Status:
COMPLETED
PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
Lead Sponsor:
BioMarin Pharmaceutical
Conditions:
Phenylketonuria
Hyperphenylalaninaemia
Eligibility:
All Genders
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 Ku...
Eligibility Criteria
Inclusion
- PKUDOS Registry
- 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
- 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
Key Trial Info
Start Date :
September 1 2008
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
January 29 2021
Estimated Enrollment :
1887 Patients enrolled
Trial Details
Trial ID
NCT00778206
Start Date
September 1 2008
End Date
January 29 2021
Last Update
October 12 2022
Active Locations (45)
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1
Childrens Hospital of Los Angeles
Los Angeles, California, United States, 90027
2
LAC and USC Medical Center
Los Angeles, California, United States, 90033
3
UCLA
Los Angeles, California, United States, 90095
4
Children's Hospital of Orange County
Orange, California, United States, 92868