PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
PKUDOS
2 other identifiers
observational
1,887
1 country
45
Brief Summary
The objective of this study is to evaluate the safety of long-term treatment with Kuvan.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Sep 2008
Longer than P75 for all trials
45 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
September 1, 2008
CompletedFirst Submitted
Initial submission to the registry
October 21, 2008
CompletedFirst Posted
Study publicly available on registry
October 23, 2008
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 29, 2021
CompletedStudy Completion
Last participant's last visit for all outcomes
January 29, 2021
CompletedOctober 12, 2022
September 1, 2022
12.4 years
October 21, 2008
October 11, 2022
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Observational Data Only
Registry data including demographic and baseline characteristics
15 years
Study Arms (2)
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.
2. PKU MOMS Subregistry
Patients with PKU who are pregnant at enrollment in the registry or who become pregnant while participating in the registry.
Interventions
-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.
Eligibility Criteria
Diagnosis of Phenylketonuria with hyperphenylalaninemia
You may qualify if:
- 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
You may not qualify if:
- 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
- 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
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (45)
Childrens Hospital of Los Angeles
Los Angeles, California, 90027, United States
LAC and USC Medical Center
Los Angeles, California, 90033, United States
UCLA
Los Angeles, California, 90095, United States
Children's Hospital of Orange County
Orange, California, 92868, United States
University of California, San Diego
San Diego, California, 92093, United States
Stanford University, Pediatrics
Stanford, California, 94305, United States
Children's Hospital, University of Colorado School of Medicine
Aurora, Colorado, 80045, United States
Children's National Medical Center
Washington D.C., District of Columbia, 20010, United States
University of Florida
Gainesville, Florida, 32611, United States
University of Miami, Miller School of Medicine, Department of Human Genetics
Miami, Florida, 33136, United States
University of South Florida
Tampa, Florida, 33606, United States
Tampa Children's Hospital, St. Joseph's Pediatric Endocrine Associates
Tampa, Florida, 33607, United States
Emory University
Decatur, Georgia, 30033, United States
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
Indiana University School of Medicine
Indianapolis, Indiana, 46202, United States
University of Iowa
Iowa City, Iowa, 52242, United States
Wesley Pediatric Faculty Clinic
Wichita, Kansas, 67208, United States
University of Kentucky
Lexington, Kentucky, 40536, United States
Kosair Charities Pediatric Clinical Research Unit
Louisville, Kentucky, 40202, United States
Tulane University Medical School
New Orleans, Louisiana, 70112, United States
Johns Hopkins University School of Medicine
Baltimore, Maryland, 21287, United States
Children's Hosptial of Boston
Boston, Massachusetts, 02115, United States
University of Minnesota
Minneapolis, Minnesota, 55455, United States
University of Missouri Health Care
Columbia, Missouri, 65212, United States
St. Louis Children's Hospital
St Louis, Missouri, 63110, United States
University of Nebraska
Omaha, Nebraska, 68182, United States
AHS Hospital Corp
Morristown, New Jersey, 07960, United States
Albany Medical Center
Albany, New York, 12208, United States
University of Rochester Medical Center
Rochester, New York, 14642, United States
Westchester Medical Center
Valhalla, New York, 10595, United States
University of North Carolina
Chapel Hill, North Carolina, 27599, United States
Akron Children's Hospital
Akron, Ohio, 44308, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
University of Oklahoma
Oklahoma City, Oklahoma, 73104, United States
Oregon Health and Science University
Portland, Oregon, 97239, United States
Penn State, Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
St. Christophers Hospital for Children
Philadelphia, Pennsylvania, 19134, United States
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
Greenwood Genetic Center
Greenwood, South Carolina, 29646, United States
Sanford Children's Specialty Clinic
Sioux Falls, South Dakota, 57117, United States
University of Utah
Salt Lake City, Utah, 84132, United States
Children's Hospital of the King's Daughters
Norfolk, Virginia, 23507, United States
West Virginia Genetics Center, Department of Pediatrics
Morgantown, West Virginia, 26506, United States
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Kristin Lindstrom, MD
BioMarin Pharmaceutical
Study Design
- Study Type
- observational
- Observational Model
- CASE ONLY
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 21, 2008
First Posted
October 23, 2008
Study Start
September 1, 2008
Primary Completion
January 29, 2021
Study Completion
January 29, 2021
Last Updated
October 12, 2022
Record last verified: 2022-09