Trial record 1 of 1 for:    nct 00778206
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PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry

This study is currently recruiting participants. (see Contacts and Locations)
Verified January 2015 by BioMarin Pharmaceutical
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:
NCT00778206
First received: October 21, 2008
Last updated: January 20, 2015
Last verified: January 2015
  Purpose

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


Condition Intervention
Phenylketonuria
Hyperphenylalaninaemia
Drug: Kuvan

Study Type: Observational
Study Design: Observational Model: Case-Only
Time Perspective: Prospective
Official Title: PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry

Resource links provided by NLM:


Further study details as provided by BioMarin Pharmaceutical:

Primary Outcome Measures:
  • Observational Data Only [ Time Frame: Periodically ] [ Designated as safety issue: No ]
    Registry data will be analyzed and reported periodically, demographic and baseline characteristics will be summarized.


Estimated Enrollment: 3500
Study Start Date: September 2008
Estimated Study Completion Date: September 2023
Estimated Primary Completion Date: September 2023 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
1. PKUDOS Registry
Patients with a confirmed diagnosis of Phenylketonuria (PKU) with hyperphenylalaninemia who have either received Kuvan therapy, or currently receive Kuvan therapy, or intend to begin receiving Kuvan therapy within 90 days of entering the registry.
2. PKU MOMS Subregistry
Patients with PKU who are pregnant at enrollment in the registry or who become pregnant while participating in the registry.
Drug: Kuvan
  • After the baby is born, follow-up assessments will be requested for six months. If Kuvan is taken by the mother during and/or after pregnancy, blood samples will be requested from the mother and the baby.
  • If the mother is lactating and taking Kuvan, breast milk samples will be requested.
Other Name: sapropterin dihydrochloride

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 intends to begin receiving Kuvan therapy within 90 days of entering the registry.

  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Diagnosis of Phenylketonuria with hyperphenylalaninemia

