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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT00778206
Other study ID # PKUDOS-01
Secondary ID PKUDOS Registry
Status Completed
Phase
First received
Last updated
Start date September 2008
Est. completion date January 29, 2021

Study information

Verified date September 2022
Source BioMarin Pharmaceutical
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

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


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 information / eligibility

Status Completed
Enrollment 1887
Est. completion date January 29, 2021
Est. primary completion date January 29, 2021
Accepts healthy volunteers No
Gender All
Age group N/A and older
Eligibility PKUDOS Registry Inclusion Criteria: - 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 Exclusion Criteria: - 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


Related Conditions & MeSH terms


Intervention

Drug:
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.

Locations

Country Name City State
United States Akron Children's Hospital Akron Ohio
United States Albany Medical Center Albany New York
United States Children's Hospital, University of Colorado School of Medicine Aurora Colorado
United States Johns Hopkins University School of Medicine Baltimore Maryland
United States Children's Hosptial of Boston Boston Massachusetts
United States University of North Carolina Chapel Hill North Carolina
United States Ann and Robert H. Lurie Children's Hospital of Chicago Chicago Illinois
United States University of Missouri Health Care Columbia Missouri
United States Nationwide Children's Hospital Columbus Ohio
United States Emory University Decatur Georgia
United States University of Florida Gainesville Florida
United States Greenwood Genetic Center Greenwood South Carolina
United States Penn State, Milton S. Hershey Medical Center Hershey Pennsylvania
United States Indiana University School of Medicine Indianapolis Indiana
United States University of Iowa Iowa City Iowa
United States University of Kentucky Lexington Kentucky
United States 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 Kosair Charities Pediatric Clinical Research Unit Louisville Kentucky
United States University of Miami, Miller School of Medicine, Department of Human Genetics Miami Florida
United States Children's Hospital of Wisconsin Milwaukee Wisconsin
United States University of Minnesota Minneapolis Minnesota
United States West Virginia Genetics Center, Department of Pediatrics Morgantown West Virginia
United States AHS Hospital Corp Morristown New Jersey
United States Tulane University Medical School New Orleans Louisiana
United States Children's Hospital of the King's Daughters Norfolk Virginia
United States University of Oklahoma Oklahoma City Oklahoma
United States University of Nebraska Omaha Nebraska
United States Children's Hospital of Orange County Orange California
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 Oregon Health and Science University Portland Oregon
United States University of Rochester Medical Center Rochester New York
United States St. Louis Children's Hospital Saint Louis Missouri
United States University of Utah Salt Lake City Utah
United States University of California, San Diego San Diego California
United States Sanford Children's Specialty Clinic Sioux Falls South Dakota
United States Stanford University, Pediatrics Stanford California
United States Tampa Children's Hospital, St. Joseph's Pediatric Endocrine Associates Tampa Florida
United States University of South Florida Tampa Florida
United States Westchester Medical Center Valhalla New York
United States Children's National Medical Center Washington District of Columbia
United States Wesley Pediatric Faculty Clinic Wichita Kansas

Sponsors (1)

Lead Sponsor Collaborator
BioMarin Pharmaceutical

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Observational Data Only Registry data including demographic and baseline characteristics 15 years
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