PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
- Registration Number
- NCT00778206
- Lead Sponsor
- BioMarin Pharmaceutical
- Brief Summary
The objective of this study is to evaluate the safety of long-term treatment with Kuvan.
- Detailed Description
The PKUDOS program is a voluntary, multicenter, strictly observational program for patients with PKU who have either received Kuvan therapy, or currently receive Kuvan, or intend to begin receiving Kuvan therapy within 90 days of entering the registry.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 1887
- Patient has confirmed diagnosis of PKU with hyperphenylalaninemia documented by a Phenylalanine level of greater than or equal to 360 umol/L (6 mg/dL)
- 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
- Patients are not eligible to participate in PKUDOS if they are participating in a BioMarin-sponsored clinical study of Kuvan
- 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
Inclusion Criteria:
- 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 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
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description 2. PKU MOMS Subregistry Kuvan Patients with PKU who are pregnant at enrollment in the registry or who become pregnant while participating in the registry.
- Primary Outcome Measures
Name Time Method Observational Data Only 15 years Registry data including demographic and baseline characteristics
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (45)
LAC and USC Medical Center
🇺🇸Los Angeles, California, United States
UCLA
🇺🇸Los Angeles, California, United States
Ann and Robert H. Lurie Children's Hospital of Chicago
🇺🇸Chicago, Illinois, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Children's Hospital of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
University of Iowa
🇺🇸Iowa City, Iowa, United States
University of Kentucky
🇺🇸Lexington, Kentucky, United States
University of Missouri Health Care
🇺🇸Columbia, Missouri, United States
West Virginia Genetics Center, Department of Pediatrics
🇺🇸Morgantown, West Virginia, United States
University of California, San Diego
🇺🇸San Diego, California, United States
University of Miami, Miller School of Medicine, Department of Human Genetics
🇺🇸Miami, Florida, United States
University of Minnesota
🇺🇸Minneapolis, Minnesota, United States
Children's Hospital of Orange County
🇺🇸Orange, California, United States
University of Rochester Medical Center
🇺🇸Rochester, New York, United States
Indiana University School of Medicine
🇺🇸Indianapolis, Indiana, United States
University of Nebraska
🇺🇸Omaha, Nebraska, United States
Oregon Health and Science University
🇺🇸Portland, Oregon, United States
University of Utah
🇺🇸Salt Lake City, Utah, United States
Children's Hospital of Wisconsin
🇺🇸Milwaukee, Wisconsin, United States
Children's Hospital, University of Colorado School of Medicine
🇺🇸Aurora, Colorado, United States
Tampa Children's Hospital, St. Joseph's Pediatric Endocrine Associates
🇺🇸Tampa, Florida, United States
Childrens Hospital of Los Angeles
🇺🇸Los Angeles, California, United States
Stanford University, Pediatrics
🇺🇸Stanford, California, United States
Children's National Medical Center
🇺🇸Washington, District of Columbia, United States
Emory University
🇺🇸Decatur, Georgia, United States
Wesley Pediatric Faculty Clinic
🇺🇸Wichita, Kansas, United States
Children's Hosptial of Boston
🇺🇸Boston, Massachusetts, United States
AHS Hospital Corp
🇺🇸Morristown, New Jersey, United States
Albany Medical Center
🇺🇸Albany, New York, United States
St. Louis Children's Hospital
🇺🇸Saint Louis, Missouri, United States
Akron Children's Hospital
🇺🇸Akron, Ohio, United States
Westchester Medical Center
🇺🇸Valhalla, New York, United States
St. Christophers Hospital for Children
🇺🇸Philadelphia, Pennsylvania, United States
Penn State, Milton S. Hershey Medical Center
🇺🇸Hershey, Pennsylvania, United States
Sanford Children's Specialty Clinic
🇺🇸Sioux Falls, South Dakota, United States
Greenwood Genetic Center
🇺🇸Greenwood, South Carolina, United States
Children's Hospital of the King's Daughters
🇺🇸Norfolk, Virginia, United States
Johns Hopkins University School of Medicine
🇺🇸Baltimore, Maryland, United States
University of Oklahoma
🇺🇸Oklahoma City, Oklahoma, United States
University of South Florida
🇺🇸Tampa, Florida, United States
Kosair Charities Pediatric Clinical Research Unit
🇺🇸Louisville, Kentucky, United States
University of North Carolina
🇺🇸Chapel Hill, North Carolina, United States
University of Florida
🇺🇸Gainesville, Florida, United States
Tulane University Medical School
🇺🇸New Orleans, Louisiana, United States