Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria
Public ClinicalTrials.gov record NCT00104260. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 2, Multicenter, Open-Label Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
The primary objective is to evaluate the degree and frequency of response to Phenoptin™ (sapropterin dihydrochloride), as demonstrated by a reduction in blood phenylalanine (Phe) level among subjects with phenylketonuria (PKU) who have elevated Phe levels. A secondary objective of this study is to evaluate the safety of Phenoptin™ treatment in this subject population, and identify individuals in this subject population who respond to Phenoptin™ treatment with a reduction in blood Phe level.
Study identification
- NCT ID
- NCT00104260
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 700 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 8 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Dec 2004 (Type not available)
- Primary completion
- Not listed
- Completion
- Nov 2005 (Actual)
- Last update posted
- Apr 9, 2007
2004 – 2005
United States locations
- U.S. sites
- 13
- U.S. states
- 12
- U.S. cities
- 13
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Not listed | Los Angeles | California | — | — |
| Not listed | Oakland | California | — | — |
| Not listed | New Haven | Connecticut | — | — |
| Not listed | Chicago | Illinois | — | — |
| Not listed | Boston | Massachusetts | — | — |
| Not listed | Minneapolis | Minnesota | — | — |
| Not listed | St Louis | Missouri | — | — |
| Not listed | New York | New York | — | — |
| Not listed | Portland | Oregon | — | — |
| Not listed | Pittsburgh | Pennsylvania | — | — |
| Not listed | Dallas | Texas | — | — |
| Not listed | Salt Lake City | Utah | — | — |
| Not listed | Madison | Wisconsin | — | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT00104260, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Apr 9, 2007 · Synced Oct 30, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT00104260 live on ClinicalTrials.gov.