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00778206

Contacts
Contact: Amy Fowles 484-374-3254 afowles@bmrn.com

  Hide Study Locations
Locations
United States, California
Childrens Hospital of Los Angeles Recruiting
Los Angeles, California, United States, 90027
LAC and USC Medical Center Recruiting
Los Angeles, California, United States, 90033
UCLA Recruiting
Los Angeles, California, United States, 90095
Contact: Naghmeh Dorrani    301-825-8084      
Sutter Medical Center, Institute for Medical Research Recruiting
Sacramento, California, United States, 95816
Contact: Ashley Dang-Chu    916-733-4482      
University of California, San Diego School of Medicine Recruiting
San Diego, California, United States, 92103
Contact: Kim Victor    619-471-9585      
Stanford University, Pediatrics Recruiting
Stanford, California, United States, 94305
United States, Colorado
Children's Hospital, University of Colorado School of Medicine Recruiting
Aurora, Colorado, United States, 80045
Contact: Shannon Scrivner    303-724-2353      
United States, Connecticut
University of Connecticut Health Partners Recruiting
West Hartford, Connecticut, United States, 06119
United States, District of Columbia
Children's National Medical Center Recruiting
Washington, District of Columbia, United States, 20010
Contact: Kara Simpson    202-476-6216      
United States, Florida
University of Florida Recruiting
Gainesville, Florida, United States, 32610
Contact: Christel Gross    352-273-7763      
Nemours Children's Clinic Recruiting
Jacksonville, Florida, United States, 32207
Contact: Carrie Crain    407-650-7156      
Universtiy of Miami, Miller School of Medicine, Department of Human Genetics Recruiting
Miami, Florida, United States, 33136
Contact: Stephanie Sacharow    305-243-7985      
Nemours Childrens Clinic - Orlando Recruiting
Orlando, Florida, United States, 32806
Tampa Children's Hospital, St. Joseph's Pediatric Endocrine Associates Recruiting
Tampa, Florida, United States, 33607
Contact: Elizabeth Pelaez    813-870-4432      
United States, Georgia
Emory University Recruiting
Decatur, Georgia, United States, 30033
Contact: Alana Aisthorpe    404-778-8575      
United States, Illinois
Ann and Robert H. Lurie Children's Hospital of Chicago Recruiting
Chicago, Illinois, United States, 60611
Contact: Sheela Shrestha    312-227-6777      
United States, Indiana
Indiana University School of Medicine Recruiting
Indianapolis, Indiana, United States, 46202
Contact: Susan Romie    317-278-6650      
United States, Iowa
University of Iowa Recruiting
Iowa City, Iowa, United States, 52242
Contact: Judy Miller    319-356-2674      
United States, Kansas
Wesley Pediatric Faculty Clinic Recruiting
Wichita, Kansas, United States, 67208
Contact: Julie Wellner    316-962-7386      
United States, Kentucky
University of Kentucky Recruiting
Lexington, Kentucky, United States, 40536
University of Louisville Recruiting
Louisville, Kentucky, United States, 40202
Contact: Kelly Jackson    502-852-5681      
United States, Louisiana
Hayward Genetics, Tulane Medical School Recruiting
New Orleans, Louisiana, United States, 70112
Contact: Amy Cunningham    504-988-2989      
United States, Massachusetts
Children's Hosptial of Boston Recruiting
Boston, Massachusetts, United States, 02115
Contact: Ann Wessel    617-355-6515      
United States, Minnesota
University of Minnesota Recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Ashley Wiesenburger    612-301-1371      
United States, Missouri
University of Missouri Health Care Recruiting
Columbia, Missouri, United States, 65212
Contact: Jessica Myers    573-882-5891      
Children's Mercy Hospital Genetics Recruiting
Kansas City, Missouri, United States, 64123
Contact: Connie Haney    816-701-1395      
Washington University School of Medicine Recruiting
St. Louis, Missouri, United States, 63110
Contact: Liz Toolan    314-747-0015      
United States, Montana
Montana Metabolic Disease Center Withdrawn
Helena, Montana, United States, 59601
United States, Nebraska
University of Nebraska Medical Center Recruiting
Omaha, Nebraska, United States, 68198
Contact: Machelle Dawson    402-559-5698      
United States, New York
Albany Medical Center Recruiting
Albany, New York, United States, 12208
Contact: Julie Lundberg    518-262-5027      
North Shore University Hospital Terminated
Manhasset, New York, United States, 11030
Mt. Sinai Medical Center Terminated
New York, New York, United States, 10028
University of Rochester Medical Center Recruiting
Rochester, New York, United States, 14642
Contact: Theresa Harte    585-275-5857      
Westchester Medical Center Recruiting
Valhalla, New York, United States, 10595
Contact: Shideh Mofidi    914-304-5316      
United States, North Carolina
University of North Carolina Recruiting
Chapel Hill, North Carolina, United States, 27599
Contact: Julie McClure    919-843-9946      
United States, Ohio
Akron Children's Hospital Recruiting
Akron, Ohio, United States, 44308
Contact: Amanda Archbold    330-543-8792      
Cincinnati Children's Hospital Medical Center Withdrawn
Cincinnati, Ohio, United States, 45229
Nationwide Children's Hospital Recruiting
Columbus, Ohio, United States, 43205
Contact: Karen Carter    614-722-2661      
United States, Oklahoma
Warren Clinic Genetics Recruiting
Tulsa, Oklahoma, United States, 74136
United States, Oregon
Oregon Health and Science University Recruiting
Portland, Oregon, United States, 97239
Contact: Abigail Hata    503-418-1061      
United States, Pennsylvania
Penn State, Milton S. Hershey Medical Center Recruiting
Hershey, Pennsylvania, United States, 17033
Contact: Amyee McMonagle    717-531-1278      
Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Jessica Burfield    215-590-3219      
St. Christophers Hospital for Children Recruiting
Philadelphia, Pennsylvania, United States, 19134
Contact: Mitzie Grant    215-427-5081      
Children's Hospital of Pittsburgh Recruiting
Pittsburgh, Pennsylvania, United States, 15224
Contact: Nancy Perrott-Taylor    412-692-3150      
United States, Rhode Island
Rhode Island Hospital, Hasbro's Children's Hospital Recruiting
Providence, Rhode Island, United States, 02903
Contact: Susan Kortick    401-444-5794      
United States, South Dakota
Sanford Children's Specialty Clinic Recruiting
Sioux Falls, South Dakota, United States, 57117
Contact: Alycia Brantz    605-328-1389      
United States, Tennessee
University of Tennessee Genetic Center Terminated
Knoxville, Tennessee, United States, 37920
United States, Texas
University of Texas Withdrawn
Houston, Texas, United States, 77030
United States, Utah
Dixie Regional Medical Center Recruiting
Saint Geroge, Utah, United States, 84770
University of Utah Recruiting
Salt Lake City, Utah, United States, 84132
Contact: Carrie Bailey    801-587-3605      
United States, Virginia
University of Virginia Hospital Withdrawn
Charlottesville, Virginia, United States, 22908
Children's Hospital of the King's Daughters Recruiting
Norfolk, Virginia, United States, 23507
Contact: Eileen Coffman    757-668-9723      
United States, West Virginia
West Virginia Genetics Center, Department of Pediatrics Recruiting
Morgantown, West Virginia, United States, 26506
United States, Wisconsin
Children's Hospital of Wisconsin Recruiting
Milwaukee, Wisconsin, United States, 53226
Contact: Ashley Boerst    414-266-8674      
Sponsors and Collaborators
BioMarin Pharmaceutical
Investigators
Study Director: Amy Fowles BioMarin Pharmaceutical
  More Information

No publications provided

Responsible Party: BioMarin Pharmaceutical
ClinicalTrials.gov Identifier: NCT00778206     History of Changes
Other Study ID Numbers: PKUDOS-01, PKUDOS Registry
Study First Received: October 21, 2008
Last Updated: January 20, 2015
Health Authority: United States: Institutional Review Board

Keywords provided by BioMarin Pharmaceutical:
Phenylketonuria
Hyperphenylalaninemia
PKU
Phenylalanine

Additional relevant MeSH terms:
Phenylketonurias
Amino Acid Metabolism, Inborn Errors
Brain Diseases
Brain Diseases, Metabolic
Brain Diseases, Metabolic, Inborn
Central Nervous System Diseases
Genetic Diseases, Inborn
Metabolic Diseases
Metabolism, Inborn Errors
Nervous System Diseases

ClinicalTrials.gov processed this record on March 26, 2015