N/A
Completed N=6
Fluorodeoxyglucose Positron Emission Tomography (FDG PET) Findings in Patients With Phenylketonuria Before and After KUVAN Therapy
Source: ClinicalTrials.gov NCT00986973 ↗Enrolled (actual)
6
Serious AEs
0.0%
Results posted
May 2015
Primary outcomePrimary: Plasma Phenylalanine Level (mg/dl) — 18.0; 17.2 mg/dl — p=>0.05
Summary
The aim of this pilot study is to determine if there are any changes in brain glucose metabolism in the gray matter of patients with Phenylketonuria (PKU) and whether administration of Sapropterin (KUVAN) therapy can improve such deficits.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Plasma Phenylalanine Level (mg/dl) |
18.0; 17.2 | >0.05 |
| SECONDARY Hopkins Verbal Learning Test (HVLT) Total Recall |
51; 53 | 0.82 |
| SECONDARY Hopkins Verbal Learning Test (HVLT) Delayed Recall |
47; 49 | 0.46 |
| SECONDARY Paced Auditory Serial Addition Task (PASAT) |
0.4700; 0.94 | 0.071 |
| SECONDARY Symbol-Digit Modalities Test (SMTD) |
63; 79 | 0.25 |
| SECONDARY Wechsler Adult Intelligence Scale (WAIS-IV)-Digit Span |
33; 37 | 0.050 |
| SECONDARY Delis-Kaplan Executive Function System Verbal Fluency Subtest (D-KEFS) |
10; 11 | 0.34 |
Eligibility Criteria
Inclusion Criteria
- Males or females over the age of 18 years
- Subject must be able to give independent informed consent
- 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.
- Subject must have a confirmed diagnosis of PKU
- Subjects with Phenylalanine (Phe) levels over 10 mg/dL
- Subjects naïve to KUVAN therapy or has not received KUVAN in the 6 months before screening
Exclusion Criteria
- Pregnancy
- Cognitive deficits resulting from physical trauma (e.g. subject with history of severe birth trauma).
- Neurologic comorbidities including a history of a stroke or a seizure disorder.
- 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
- Subjects using medications such as steroids, insulin and glucagons that may interfere with the results of PET scan.
- Subjects using medications that inhibit folate metabolism such as methotrexate
- Subjects using medications known to affect nitric oxide-mediated vasorelaxation.
- Subjects using Levodopa
- Treatment with KUVAN in the past 6 months before study entry.
- Treatment with any investigational product in the last 90 days before study entry
Data sourced from ClinicalTrials.gov (NCT00986973). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.