PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry (PKUDOS)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT00778206|
Recruitment Status : Recruiting
First Posted : October 23, 2008
Last Update Posted : June 8, 2017
|Condition or disease||Intervention/treatment|
|Phenylketonuria Hyperphenylalaninaemia||Drug: Kuvan|
|Study Type :||Observational|
|Estimated Enrollment :||3500 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.
Other Name: sapropterin dihydrochloride
- Observational Data Only [ Time Frame: Periodically ]Registry data will be analyzed and reported periodically, demographic and baseline characteristics will be summarized.
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
|Contact: Caroline Moore||(651) 523-0310||MEDINFO@bmrn.com|
|Contact: Raivo Kittus||(651) 523-0310||MEDINFO@bmrn.com|
Show 56 Study Locations
|Study Director:||Joshua Lilienstein, MD||BioMarin Pharmaceutical|