Actively Recruiting

Age: 10Years +
All Genders
NCT05128149

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

1

Research Sites

215 weeks

Total Duration

On this page

Sponsors

U

University Children's Hospital, Zurich

Lead Sponsor

U

UChildrenVienna

Collaborating Sponsor

AI-Summary

What this Trial Is About

Treatment of PKU implies for most patients that with strict adherence to dietary treatment they can achieve excellent neurocognitive outcome. Dietary treatment, though, is hard to comply to every day and with every single meal. Unsurprisingly, health-related quality of life (HrQol) is negatively affected if patients have to follow a dietary regime of this kind. Adherence to treatment in PKU is very variable. Factors of significant impact on adherence to treatment and well-being in chronic disease such as self-efficacy or parenting stress have not yet been widely investigated in PKU patients. The ideal treatment prescription (and guideline) recommends as much as necessary and as little as possible, based on the best evidence available. Patients should neither be deprived of treatment options nor be exposed to overtreatment. This study investigates adherence, metabolic control, HrQol in PKU patients treated by centres which follow different guidelines

CONDITIONS

Official Title

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

Who Can Participate

Age: 10Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Patients with PKU aged 10 years or older requiring dietary and/or pharmacological treatment according to local guidelines
  • Parents of patients aged 10 to 18 years requiring dietary and/or pharmacological treatment according to local guidelines
  • Ability and willingness to answer questionnaires and follow study procedures
  • Signed informed consent
Not Eligible

You will not qualify if you...

  • Inability to follow study procedures due to language problems or severely reduced health status
  • Pregnant patients or those planning pregnancy soon
  • Patients with hyperphenylalaninaemia, mild PKU forms, or other inborn errors with elevated Phe not requiring dietary or pharmacological treatment
  • No informed consent

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

1
2
3
+1

Trial Site Locations

Total: 1 location

1

University Childrens Hospital Zürich

Zurich, Switzerland, 8032

Actively Recruiting

Loading map...

Research Team

M

Martina Huemer, Prof

CONTACT

How is the study designed?

Study Type

OBSERVATIONAL

Masking

N/A

Allocation

N/A

Model

N/A

Primary Purpose

N/A

Number of Arms

0

Not the Right Trial for You?

Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.

Already have an account? Log in here

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