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NCT00730080 · ClinicalTrials.gov registry record
Sapropterin in Individuals With Phenylketonuria
A clinical trial of Phenylketonuria, sponsored by Washington University School of Medicine.
- Completed
- Registry status
- 45
- Enrollment target
- 1
- Study location
NCT00730080 is a study of Phenylketonuria that has completed, run by Washington University School of Medicine. The registered enrollment target is 45 participants, below the 95-participant average among 12 other Phenylketonuria trials with a reported enrollment target (53% lower). The trial reports 1 study location across 1 state.
The verdict
NCT00730080, a study of Phenylketonuria, has completed, sponsored by Washington University School of Medicine.
- COMPLETED
- Registry status
- 45 participants
- Enrollment target
- 1
- Study location
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, categorized as an unspecified phase. The registered enrollment target is 45 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 95-participant average among 12 other Phenylketonuria trials with a reported enrollment target (53% lower). The listed sponsor is Washington University School of Medicine, which has 1,502 total studies on file at ClinicalTrials.gov.
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.
NCT00730080 reports 1 study location spanning 1 distinct geographic area - top geographies include Missouri.
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.
What interventions are being tested in trial NCT00730080?
The interventions under investigation include: Sapropterin (Kuvan) (DRUG).
Who is sponsoring clinical trial NCT00730080?
This trial is sponsored by Washington University School of Medicine, which has 1,502 total clinical trials registered on ClinicalTrials.gov.
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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Related
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