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RECRUITINGOBSERVATIONAL

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

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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

Who May Be Eligible (Plain English)

Who May Qualify:- 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 - willing to sign a consent form 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 Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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

Treatments Being Tested

OTHER

no intervention

no intervention

Locations (1)

University Childrens Hospital Zürich
Zurich, Switzerland