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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT05128149
Other study ID # PKU_Guide
Secondary ID
Status Recruiting
Phase
First received
Last updated
Start date November 15, 2021
Est. completion date December 31, 2024

Study information

Verified date December 2023
Source University Children's Hospital, Zurich
Contact Martina Huemer, Prof
Phone +41 44 2660
Email martina.huemer@kispi.uzh.ch
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

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


Recruitment information / eligibility

Status Recruiting
Enrollment 200
Est. completion date December 31, 2024
Est. primary completion date June 30, 2024
Accepts healthy volunteers No
Gender All
Age group 10 Years and older
Eligibility 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

Study Design


Related Conditions & MeSH terms


Intervention

Other:
no intervention
no intervention

Locations

Country Name City State
Switzerland University Childrens Hospital Zürich Zürich

Sponsors (4)

Lead Sponsor Collaborator
University Children's Hospital, Zurich Insel Gruppe AG, University Hospital Bern, UChildrenVienna, University Children's Hospital Basel

Country where clinical trial is conducted

Switzerland, 

Outcome

Type Measure Description Time frame Safety issue
Primary Phe values biochemical marker for PKU report on measurements from past 24 months
Secondary Health-related quality of life PKU-QoL© Questionnaire one hour
Secondary Self-efficacy 10 items Self-efficacy scale (Schwarzer& Jerusalem) one hour
Secondary Burden on family (patients from 10-18 years) Revised Impact on Family Scale one hour
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