Evaluating PKU GOLIKE versus standard care in patients with phenylketonuria

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

This study is testing if a new amino-acid treatment called PKU GOLIKE can help people with phenylketonuria manage their phenylalanine levels better than the usual care they receive.

Quick facts

• Phase: Not applicable
• Study type: Interventional
• Enrollment: 20 (estimated)
• Ages: 16 Years and up
• Sex: All
• Sponsor: APR Applied Pharma Research s.a. (Academic / other)
• Locations: 1 site (Milan)
• Trial ID: NCT05827536 on ClinicalTrials.gov

What this trial studies

This study is an open-label, randomized, 2-way crossover trial designed to compare the effects of PKU GOLIKE, a prolonged-release amino-acid mixture, against standard care in patients aged 16 and older with phenylketonuria (PKU). Participants will be randomly assigned to receive either treatment first, followed by the other, with each treatment period lasting two consecutive days. The study will monitor daily fluctuations in phenylalanine (PHE) levels, with patients self-administering the treatments at home. The aim is to determine if PKU GOLIKE can provide better management of PHE levels compared to standard care.

Who should consider this trial

Good fit: Ideal candidates are individuals aged 16 and older with a confirmed diagnosis of phenylketonuria and elevated blood PHE levels.

Not a fit: Patients currently receiving PKU GOLIKE or those with hypersensitivity to its components may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to improved management of phenylketonuria, potentially stabilizing PHE levels more effectively than current treatments.

How similar studies have performed: While this approach is novel in its specific formulation, similar studies have shown promise in managing PKU with alternative amino-acid therapies.

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.

Where this trial is running

Milan

• ASST Santi Paolo e Carlo, Presidio Ospedale San Paolo, Via Antonio di Rudinì 8 — Milan, Italy (Recruiting)

Study contacts

• Principal investigator: Valentina Rovelli, MD — ASST Santi Paolo e Carlo, Presidio Ospedale San Paolo, Via Antonio di Rudinì 8 - 20142, Milano (Italy)
• Study coordinator: Giorgio Reiner

Conditions Phenylketonurias