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

• Status: Completed
• Trial end date: 2021-01-29
• Target enrollment: 0
• Participant gender: All

Summary

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

• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: BioMarin Pharmaceutical

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