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

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Trial Status Completed

Trial Identifier

NCT00778206

Condition

Phenylketonuria, Hyperphenylalaninaemia

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial Summary

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

Eligibility Criteria

Birth Sex

All

Age

Any

Healthy Volunteers

No

Drug/Treatment:

Kuvan

Study Type:

Observational

Number of Participants:

1887

Study Started:

2008-09

Study Updated:

2022-10-12

Trial Locations

  • 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, 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

    Saint Louis, Missouri, United States

  • University of Nebraska

    Omaha, Nebraska, United States

  • AHS Hospital Corp

    Morristown, New Jersey, United States

  • Albany Medical Center

    Albany, New York, United States

  • University of Rochester Medical Center

    Rochester, New York, United States

  • Westchester Medical Center

    Valhalla, New York, United States

  • University of North Carolina

    Chapel Hill, North Carolina, United States

  • Akron Children's Hospital

    Akron, Ohio, United States

  • Nationwide Children's Hospital

    Columbus, Ohio, United States

  • University of Oklahoma

    Oklahoma City, Oklahoma, United States

  • Oregon Health and Science University

    Portland, Oregon, United States

  • Penn State, Milton S. Hershey Medical Center

    Hershey, Pennsylvania, 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

  • Greenwood Genetic Center

    Greenwood, South Carolina, United States

  • Sanford Children's Specialty Clinic

    Sioux Falls, South Dakota, United States

  • University of Utah

    Salt Lake City, Utah, United States

  • Children's Hospital of the King's Daughters

    Norfolk, Virginia, United States

  • West Virginia Genetics Center, Department of Pediatrics

    Morgantown, West Virginia, United States

  • Children's Hospital of Wisconsin

    Milwaukee, Wisconsin, United States

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 Criteri

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
  • 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 MOM

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