Investigating the impact of dietary guidelines on well-being in patients with Phenylketonuria
Metabolic Control and Patient Well-being in Phenylketonuria: do Guidelines Make a Difference?
This study looks at how following dietary guidelines affects the health and well-being of people with Phenylketonuria (PKU) who are 10 years and older.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 200 (estimated) |
| Ages | 10 Years and up |
| Sex | All |
| Sponsor | University Children's Hospital, Zurich Academic / other |
| Locations | 1 site (Zurich) |
| Trial ID | NCT05128149 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates include patients aged 10 years and older with Phenylketonuria requiring dietary or pharmacological treatment.
Not a fit: Patients with mild forms of PKU or those not requiring dietary treatment may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to improved dietary guidelines that enhance the quality of life for PKU patients.
How similar studies have performed: While there have been studies on dietary adherence in chronic diseases, this specific approach to PKU treatment guidelines is relatively novel.
Eligibility criteria
Show full inclusion / exclusion criteria
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 this trial is running
Zurich
- University Childrens Hospital Zürich — Zurich, Switzerland (Recruiting)
Study contacts
- Study coordinator: Martina Huemer, Prof
- Email: martina.huemer@kispi.uzh.ch
- Phone: +41 44 2660
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.