Evaluation of Phe Fluctuation in PKU Pts Treated With PKU GOLIKE Versus Standard Amino Acid Protein Substitute.
Phenylketonurias
About this trial
This is an interventional treatment trial for Phenylketonurias
Eligibility Criteria
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.
- History of hypersensitivity to any excipients/components of the investigational product.
- Pregnancy or breastfeeding during the study.
- Any moderate to severe acute illness which in the opinion of the Investigator would interfere with the study procedures or study outcome.
- History of poor co-operation, non-adherence with dietary management, or poor adherence to investigation procedures.
- Participation in any other studies involving investigational or marketed products concomitantly or within two weeks prior to entry into the study.
Sites / Locations
- Birmingham Children's Hospital Steelhouse LaneRecruiting
Arms of the Study
Arm 1
Arm 2
Other
Other
1st PKU GOLIKE 2nd AA protein substitute
1st AA protein substitute 2nd PKU GOLIKE
7 days with usual protein substitute and PKU GOLIKE as their last dose of protein substitute for the day (at least one sachet with15g PE) in an amount equivalent to their usual protein substitute PE followed by a 2-week washout period on their usual protein substitute, and then 7 days with AA protein substitute for all daily doses.
7 days with AA protein substitute for all daily doses followed by a 2-week washout period on their usual protein substitute, and then 7 days with usual protein substitute and PKU GOLIKE as their last dose of protein substitute for the day (at least one sachet with15g PE) in an amount equivalent to their usual protein substitute PE