PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
The objective of this study is to evaluate the safety of long-term treatment with Kuvan.
|Study Design:||Observational Model: Cohort
Time Perspective: Prospective
|Official Title:||PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry|
|Study Start Date:||September 2008|
|Estimated Primary Completion Date:||September 2023 (Final data collection date for primary outcome measure)|
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: Sapropterin Dihydrochloride
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00778206
|Contact: Amy Fowlesfirstname.lastname@example.org|
Show 51 Study Locations
|Study Director:||Amy Fowles||BioMarin Pharmaceutical|