PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry

Completed

Conditions studied: Phenylketonuria, Hyperphenylalaninaemia

In brief

The objective of this study is to evaluate the safety of long-term treatment with Kuvan.

Key facts

Study ID

NCT00778206

Run by

BioMarin Pharmaceutical

People needed

1887

Starts

2008-09-01

Expected to finish

2021-01-29

Last updated by the study team

2022-10-12

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

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
• 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

Where it is running

• 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

Full record on ClinicalTrials.gov

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