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

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Trial Status Completed

Trial Identifier

NCT03505125

Condition

Phenylketonuria, Phenylketonuria (PKU)

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial Summary

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.

Eligibility Criteria

Birth Sex

All

Age

18 Years to 70 Years

Healthy Volunteers

No

Study Type:

Observational

Number of Participants:

63

Study Started:

2018-03-31

Study Updated:

2020-01-14

Trial Locations

  • 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

  • Ege University School of Medicine

    İzmir, , Turkey

  • National Hospital for Neurology and Neurosurgery

    London, England, United Kingdom

Inclusion Criteria

Patients eligible to participate in this study must meet all of the following 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

Patients meeting any of the following criteria will not be included in the study:

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

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