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A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults | Clinical Trials | Clareo Health

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

A Non-Interventional Observational Study of Pegvaliase-Naïve Adults With Phenylketonuria (PKU): Concept Elicitation and Cognitive Interviews

NCT03505125•BioMarin Pharmaceutical

View on ClinicalTrials.gov

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.

Detailed Description

Clinical studies have suggested that treatment with pegvaliase results in lowering of blood Phenylalanine levels in adults with Phenylketonuria (PKU). Because these individuals often exhibit inattention and mood difficulties, and blood Phe concentration affects cognition and mood, life-long blood Phe control with pegvaliase may improve cognitive abilities and emotional and mental health in patients with PKU. In order to adequately quantify potential pegvaliase treatment impact on cognition and mood, it is necessary to employ clinical outcome assessments (COAs) that are fit for purpose and that comprehensively address concepts important, relevant, and meaningful to PKU patients. This is an observational study to identify symptom, function, and health-related quality of life (HRQoL) concepts of interest (COIs) to patients with PKU and to seek additional patient, observer, and expert input on the appropriateness of concepts of BioMarin's proposed COAs for use in future efficacy trials in PKU.

Eligibility

Age

18 - 70 years

Sex

ALL

Healthy Volunteers

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.

Locations (14)

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

Key Dates

Start Date

March 31, 2018

Primary Completion Date

July 31, 2019

Completion Date

July 31, 2019

Last Updated

January 14, 2020

Enrollment

ACTUAL participants

Conditions

Phenylketonuria (PKU)

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: January 14, 2020Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria

AAV Gene Therapy Study for Subjects with PKU

A Study to Evaluate the Long-Term Safety and Efficacy of JNT-517 in Participants With Phenylketonuria

Iron Supplements Effect on B-Phe Levels in PKU Patients During 3 Years Study

PANDA: PKU Amino Acid Evaluation

Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency