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Sapropterin in Individuals With Phenylketonuria
NCT00730080 · View on ClinicalTrials.gov ↗
Study Summary
Investigators at Washington University will examine the effects of sapropterin (Kuvan) on brain and cognition in individuals with phenylketonuria (PKU) using neuropsychological and neuroimaging procedures. Sapropterin is a medication developed by BioMarin Pharmaceutical Inc. that is approved by the FDA for treatment of patients with PKU to reduce phenylalanine (Phe) levels. Patients beginning treatment with sapropterin as standard clinical care will be enrolled in the study. As a first step, patients with PKU will receive baseline neuropsychological and neuroimaging evaluations 1 day prior to beginning treatment with sapropterin. Screening for response to sapropterin will occur over 4 weeks. At the end of 4 weeks, response to sapropterin will be reviewed. Patients with a reduction of ≥ 20% in blood Phe (i.e., responders) will receive follow-up neuropsychological and neuroimaging evaluations after 6 months of treatment with sapropterin. Patients (both responders and nonresponders) will receive long-term follow-up neuropsychological and neuroimaging evaluations 3 to 5 years after initial enrollment in the study. The focus of neuropsychological testing will be executive abilities, as these abilities are particularly susceptible to disruption in individuals with PKU. We hypothesize that improvements in these abilities will occur following treatment with sapropterin. For neuroimaging assessments, structural magnetic resonance imaging (MRI) will permit evaluation of changes in the structure and volume of the gray and white matter of the brain, whereas diffusion tensor imaging (DTI) will permit evaluation of microstructural white matter integrity.
Conditions Studied
Interventions
- DRUG Sapropterin (Kuvan)
Study Locations (1)
Missouri
- Washington University — St Louis
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 45 participants |
| Start Date | 2008-07 |
| Est. Completion | 2018-05 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00730080
The ClinicalTrials.gov registry entry for NCT00730080 describes a study currently listed as completed. It is categorized as an unspecified phase, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 45 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Washington University School of Medicine, which has 1,036 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Phenylketonuria appearing as the primary indexed condition, and to 1 intervention — of which Sapropterin (Kuvan) is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT00730080 reports 1 study location spanning 1 distinct geographic area — top geographies include Missouri. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT00730080 about?
NCT00730080 is a clinical study titled "Sapropterin in Individuals With Phenylketonuria". Investigators at Washington University will examine the effects of sapropterin (Kuvan) on brain and cognition in individuals with phenylketonuria (PKU) using neuropsychological and neuroimaging procedures. Sapropterin is a medication developed by BioMarin Pharmaceutical Inc. that is approved by the ...
What is the current status of trial NCT00730080?
This trial is currently completed. The enrollment target is 45 participants. The study started on 2008-07. Estimated completion is 2018-05.
What conditions does trial NCT00730080 study?
This clinical trial studies the following conditions: Phenylketonuria. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT00730080?
The interventions under investigation include: Sapropterin (Kuvan) (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT00730080?
This trial is sponsored by Washington University School of Medicine, which has 1,036 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT00730080 being conducted?
This trial has 1 study location across Missouri. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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