Clinical trial NCT00778206: PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
Official title: PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Phenylketonuria
Hyperphenylalaninaemia
Keywords: Phenylketonuria
Hyperphenylalaninemia
PKU
Phenylalanine
Lead sponsor: BioMarin Pharmaceutical
Sponsor class: INDUSTRY
First posted: 2008-10-23
Enrollment: 1887
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Diagnosis of Phenylketonuria with hyperphenylalaninemia

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.

Eligibility criteria: 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

Primary outcomes:
measure: Observational Data Only
timeFrame: 15 years
Secondary outcomes:
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Childrens Hospital of Los Angeles
state: California
city: Los Angeles
country: United States
facility: LAC and USC Medical Center
state: California
city: Los Angeles
country: United States
facility: UCLA
state: California
city: Orange
country: United States
facility: Children's Hospital of Orange County
state: California
city: San Diego
country: United States
facility: University of California, San Diego
state: California
city: Stanford
country: United States
facility: Stanford University, Pediatrics
state: California
city: Aurora
country: United States
facility: Children's Hospital, University of Colorado School of Medicine
state: Colorado
city: Washington
country: United States
facility: Children's National Medical Center
state: District of Columbia
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Miami
country: United States
facility: University of Miami, Miller School of Medicine, Department of Human Genetics
state: Florida
city: Tampa
country: United States
facility: University of South Florida
state: Florida
city: Tampa
country: United States
facility: Tampa Children's Hospital, St. Joseph's Pediatric Endocrine Associates
state: Florida
city: Decatur
country: United States
facility: Emory University
state: Georgia
city: Chicago
country: United States
facility: Ann and Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Indianapolis
country: United States
facility: Indiana University School of Medicine
state: Indiana
city: Iowa City
country: United States
facility: University of Iowa
state: Iowa
city: Wichita
country: United States
facility: Wesley Pediatric Faculty Clinic
state: Kansas
city: Lexington
country: United States
facility: University of Kentucky
state: Kentucky
city: Louisville
country: United States
facility: Kosair Charities Pediatric Clinical Research Unit
state: Kentucky
city: New Orleans
country: United States
facility: Tulane University Medical School
state: Louisiana
city: Baltimore
country: United States
facility: Johns Hopkins University School of Medicine
state: Maryland
city: Boston
country: United States
facility: Children's Hosptial of Boston
state: Massachusetts
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Columbia
country: United States
facility: University of Missouri Health Care
state: Missouri
city: Saint Louis
country: United States
facility: St. Louis Children's Hospital
state: Missouri
city: Omaha
country: United States
facility: University of Nebraska
state: Nebraska
city: Morristown
country: United States
facility: AHS Hospital Corp
state: New Jersey
city: Albany
country: United States
facility: Albany Medical Center
state: New York
city: Rochester
country: United States
facility: University of Rochester Medical Center
state: New York
city: Valhalla
country: United States
facility: Westchester Medical Center
state: New York
city: Chapel Hill
country: United States
facility: University of North Carolina
state: North Carolina
city: Akron
country: United States
facility: Akron Children's Hospital
state: Ohio
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Oklahoma City
country: United States
facility: University of Oklahoma
state: Oklahoma
city: Portland
country: United States
facility: Oregon Health and Science University
state: Oregon
city: Hershey
country: United States
facility: Penn State, Milton S. Hershey Medical Center
state: Pennsylvania
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Philadelphia
country: United States
facility: St. Christophers Hospital for Children
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Children's Hospital of Pittsburgh
state: Pennsylvania
city: Greenwood
country: United States
facility: Greenwood Genetic Center
state: South Carolina
city: Sioux Falls
country: United States
facility: Sanford Children's Specialty Clinic
state: South Dakota
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
city: Norfolk
country: United States
facility: Children's Hospital of the King's Daughters
state: Virginia
city: Morgantown
country: United States
facility: West Virginia Genetics Center, Department of Pediatrics
state: West Virginia
city: Milwaukee
country: United States
facility: Children's Hospital of Wisconsin
state: Wisconsin
Study officials:
affiliation: BioMarin Pharmaceutical
name: Kristin Lindstrom, MD
role: STUDY_DIRECTOR
References: