NCT05487378

Evaluation of Phe Fluctuation in PKU Pts Treated With PKU GOLIKE Versus Standard Amino Acid Protein Substitute.

Completed NA Results posted Last updated 23 February 2026

What this trial tests

NA trial testing PKU GOLIKE in Phenylketonurias in 16 participants. Completed in 26 May 2024.

Timeline

30 June 2023

Primary endpoint
26 May 2024

26 May 2024

Quick facts

Lead sponsor	APR Applied Pharma Research s.a.
Phase	NA
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	crossover
Masking	none
Primary purpose	treatment
Enrollment	16
Start date	30 June 2023
Primary completion	26 May 2024
Estimated completion	26 May 2024
Sites	1 location across United Kingdom

Drugs / interventions tested

• PKU GOLIKE

Conditions studied

• Phenylketonurias — all drugs for Phenylketonurias →

Sponsor

APR Applied Pharma Research s.a. — full company profile →

Who can join

Adults 5 to 16, any sex, with Phenylketonurias. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Blood Phe After 7 Days of Each Treatment Primary · Mean value of Blood Sample after 7 treatment days (days 7 and 28 together depending on the period)

Measurement of blood phenylalanine (Phe) levels

Phe level Before All study partecipants from both arms start GolikeTreatment

Group	Value	95% CI
All Study Participants	357.5	± 155.58

Phe level After All study Partcipants from both arms are 7 days treated with Golike Treatment

Group	Value	95% CI
All Study Participants	294.0	± 149.93

Phe level Before All study partecipants from both arms start AA Treatment

Group	Value	95% CI
All Study Participants	346.8	± 143.72

Phe level After All study Partcipants from both arms are 7 days treated with AA Treatment

Group	Value	95% CI
All Study Participants	442.4	± 177.58

Dosage of Tyr in Blood (Umol/L) With Dried Blood Spots Before Breakfast Secondary · Mean value of Blood Sample after 7 treatment days (days 7 and 28 together depending on the period)

Measurement of blood tyrosine (Tyr) levels

Tyr level Before All study partecipants from both arms start GolikeTreatment

Group	Value	95% CI
All Study Participants	46.4	± 15.76

Tyr level after All study partecipants from both arms are 7 days treated with GolikeTreatment

Group	Value	95% CI
All Study Participants	62.1	± 23.43

Tyr level Before All study partecipants from both arms start AA Treatment

Group	Value	95% CI
All Study Participants	49.5	± 11.93

Tyr level after All study partecipants from both arms are 7 days treated with AA Treatment

Group	Value	95% CI
All Study Participants	51.6	± 22.02

Adverse events — posted to ClinicalTrials.gov

Time frame: Safety information collected from the signature of the ICF (V0) to the end of study (about 5 weeks).. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

PKU GOLIKE TREATMENT

Serious: 0/16 (0%)

Deaths: 0/16

AA TREATMENT

Serious: 0/16 (0%)

Deaths: 0/16

Other adverse events (2 terms — click to expand)

Reaction	System	PKU GOLIKE TREATMENT	AA TREATMENT
viral infections	Infections and infestations	—	—
vomiting	Infections and infestations	—	—

Data from ClinicalTrials.gov NCT05487378 adverse events section.

Sponsor's own description

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.

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

1. Protein Substitute Absorption: A Randomised Controlled Trial Comparing CGMP vs. Amino Acids vs. Micellar Casein in Healthy Volunteers.
   Daly A, Pinto A, Evans S, Geberhiwot T, et al · · 2025 · cited 1× · PMID 40871699 · DOI 10.3390/nu17162671