Trial record 2 of 22 for:    PKU | Open Studies

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

This study is currently recruiting participants. (see Contacts and Locations)
Verified August 2014 by BioMarin Pharmaceutical
Sponsor:
Information provided by (Responsible Party):
BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:
NCT00778206
First received: October 21, 2008
Last updated: August 15, 2014
Last verified: August 2014
  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

  Show 52 Study Locations
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: August 15, 2014
Health Authority: United States: Institutional Review Board

Keywords provided by BioMarin Pharmaceutical:
Phenylketonuria
Hyperphenylalaninemia
PKU
Phenylalanine

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

ClinicalTrials.gov processed this record on September 18, 2014