Investigating the impact of dietary guidelines on well-being in patients with Phenylketonuria

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational study examines how adherence to dietary guidelines affects metabolic control and health-related quality of life (HrQol) in patients with Phenylketonuria (PKU). It aims to understand the variability in treatment adherence and the factors influencing well-being, such as self-efficacy and parenting stress. The study will involve patients aged 10 years and older who require dietary or pharmacological treatment according to local guidelines, and it will gather data through questionnaires. By comparing outcomes across different treatment centers, the study seeks to identify best practices in managing PKU.

Why it matters

Who can join: the full rules

Inclusion Criteria:- Patients with PKU from age 10 years requiring dietary and / or pharmacological treatment according to locally applied guidelines

* Parents of patients with PKU aged 10 to 18 years requiring dietary and / or pharmacological treatment according to locally applied guidelines
* Ability and willingness to answer the questionnaires and follow the study procedures
* Informed consent as documented by signature

Exclusion criteria for patients and parents :

* Inability to follow the procedures of the study, e.g. due to language problems or severely reduced health status
* Pregnant patients or patients who have disclosed to their physician that they are planning a pregnancy in the near future
* Hyperphenylalaninaemia or mild forms of PKU or other inborn errors associated with elevated Phe but not requiring dietary and / or pharmacological treatment according to locally applied guidelines
* No inform

Where it's running

• University Childrens Hospital Zürich
  Zurich, Switzerland Recruiting

Study contacts

• Study coordinator: Martina Huemer, Prof
• Email: martina.huemer@kispi.uzh.ch
• Phone: +41 44 2660

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Walking program for children with fatty liver and phenylketonuria

  This study is testing if a daily online walking program can help children with phenylketonuria and fatty liver feel better and improve their health.

  Recruiting Cairo University · Dokki, Giza Governorate · NCT06718842

• Evaluating NGGT002 for treating adult Phenylketonuria

  This study is testing a new gene therapy called NGGT002 to see if it can safely help adults with classic Phenylketonuria lower their blood phenylalanine levels.

  Recruiting at 2 sites Phase 1/2 NGGT (Suzhou) Biotechnology Co., Ltd. · Bengbu, Anhui and 1 other locations · NCT06687733

• New psychological tools and anxiety intervention for adolescents with PKU

  This study is testing a new online program to help teenagers with PKU manage anxiety and depression better over 10 weeks.

  Recruiting University of Missouri-Columbia · Columbia, Missouri · NCT06560736

• Evaluating Large Neutral Amino Acids for Treating Phenylketonuria

  This study is testing if Large Neutral Amino Acids can be a better treatment for adults with Phenylketonuria than the usual diet that limits phenylalanine.

  Recruiting Copenhagen University Hospital, Rigshospitalet · Copenhagen and 1 other locations · NCT06337864

• Gene therapy for adults with classic Phenylketonuria

  This study is testing a new gene therapy called NGGT002 to see if it can help adults with classic Phenylketonuria feel better and manage their condition.

  Recruiting at 5 sites Phase 1/2 Nggt INC. · Orange, California and 4 other locations · NCT06332807

• Effects of Palynziq on diet and health in patients with PKU

  This study is testing how the medication Palynziq affects the diet and overall health of people with PKU who are 16 and older.

  Recruiting at 2 sites Emory University · Atlanta, Georgia and 1 other locations · NCT04404530

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.