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

• Register: ClinicalTrials.gov
• Last refreshed on: 24 October 2022
• Main ID: NCT00778206
• Date of registration: 21/10/2008
• Prospective Registration: No
• Primary sponsor: BioMarin Pharmaceutical
• Public title: PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry PKUDOS
• Scientific title: PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
• Date of first enrolment: September 2008
• Target sample size: 1887
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00778206
• Study type: Observational

Countries of recruitment

Puerto Rico, United States

Contacts

• Name: Kristin Lindstrom, MD
• Affiliation: BioMarin Pharmaceutical

Key inclusion & exclusion 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



Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Phenylketonuria

Hyperphenylalaninaemia

Intervention(s)

Drug: Kuvan

Primary Outcome(s)

Observational Data Only [Time Frame: 15 years]

Secondary ID(s)

PKUDOS-01

PKUDOS Registry

Source(s) of Monetary Support

Please refer to primary and secondary sponsors