Evaluation of Phe Fluctuation in PKU Pts Treated With PKU GOLIKE Versus Standard Amino Acid Prote… (NCT05487378) | Clinical Trial Compass
CompletedNot Applicable
Evaluation of Phe Fluctuation in PKU Pts Treated With PKU GOLIKE Versus Standard Amino Acid Protein Substitute.
United Kingdom16 participantsStarted 2023-06-30
Plain-language summary
This is a 2 arm, randomised, controlled, cross-over study in 16 children with PKU. Subjects who are currently taking a Phe free/low Phe protein substitute will be recruited for a 31-day trial.
Patients will be randomised to receive:
1. The study product for 7 days as their last dose of protein substitute for the day (at least one sachet with 15g PE) in an amount equivalent to their usual protein substitute PE; or
2. An amino acid protein substitute for all daily doses for 7 days; followed by a 2-week washout period on their usual protein substitute,
and then 7 days of the other study arm.
During this time, patients/caregivers will be asked to:
* Collect 3 finger prick blood spots on days -1, 0, 6, 7, 20, 21, 27 and 28.
* Collect urine sample, second void of the day on days 0, 7, 21 and 28.
* Complete a questionnaire on sleep quality on day 0, 7, 21 and 28.
* Complete a 24 hour food diary on days -1, 0, 6, 7, 20, 21, 27 and 28.
APR will supply the study product for participants free of charge.
Who can participate
Age range5 Years – 16 Years
SexALL
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Inclusion criteria
✓. Male and female PKU patients ≥5 years and ≤16 years of age.
✓. Patients diagnosed with PKU via new born screening.
✓. Taking a Phe free/low Phe protein substitute
✓. On a low phenylalanine diet .
✓. Absence of neurological deficiencies.
✓. Adherence with dietary management and protein substitute.
✓. Able to understand and comply with the requirements of the investigation and sign the Informed Consent Form/Assent form.
Exclusion criteria
✕. Age \<5 years old and \>16 years old.
✕. Patients with mild PKU or HPA.
✕. On sapropterin therapy.
✕. Patients with late diagnosis of PKU and neurological problems.
What they're measuring
1
Blood Phe After 7 Days of Each Treatment
Timeframe: Mean value of Blood Sample after 7 treatment days (days 7 and 28 together depending on the period)