At a glance
ClinicalIndex Comparison Record- ✓Age 5 to 16 years old
- ✓Diagnosed with PKU via newborn screening
- ✓Currently on phenylalanine-free or low-phenylalanine protein substitute
- ✓On a low phenylalanine diet
- ✕Age less than 5 years or greater than 16 years
- ✕Mild PKU or HPA (hyperphenylalaninemia)
- ✕Currently on sapropterin therapy
- ✕Late PKU diagnosis with neurological problems
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
Randomised Investigation to Evaluate Phe Fluctuation in PKU Patients Treated With PKU GOLIKE Versus Standard Amino Acid Protein Substitute.
In Brief
A clinical study evaluating PKU GOLIKE for Phenylketonurias. Completed, enrolled 16 participants across 1 site.
Detailed 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.
Study Details
Timeline
Interventions
AA protein Substitute for the dietary management of PKU, PKU GOLIKE is a food for special medicinal purposes (FSMP) for the dietary management of PKU.