Interventional |
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Study design:
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Phase:
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N/A
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Countries of recruitment
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United States
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Contacts
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Name:
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Can Ficicioglu, MD, PhD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Children's Hospital of Philadelphia,University of Pennsylvania |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
1. Males or females over the age of 18 years
2. Subject must be able to give independent informed consent
3. Girls must have a negative urine pregnancy test and must use an acceptable method of
contraception, including abstinence, a barrier method (diaphragm or condom),
Depo-Provera, or an oral contraceptive, for the duration of the study.
4. Subject must have a confirmed diagnosis of PKU
5. Subjects with Phenylalanine (Phe) levels over 10 mg/dL
6. Subjects naïve to KUVAN therapy or has not received KUVAN in the 6 months before
screening
Exclusion Criteria:
1. Pregnancy
2. Cognitive deficits resulting from physical trauma (e.g. subject with history of severe
birth trauma).
3. Neurologic comorbidities including a history of a stroke or a seizure disorder.
4. Laboratory abnormalities that indicate clinically significant hepatic disease
Aspartate aminotransferase (AST)> 2.0 times the upper limit of normal, Alanine
transaminase (ALT) > 2.0 times the upper limit of normal, Prothrombin Time (PT) > 2.0
times the upper limit of normal, Partial Thromboplastin Time(PTT) > 2.0 times the
upper limit of normal
5. Subjects using medications such as steroids, insulin and glucagons that may interfere
with the results of PET scan.
6. Subjects using medications that inhibit folate metabolism such as methotrexate
7. Subjects using medications known to affect nitric oxide-mediated vasorelaxation.
8. Subjects using Levodopa
9. Treatment with KUVAN in the past 6 months before study entry.
10. Treatment with any investigational product in the last 90 days before study entry
Age minimum:
18 Years
Age maximum:
50 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Phenylketonuria
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Intervention(s)
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Drug: Sapropterin
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Primary Outcome(s)
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Plasma Phenylalanine Level (mg/dl)
[Time Frame: Measurements were obtained at the beginning and conclusion of each study period (baseline and 4 months)]
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Secondary Outcome(s)
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Delis-Kaplan Executive Function System Verbal Fluency Subtest (D-KEFS)
[Time Frame: Measures were obtained at the beginning and conclusion of each study period (baseline and 4 months)]
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Hopkins Verbal Learning Test (HVLT) Delayed Recall
[Time Frame: Measures were obtained at the beginning and conclusion of each study period (baseline and 4 months)]
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Symbol-Digit Modalities Test (SMTD)
[Time Frame: Measures were obtained at the beginning and conclusion of each study period (baseline and 4 months)]
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Paced Auditory Serial Addition Task (PASAT)
[Time Frame: Measures were obtained at the beginning and conclusion of each study period (baseline and 4 months)]
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Wechsler Adult Intelligence Scale (WAIS-IV)-Digit Span
[Time Frame: Measures were obtained at the beginning and conclusion of each study period (baseline and 4 months)]
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Hopkins Verbal Learning Test (HVLT) Total Recall
[Time Frame: Measures were obtained at the beginning and conclusion of each study period (baseline and 4 months)]
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Secondary ID(s)
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IRB 09-007154
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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