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

This study is currently recruiting participants. (see Contacts and Locations)
Verified July 2013 by BioMarin Pharmaceutical
Sponsor:
Information provided by:
BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:
NCT00778206
First received: October 21, 2008
Last updated: July 22, 2013
Last verified: July 2013

October 21, 2008
July 22, 2013
September 2008
September 2023   (final data collection date for primary outcome measure)
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Complete list of historical versions of study NCT00778206 on ClinicalTrials.gov Archive Site
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PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry

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

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.

Observational
Observational Model: Cohort
Time Perspective: Prospective
Not Provided
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Non-Probability Sample

Diagnosis of Phenylketonuria with hyperphenylalaninemia

  • Phenylketonuria
  • Hyperphenylalaninaemia
Drug: Sapropterin Dihydrochloride
  • 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.
  • 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: Sapropterin Dihydrochloride
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
3500
Not Provided
September 2023   (final data collection date for primary outcome measure)

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
Both
Not Provided
No
Contact: Amy Fowles 484-374-3254 afowles@bmrn.com
United States
 
NCT00778206
PKUDOS-01, PKUDOS Registry
No
BioMarin Pharmaceutical Inc.
BioMarin Pharmaceutical
Not Provided
Study Director: Amy Fowles BioMarin Pharmaceutical
BioMarin Pharmaceutical
July 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP