A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults

Completed

Conditions studied: Phenylketonuria (PKU)

In brief

The 165-901 study is designed to identify the appropriate tools for use in future interventional studies on the neurocognitive effects of pegvaliase on adults with PKU.

Key facts

Study ID

NCT03505125

Run by

BioMarin Pharmaceutical

People needed

63

Starts

2018-03-31

Expected to finish

2019-07-31

Last updated by the study team

2020-01-14

Who can join

Age: 18 and older, up to 70. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Patients eligible to participate in this study must meet all of the following criteria:
• ≥18 and ≤70 years of age at the time of consent;
• Clinically-confirmed diagnosis of Phenylketonuria (PKU);
• Blood Phe test completed within eight weeks prior to screening date or blood Phe test completed within eight weeks post screening date;
• Able to read, understand, and speak fluently the language(s) of their designated country sufficiently to participate in an interview and/or complete all assessments;
• Willing and able to participate in a telephone interview lasting approximately 90 minutes;
• Willing to be audio-recorded during the interview session;
• Willing and able to provide written informed consent indicating that they understand the purpose and procedures required for the study and are willing to participate.

You may not qualify if…

• Patients meeting any of the following criteria will not be included in the study:
• Currently pregnant;
• Current or previous exposure to pegvaliase treatment or participation in a pegvaliase clinical trial;
• Has a significant cognitive impairment, hearing difficulty, visual impairment, acute psychopathology, medical condition, or insufficient knowledge of the interview language that, in the opinion of the investigator or interviewer, would interfere with his or her ability to provide written consent and participate/complete an interview.

Where it is running

• University of Florida — Gainesville, Florida, United States
• Emory University Department of Human Genetics — Decatur, Georgia, United States
• Ann and Robert H Lurie Children's Hospital — Chicago, Illinois, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• Adult Metabolic Diseases Clinic, Vancouver General Hospital — Vancouver, British Columbia, Canada
• Necker Children's Hospital — Paris, Cedex 15, France
• Bretonneau Hospital, Internal Medicine Department — Tours, Cedex 9, France
• University Klinik Jena, Klinik für Neuropädiatrie — Jena, Thuringia, Germany
• Rheumatology Unit, Clinic for Inborn Errors of Metabolism — Leipzig, Germany
• Hospital of Padova — Padova, Italy
• Umberto I Polyclinic of Rome — Roma, Italy
• Hacettepe University — Ankara, Turkey (Türkiye)
• Ege University School of Medicine — Izmir, Turkey (Türkiye)
• National Hospital for Neurology and Neurosurgery — London, England, United Kingdom

Full record on ClinicalTrials.gov

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