NCT05827536

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.

Terminated NA Results posted Last updated 11 March 2026

What this trial tests

NA trial testing PKU GOLIKE in Phenylketonurias in 21 participants. Terminated before completion.

Timeline

30 October 2023

Primary endpoint
27 November 2024

27 November 2024

Quick facts

Lead sponsor	APR Applied Pharma Research s.a.
Phase	NA
Status	Terminated
Study type	INTERVENTIONAL
Allocation	randomized
Design	crossover
Masking	none
Primary purpose	treatment
Enrollment	21
Start date	30 October 2023
Primary completion	27 November 2024
Estimated completion	27 November 2024
Sites	1 location across Italy

Drugs / interventions tested

• PKU GOLIKE
• Standard of Care

Conditions studied

• Phenylketonurias — all drugs for Phenylketonurias →

Sponsor

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

Who can join

16 and older, 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.

Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With Standard of Care Right Before Product Self-administration and Before Any Food Intake Primary · at 8:00 am(±15 min) on the second test day (T2; T4) with SoC

Measurement of blood phenylalanine (Phe) levels

Group	Value	95% CI
SoC at T2	620.2	± 154.56
SoC at T4	554.2	± 202.28

Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With GOLIKE PLUS Right Before Product Self-administration and Before Any Food Intake Primary · at 8:00 am(±15 min) on the second test day (T2; T4) with Golike Plus

Group	Value	95% CI
PKU Golike at T2	553.4	± 230.99
PKU Golike at T4	537.4	± 147.87

Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With Standard of Care Right Before Product Self-administration and Before Food Intake Primary · at 12:00 am (±15 min) on the second test day (T2; T4)with SoC

Group	Value	95% CI
SoC at T2	574.9	± 127.60
SoC at T4	533.3	± 206.68

Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With GOLIKE PLUS Right Before Product Self-administration and Before Food Intake Primary · at 12:00 am (±15 min) on the second test day (T2; T4) with Golike Plus

Group	Value	95% CI
PKU Golike at T2	523.2	± 216.90
PKU Golike at T4	527.9	± 195.50

Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With Standard of Care Before Food Intake Primary · At 4:00 pm (±15 min) on the second test (T2;T4) day with SoC

Group	Value	95% CI
SoC at T2	559.9	± 141.58
SoC at T4	522.8	± 162.32

Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With GOLIKE PLUS Before Food Intake Primary · At 4:00 pm (±15 min) on the second test day (T2; T4) with Golike Plus

Group	Value	95% CI
PKU Golike at T2	505.5	± 244.91
PKU Golike at T4	504.7	± 176.47

Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With Standard of Care Right Before Product Self-administration and Before Any Food Intake Primary · at 8:00 pm (±15 min) on the second test day (T2; T4) with SoC

Group	Value	95% CI
SoC at T2	589.8	± 144.38
SoC at T4	532.5	± 198.72

Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With GOLIKE PLUS Right Before Product Self-administration and Before Any Food Intake Primary · at 8:00 pm (±15 min) on the second test day (T2; T4) with Golike Plus

Group	Value	95% CI
PKU Golike at T2	509.7	± 229.24
PKU Golike at T4	519.9	± 187.42

Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Day Following the Second Test Day With Standard of Care on the Following Morning, Before Any Food Intake. Primary · at 8:00 am (±15 min) on the day following the second test day (T2; T4) with SoC

Group	Value	95% CI
SoC at T2	585.1	± 158.88
SoC at T4	532.0	± 205.12

Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Day Following the Second Test Day With GOLIKE PLUS on the Following Morning, Before Any Food Intake. Primary · at 8:00 am (±15 min) on the day following the second test day (T2; T4) with Golike Plus

Group	Value	95% CI
PKU Golike at T2	506.1	± 222.73
PKU Golike at T4	531.4	± 194.77

Tyr Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With Standard of Care Right Before Product Self-administration and Before Any Food Intake Secondary · at 8:00 am(±15 min) on the second test day (T2; T4) with SoC

Measurement of blood Tyrosine (Tyr) levels

Group	Value	95% CI
Soc at T2	56.6	± 15.26
SoC at T4	54.8	± 15.82

Tyr Blood Concentration With Dried Blood Spots (Umol/L)on the Second Test Day With GOLIKE PLUS Right Before Product Self-administration and Before Any Food Intake Secondary · at 8:00 am(±15 min)on the second test day (T2; T4) with Golike Plus

Group	Value	95% CI
PKU Golike at T2	64.6	± 10.36
PKU Golike at T4	59.7	± 13.41

Adverse events — posted to ClinicalTrials.gov

Time frame: From the signature of the informed Consent form (V0) to the completion of the study (end of study visit) this means about 4 weeks.. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

A Where A=Standard of Care (SoC)

Serious: 0/21 (0%)

Deaths: 0/21

B Where B=PKU GOLIKE

Serious: 0/21 (0%)

Deaths: 0/21

Other adverse events (1 terms — click to expand)

Reaction	System	A Where A=Standard of Care…	B Where B=PKU GOLIKE
ASTHENIA	General disorders	—	—

Data from ClinicalTrials.gov NCT05827536 adverse events section.

Sponsor's own description

This is an open-label, randomized, 2-way crossover, monocentric controlled study in patients (≥ 16 years old) with phenylketonuria (PKU).

The comparison will be between the test product (PKU GOLIKE, a prolonged-release amino-acids (AAs) mixture) and standard of care.

Publications & conference data

No peer-reviewed publications indexed yet for this trial.