A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults
NCT ID: NCT03505125
Last Updated: 2020-01-14
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
63 participants
OBSERVATIONAL
2018-03-31
2019-07-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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OTHER
RETROSPECTIVE
Study Groups
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PKU Patients
Adults with PKU will be interviewed about the symptoms and impacts of PKU.
No interventions assigned to this group
Observers
Close friends and family members of adults with PKU will be interviewed about the behaviors they have observed in adults with PKU
No interventions assigned to this group
Clinical Experts
Experienced, practicing clinicians currently treating adults with PKU will be interviewed about the symptoms and impacts of PKU on their patients.
No interventions assigned to this group
Eligibility Criteria
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Inclusion Criteria
1. ≥18 and ≤70 years of age at the time of consent;
2. Clinically-confirmed diagnosis of Phenylketonuria (PKU);
3. Blood Phe test completed within eight weeks prior to screening date or blood Phe test completed within eight weeks post screening date;
4. 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;
5. Willing and able to participate in a telephone interview lasting approximately 90 minutes;
6. Willing to be audio-recorded during the interview session;
7. 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.
Exclusion Criteria
1. Currently pregnant;
2. Current or previous exposure to pegvaliase treatment or participation in a pegvaliase clinical trial;
3. 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.
18 Years
70 Years
ALL
No
Sponsors
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BioMarin Pharmaceutical
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director, MD
Role: STUDY_DIRECTOR
BioMarin Pharmaceutical
Locations
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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
Padua, , 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
Countries
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Other Identifiers
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165-901
Identifier Type: -
Identifier Source: org_study_id
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