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: Case-Only
Time Perspective: Prospective
|Official Title:||PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry|
- 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.
|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)|
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.
Other Name: 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 54 Study Locations
|Study Director:||Amy Fowles||BioMarin Pharmaceutical|