PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
NCT ID: NCT00778206
Last Updated: 2022-10-12
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
1887 participants
OBSERVATIONAL
2008-09-30
2021-01-29
Brief Summary
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Detailed Description
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Conditions
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Study Design
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CASE_ONLY
PROSPECTIVE
Study Groups
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1. PKUDOS Registry
Patients with a confirmed diagnosis of Phenylketonuria (PKU) with hyperphenylalaninemia who have either received Kuvan therapy, or currently receive Kuvan therapy, or intend to begin receiving Kuvan therapy within 90 days of entering the registry.
No interventions assigned to this group
2. PKU MOMS Subregistry
Patients with PKU who are pregnant at enrollment in the registry or who become pregnant while participating in the registry.
Kuvan
-Recommended post-partum Assessments include: collection of baby's plasma at Birth, 1 Month and 6 months, and collection of mother's plasma and expressed breast milk sample at 1 Month.
Interventions
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Kuvan
-Recommended post-partum Assessments include: collection of baby's plasma at Birth, 1 Month and 6 months, and collection of mother's plasma and expressed breast milk sample at 1 Month.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Patient has previously received Kuvan
* Patient is currently receiving Kuvan
* Patient intends to receive Kuvan therapy within 90 days of enrollment into the registry
* The Patient is being followed at a PKUDOS participating center
* Willing and able to provide written authorization or, if under the age of 18 years, provide written assent (if required) and written patient authorization by a parent or legal guardian
* Willing to provide personal health information
* Willing to enroll in (or are already enrolled in) PKUDOS
* Agree to follow the standard of care for pregnant women with PKU in the United States (NIH, 200, NIH Consensus Statement)
* Agree to be followed by a hospital or PKU clinic offering the standard of care for maternal PKU
* Are within 10 weeks of their last menstrual period
Exclusion Criteria
* Patients not previously treated with Kuvan and patients that are unwilling to begin Kuvan therapy within 90 days of entry into the registry
PKU MOMS Subregistry
* Patients who have not adhered to the standard of care for pregnant women with PKU in the United States are not eligible to participate in PKU MOMS
ALL
No
Sponsors
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BioMarin Pharmaceutical
INDUSTRY
Responsible Party
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Principal Investigators
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Kristin Lindstrom, MD
Role: STUDY_DIRECTOR
BioMarin Pharmaceutical
Locations
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Childrens Hospital of Los Angeles
Los Angeles, California, United States
LAC and USC Medical Center
Los Angeles, California, United States
UCLA
Los Angeles, California, United States
Children's Hospital of Orange County
Orange, California, United States
University of California, San Diego
San Diego, California, United States
Stanford University, Pediatrics
Stanford, California, United States
Children's Hospital, University of Colorado School of Medicine
Aurora, Colorado, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
University of Florida
Gainesville, Florida, United States
University of Miami, Miller School of Medicine, Department of Human Genetics
Miami, Florida, United States
University of South Florida
Tampa, Florida, United States
Tampa Children's Hospital, St. Joseph's Pediatric Endocrine Associates
Tampa, Florida, United States
Emory University
Decatur, Georgia, United States
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Indiana University School of Medicine
Indianapolis, Indiana, United States
University of Iowa
Iowa City, Iowa, United States
Wesley Pediatric Faculty Clinic
Wichita, Kansas, United States
University of Kentucky
Lexington, Kentucky, United States
Kosair Charities Pediatric Clinical Research Unit
Louisville, Kentucky, United States
Tulane University Medical School
New Orleans, Louisiana, United States
Johns Hopkins University School of Medicine
Baltimore, Maryland, United States
Children's Hosptial of Boston
Boston, Massachusetts, United States
University of Minnesota
Minneapolis, Minnesota, United States
University of Missouri Health Care
Columbia, Missouri, United States
St. Louis Children's Hospital
St Louis, Missouri, United States
University of Nebraska
Omaha, Nebraska, United States
AHS Hospital Corp
Morristown, New Jersey, United States
Albany Medical Center
Albany, New York, United States
University of Rochester Medical Center
Rochester, New York, United States
Westchester Medical Center
Valhalla, New York, United States
University of North Carolina
Chapel Hill, North Carolina, United States
Akron Children's Hospital
Akron, Ohio, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
University of Oklahoma
Oklahoma City, Oklahoma, United States
Oregon Health and Science University
Portland, Oregon, United States
Penn State, Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
St. Christophers Hospital for Children
Philadelphia, Pennsylvania, United States
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
Greenwood Genetic Center
Greenwood, South Carolina, United States
Sanford Children's Specialty Clinic
Sioux Falls, South Dakota, United States
University of Utah
Salt Lake City, Utah, United States
Children's Hospital of the King's Daughters
Norfolk, Virginia, United States
West Virginia Genetics Center, Department of Pediatrics
Morgantown, West Virginia, United States
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, United States
Countries
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Other Identifiers
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PKUDOS Registry
Identifier Type: REGISTRY
Identifier Source: secondary_id
PKUDOS-01
Identifier Type: -
Identifier Source: org_study_id
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