CI

At a glance

ClinicalIndex Comparison Record
N/ACompleted· 6 enrolled
Drug / intervention
Sapropterindrug
Likely dose
Sapropterin 20 mg/kgfrom record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT00986973
NCT00986973N/ACompleted
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A Pilot Study of FDG PET Findings in Patients With Phenylketonuria Before and After BH4 Supplementation

Children's Hospital of Philadelphia·interventional·Posted Sep 30, 2009·Updated Jun 8, 2015

In Brief

A clinical study evaluating Sapropterin for Phenylketonuria. Completed, enrolled 6 participants across 1 site.

Detailed 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.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsPhenylketonuria
CountriesUnited States
Collaborators--

Timeline

N/ACompletedFinished
20102011201220132014201520162017201820192020202120222023202420252026
First PostedSep 30, 2009
Enrollment StartMar 1, 2010
Primary CompletionJul 1, 2011
Study CompletionSep 1, 2011
TodayJul 2, 2026
Enrollment to primary: 1.3 yearsPosted 16.8 years ago

Interventions

Sapropterindrug

All subjects will receive 20 mg/kg/day Sapropterin (KUVAN) for four months. Subjects will be examined with fluorodeoxyglucose positron emission tomography (FDG-PET) brain imaging, physical and neurological exam, blood tests for phenylalanine (Phe) and tyrosine levels, and neuropsychological testing before and 4 months after KUVAN therapy. Subjects Phe and tyrosine levels will be monitored weekly during the study and subjects will keep 3-day diet records to allow for calculation of Phe intake.