What is this study trying to find out about phenylketonuria (PKU)?

This study is investigating how adherence to treatment and different guidelines affect metabolic control and quality of life in patients with PKU. It aims to understand how these factors influence well-being and family burden. The research is sponsored by the University Children's Hospital, Zurich.

Who may be eligible for this phenylketonuria clinical trial?

Participants must be at least 10 years old with PKU requiring dietary or pharmacological treatment according to local guidelines. Parents of patients aged 10 to 18 may also join. People unable to follow study procedures or who are pregnant may not qualify. Please review eligibility criteria for more details.

Is there any cost to participate in this phenylketonuria clinical trial?

Study-related care, including all study procedures, is provided at no cost. Personal travel and other expenses may vary and are not guaranteed to be covered. Participants should plan accordingly.

Actively Recruiting

Age: 10Years +

All Genders

ID05128149

Metabolic Control and Patient Well-being in Phenylketonuria: do Guidelines Make a Difference?

Led by University Children's Hospital, Zurich · Updated on 2025-04-13

200

Participants Needed

Research Sites

39 weeks

Total Duration

AI-Summary

What this Trial Is About

This research aims to understand how different treatment guidelines affect metabolic control and quality of life in patients with phenylketonuria (PKU), a condition requiring careful dietary or pharmacological management. It focuses on how patients adhere to their treatment plans and how this impacts their well-being and family burden, especially since strict diets can be challenging and affect daily life. The study is observational and sponsored by the University Children's Hospital, Zurich. Participants include patients with PKU aged 10 years and older who follow dietary and/or pharmacological treatments based on local guidelines. Parents of patients aged 10 to 18 years may also participate. The study involves no intervention or change to current treatment but collects data from centers following varying guidelines to compare outcomes. During the study, researchers will review phenylalanine (Phe) levels from the past 24 months and assess health-related quality of life, self-efficacy, and family burden through questionnaires. The study requires participants to be able and willing to complete these questionnaires and follow study procedures. The total duration and specific visits are not detailed, but assessments will take about one hour each. Safety monitoring and long-term follow-up are not specified.