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

Phenylketonuria Clinical Trial

— PKUDOS  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00778206
• Other study ID #: PKUDOS-01
• Secondary ID: PKUDOS Registry
• Status: Completed
• 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
• 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

Related Conditions & MeSH terms

• Hyperphenylalaninaemia
• Phenylketonuria
• Phenylketonurias

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