Open-label, Randomized, 2-way Crossover, Monocentric, Controlled Study to Evaluate the Effect on Daily PHE Fluctuation of PKU GOLIKE Versus SoC in Patients With PKU.

Launched by APR APPLIED PHARMA RESEARCH S.A. · Apr 12, 2023

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called PKU GOLIKE, which is a special mixture of amino acids, to see how it affects daily fluctuations in blood levels of phenylalanine (Phe) in patients with phenylketonuria (PKU). The study will compare this new treatment to the standard care that patients currently receive. It is open to adults aged 16 and older who have a confirmed diagnosis of PKU and have had higher than normal blood Phe levels in the past year.

To participate, individuals need to give their consent and be willing to follow the study's procedures. They must also be using amino acid supplements as part of their regular treatment. Participants will not be able to join if they have certain medical conditions, are pregnant or breastfeeding, or are currently involved in another clinical trial. This is an exciting opportunity for eligible patients to potentially benefit from a new treatment option while contributing to important research in the management of PKU.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Signed, informed consent obtained by the patient prior to beingenrolled into the study and prior to starting any data collection. For legally minor patients, signed written consent shall be obtained also by the parents/legal guardian
• • 2. Male or female, aged ≥16 years.
• • 3. Patients with a registered diagnosis of PKU
• • 4. Ability and willingness to comply with all study procedures and availability for the duration of the study.
• • 5. Patients with mean value of blood Phe \>360 µM in the previous 12 months (calculated on at least 3 samples during the previous 12 months; the last sample should be preferably obtained in the 30 days preceding inclusion in the study).
• • 6. Patient taking free-AA and/or GMP as usual amino-acidssupplementation.
• Exclusion Criteria:
• • 1. Known or suspected hypersensitivity to any excipients/components of PKU GOLIKE.
• • 2. Treatment with any drug therapy for PKU
• • 3. Patient taking PKU GOLIKE as usual amino-acids supplementation
• • 4. Patient taking LNAA as usual amino-acids supplementation
• • 5. Any moderate to severe medical condition, which in the opinion of the Investigator would interfere with the study procedures or study outcome (reason to be provided)
• • 6. Any current participation in another clinical trial involving investigational or marketed products in the 3 months prior to the inclusion in this study.
• • 7. Pregnancy or lactation.