Sapropterin Expanded Access Program
1 other identifier
expanded_access
N/A
1 country
36
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
36 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
June 8, 2007
CompletedFirst Posted
Study publicly available on registry
June 12, 2007
CompletedApril 15, 2008
April 1, 2008
June 8, 2007
April 11, 2008
Conditions
Keywords
Interventions
20mg/kg/day orally, physicians may adjust the dose within a range of 5-20mg/kg/day as warranted by their clinical judgment.
Eligibility Criteria
You may qualify if:
- Patient has hyperphenylalaninemia due to PKU, a rare and serious disease
- Patient is not participating in a sapropterin dihydrochloride clinical study
- Patient is older than 8 years of age
- Patient is willing and able to provide written informed consent or, in the case of under the age of 18, provide written assent (if required) and written informed consent by a parent or legal guardian
- If female and of child bearing potential, the patient has a negative urine pregnancy test within 24 hours prior to enrollment (females of child-bearing potential only) and will be using adequate contraceptive methods to avoid pregnancy while participating in the program
- Patient is willing and able to comply with program procedures
- Patient lives in the United States
You may not qualify if:
- Patient is perceived to be unreliable or unwilling to comply with program participation or, if under the age of 18, have parents or legal guardians who are perceived to be unreliable or unwilling to comply with program participation
- Patient has a concurrent disease or condition that would interfere with program participation or safety
- Patient is 8 years old or younger
- Patients is eligible for enrolling in PKU-010
- Patient is participating in an ongoing study with sapropterin dihydrochloride
- Patient is pregnant, breast feeding or considering pregnancy
- Patient is taking levodopa
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (36)
Unknown Facility
Los Angeles, California, United States
Unknown Facility
Orange, California, United States
Unknown Facility
San Diego, California, United States
Unknown Facility
Stanford, California, United States
Unknown Facility
Denver, Colorado, United States
Unknown Facility
Gainseville, Florida, United States
Unknown Facility
Tampa, Florida, United States
Unknown Facility
Chicago, Illinois, United States
Unknown Facility
Indianapolis, Indiana, United States
Unknown Facility
Louisville, Kentucky, United States
Unknown Facility
New Orleans, Louisiana, United States
Unknown Facility
Bangor, Maine, United States
Unknown Facility
Boston, Massachusetts, United States
Unknown Facility
Minneapolis, Minnesota, United States
Unknown Facility
Columbia, Missouri, United States
Unknown Facility
Kansas City, Missouri, United States
Unknown Facility
St Louis, Missouri, United States
Unknown Facility
Newark, New Jersey, United States
Unknown Facility
Manhasset, New York, United States
Unknown Facility
Rochester, New York, United States
Unknown Facility
Stony Brook, New York, United States
Unknown Facility
Valhalla, New York, United States
Unknown Facility
Akron, Ohio, United States
Unknown Facility
Columbus, Ohio, United States
Unknown Facility
Hershey, Pennsylvania, United States
Unknown Facility
Philadelphia, Pennsylvania, United States
Unknown Facility
Pittsburgh, Pennsylvania, United States
Unknown Facility
Florence, South Carolina, United States
Unknown Facility
Sioux Falls, South Dakota, United States
Unknown Facility
Galveston, Texas, United States
Unknown Facility
Houston, Texas, United States
Unknown Facility
Salt Lake City, Utah, United States
Unknown Facility
Charlottesville, Virginia, United States
Unknown Facility
Norfolk, Virginia, United States
Unknown Facility
Madison, Wisconsin, United States
Unknown Facility
Milwakee, Wisconsin, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- expanded access
- Sponsor Type
- INDUSTRY
Study Record Dates
First Submitted
June 8, 2007
First Posted
June 12, 2007
Last Updated
April 15, 2008
Record last verified: 2008-04