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Approved For Marketing No phase listed Expanded access

Sapropterin Expanded Access Program

ClinicalTrials.gov ID: NCT00484991

Public ClinicalTrials.gov record NCT00484991. 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.

ClinicalTrials.gov public records Last synced Sep 2, 2026, 4:47 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

The Purpose of this study is to provide patients with hyperphenylalaninemia (HPA) due to Phenylketonuria (PKU) access to sapropterin dihydrochloride and to collect more information about the safety of the drug in an expanded access program (EAP) until commercial product is available.

Study identification

NCT ID
NCT00484991
Recruitment status
Approved For Marketing
Study type
Expanded access
Phase
Not listed
Lead sponsor
BioMarin Pharmaceutical
Industry
Enrollment
Not listed

Conditions and interventions

Interventions

Drug

Eligibility (public fields only)

Age range
9 Years and older
Sex
All
Healthy volunteers
Not listed

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Not listed
Primary completion
Not listed
Completion
Not listed
Last update posted
Apr 14, 2008

United States locations

U.S. sites
36
U.S. states
21
U.S. cities
36
Facility City State ZIP Site status
Not listed Los Angeles California
Not listed Orange California
Not listed San Diego California
Not listed Stanford California
Not listed Denver Colorado
Not listed Gainseville Florida
Not listed Tampa Florida
Not listed Chicago Illinois
Not listed Indianapolis Indiana
Not listed Louisville Kentucky
Not listed New Orleans Louisiana
Not listed Bangor Maine
Not listed Boston Massachusetts
Not listed Minneapolis Minnesota
Not listed Columbia Missouri
Not listed Kansas City Missouri
Not listed St Louis Missouri
Not listed Newark New Jersey
Not listed Manhasset New York
Not listed Rochester New York
Not listed Stony Brook New York
Not listed Valhalla New York
Not listed Akron Ohio
Not listed Columbus Ohio
Not listed Hershey Pennsylvania
Not listed Philadelphia Pennsylvania
Not listed Pittsburgh Pennsylvania
Not listed Florence South Carolina
Not listed Sioux Falls South Dakota
Not listed Galveston Texas
Not listed Houston Texas
Not listed Salt Lake City Utah
Not listed Charlottesville Virginia
Not listed Norfolk Virginia
Not listed Madison Wisconsin
Not listed Milwakee 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 NCT00484991, 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 14, 2008 · Synced Sep 2, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT00484991 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →