NCT05128149 Metabolic Control and Patient Well-being in Phenylketonuria: do Guidelines Make a Difference?
| NCT ID | NCT05128149 |
| Status | Recruiting |
| Phase | — |
| Sponsor | University Children's Hospital, Zurich |
| Condition | Phenylketonurias |
| Study Type | OBSERVATIONAL |
| Enrollment | 200 participants |
| Start Date | 2021-11-15 |
| Primary Completion | 2025-03-30 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 200 participants in total. It began in 2021-11-15 with a primary completion date of 2025-03-30.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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
Eligibility 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
Contact & Investigator
Frequently Asked Questions
Who can join the NCT05128149 clinical trial?
This trial is open to participants of all sexes, aged 10 Years or older, studying Phenylketonurias. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT05128149 currently recruiting?
Yes, NCT05128149 is actively recruiting participants. Contact the research team at martina.huemer@kispi.uzh.ch for enrollment information.
Where is the NCT05128149 trial being conducted?
This trial is being conducted at Zurich, Switzerland.
Who is sponsoring the NCT05128149 clinical trial?
NCT05128149 is sponsored by University Children's Hospital, Zurich. The trial plans to enroll 200 participants.