Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00778206
Recruitment Status : Completed
First Posted : October 23, 2008
Last Update Posted : May 7, 2021
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical

Tracking Information
First Submitted Date October 21, 2008
First Posted Date October 23, 2008
Last Update Posted Date May 7, 2021
Study Start Date September 2008
Actual Primary Completion Date January 29, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: May 14, 2020)
Observational Data Only [ Time Frame: 15 years ]
Registry data including demographic and baseline characteristics
Original Primary Outcome Measures Not Provided
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
Official Title PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
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.
Study Type Observational
Study Design Observational Model: Case-Only
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Diagnosis of Phenylketonuria with hyperphenylalaninemia
Condition
  • Phenylketonuria
  • Hyperphenylalaninaemia
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.
Other Name: sapropterin dihydrochloride
Study Groups/Cohorts
  • 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.
    Intervention: Drug: Kuvan
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Completed
Actual Enrollment
 (submitted: May 6, 2021)
1922
Original Estimated Enrollment
 (submitted: October 22, 2008)
3500
Actual Study Completion Date January 29, 2021
Actual Primary Completion Date January 29, 2021   (Final data collection date for primary outcome measure)
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
Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries United States
Removed Location Countries Puerto Rico
 
Administrative Information
NCT Number NCT00778206
Other Study ID Numbers PKUDOS-01
PKUDOS Registry ( Registry Identifier: PKUDOS )
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party BioMarin Pharmaceutical
Study Sponsor BioMarin Pharmaceutical
Collaborators Not Provided
Investigators
Study Director: Kristin Lindstrom, MD BioMarin Pharmaceutical
PRS Account BioMarin Pharmaceutical
Verification Date April 2021