PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry (PKUDOS)
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|ClinicalTrials.gov Identifier: NCT00778206|
Recruitment Status : Active, not recruiting
First Posted : October 23, 2008
Last Update Posted : May 15, 2020
|Condition or disease||Intervention/treatment|
|Phenylketonuria Hyperphenylalaninaemia||Drug: Kuvan|
|Study Type :||Observational|
|Actual Enrollment :||1993 participants|
|Official Title:||PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry|
|Study Start Date :||September 2008|
|Estimated Primary Completion Date :||September 2023|
|Estimated Study Completion Date :||September 2023|
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.
-Recommended post-partum Assessments include: collection of baby's plasma at Birth, 1 Month and 6 months, and collection of mother's plasma and expressed breast milk sample at 1 Month.
Other Name: sapropterin dihydrochloride
- Observational Data Only [ Time Frame: 15 years ]Registry data including demographic and baseline characteristics
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00778206
|Study Director:||Joshua Lilienstein, MD||BioMarin Pharmaceutical|