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TerminatedNCT05827536Updated Mar 11, 2026Results posted

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.

An interventional study of PKU GOLIKE and Standard of Care in Phenylketonurias, sponsored by APR Applied Pharma Research s.a.. Terminated at 1 site in Italy. Open to participants aged 16 Years and older. Per ClinicalTrials.gov, last updated 2026-03-11.

Sponsored by APR Applied Pharma Research s.a. · Not applicable, Interventional, and Treatment

Why this study was terminated
Due to recruitment challenges, and despite an extension of the study period, the decision to terminate the trial early was made in agreement with the Principal Investigator
Phase
Not applicable
Study type
Interventional
Enrollment
21
Allocation
Randomized
Ages
16 Years and older
Sex
All
01

Study summary

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.

Read the detailed description

The study will consist of a screening visit (V1), two treatment periods with two test days each and a final visit planned at the end of the second treatment period (V2). Test days will occur on two consecutive preferably non-working/school days (T1-T2 and T3-T4,) preferably over two consecutive weeks. Treatment days in the two periods will have to be the same days of the week. T1 should be within two weeks after V1. The study products will be self-administered at home. Following informed consent and verification of eligibility criteria, 20 patients with PKU will be randomized in a 1:1 ratio to one of the following two sequences: AB or BA where A=standard of care and B=PKU GOLIKE. In details:

  • AB: patients will receive 3 daily self-administrations of their usual standard of care for 2 consecutive days in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in thesecond period.
  • BA: patients will receive 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the first period followed by 3 daily self administrations of their usual standard of care for 2 consecutive days in the second period.

No change in the randomization sequence will be allowed.

Each patient will follow the same diet in terms of food, calories and nutrients ranges during the first days (T1 and T3) of each period and the same diet during the second days (T2 and T4) of each period, according to the age and body weight of the patient. The days before the test days each patient will follow her/his standard diet.

PKU GOLIKE /standard of care will be the only protein substitute allowed on each test day, and no sports activities will be allowed on sampling days. Five blood spots will be collected on the second test day of each period. Timing of self-administrations, meals and blood spots will be standardized for each test day. On the test days, no food will be allowed outside of the defined time windows. The first self-administration of each test day will be performed after an overnight fasting (10 -12 h) and before any food intake.

A patient's e-diary will be used to collect information on patient compliance, 24-hour blood spot collections, diet, daily activities, adverse events and event-related concomitant medications.

Adverse events will be continuously monitored during the study, starting from informed consent. Adverse events will be collected by the patients (or by a parent/guardian) in the e-diary and during the telephone calls made by the Investigator to the patients. Moreover, patients (or parents/guardians) will be instructed to promptly report adverse events occurring during the study to the Investigator.

The end of study (V2) will be performed (remotely, by phone) at the end ofthe second treatment period, within 2 weeks after the last test day. Patients prematurely discontinued from the study will be asked to attend (remotely, by phone) a discontinuation visit possibly taking place within 2 weeks from the last test day. At the final/discontinuation visit, the investigator will organize collection of blood spots test and residual PKU GOLIKE product from the patient's domicile through a dedicated courier service. The account of the e-diary will be inactivated.

02

Conditions studied

  • Phenylketonurias

Browse trials for

03

In context

Phenylketonurias

183 studies on the registry are indexed under Phenylketonurias; 38 are open to participants now.

This study's enrollment of 21 is below the median of 25 across 114 interventional studies indexed under Phenylketonurias.

Browse Phenylketonurias studies →

Lead sponsor

APR Applied Pharma Research s.a. is the lead sponsor of 12 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
16 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

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

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.
05

Study design

Phase
Not applicable
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Crossover assignment
Masking
None (open label)
Enrollment
21 participants (actual)

Study arms

  • Other
    AB where A=standard of care and B=PKU GOLIKE

    Patients will receive 3 daily self-administrations of their usual standard of care for 2 consecutive days in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period.

    Dietary Supplement: PKU GOLIKE · Dietary Supplement: Standard of Care

  • Other
    BA where B=PKU GOLIKE and A=standard of care

    Patients will receive 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the first period followed by 3 daily self-administrations of their usual standard of care for 2 consecutive days in the second period.

    Dietary Supplement: PKU GOLIKE · Dietary Supplement: Standard of Care

Interventions

  • Dietary supplementPKU GOLIKE

    PKU GOLIKE is a food for special medicinal purposes (FSMP) for the dietary management of PKU.

  • Dietary supplementStandard of Care

    Protein substitute

06

What researchers measure

Primary outcomes

  1. 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

    Measurement of blood phenylalanine (Phe) levels

    Time frame: at 8:00 am(±15 min) on the second test day (T2; T4) with SoC

  2. 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

    Time frame: at 8:00 am(±15 min) on the second test day (T2; T4) with Golike Plus

  3. 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

    Time frame: at 12:00 am (±15 min) on the second test day (T2; T4)with SoC

  4. 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

    Time frame: at 12:00 am (±15 min) on the second test day (T2; T4) with Golike Plus

  5. Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With Standard of Care Before Food Intake

    Time frame: At 4:00 pm (±15 min) on the second test (T2;T4) day with SoC

  6. Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With GOLIKE PLUS Before Food Intake

    Time frame: At 4:00 pm (±15 min) on the second test day (T2; T4) with Golike Plus

  7. 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

    Time frame: at 8:00 pm (±15 min) on the second test day (T2; T4) with SoC

  8. 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

    Time frame: at 8:00 pm (±15 min) on the second test day (T2; T4) with Golike Plus

  9. 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.

    Time frame: at 8:00 am (±15 min) on the day following the second test day (T2; T4) with SoC

  10. 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.

    Time frame: at 8:00 am (±15 min) on the day following the second test day (T2; T4) with Golike Plus

Secondary outcomes

  1. 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

    Measurement of blood Tyrosine (Tyr) levels

    Time frame: at 8:00 am(±15 min) on the second test day (T2; T4) with SoC

  2. 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

    Time frame: at 8:00 am(±15 min)on the second test day (T2; T4) with Golike Plus

  3. 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

    Time frame: at 12:00 am(±15 min) on the second test day (T2; T4) with SoC

  4. 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

    Time frame: at 12:00 am(±15 min) on the second test day (T2; T4) with Golike Plus

  5. Tyr Blood Concentration With Dried Blood Spots (Umol/L)on the Second Test Day With Standard of Care Before Food Intake

    Time frame: at 4:00 pm(±15 min)on the second test day (T2; T4) with SoC

  6. Tyr Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With GOLIKE PLUS Before Food Intake

    Time frame: at 4:00 pm(±15 min)on the second test day (T2; T4) with Golike Plus

  7. 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

    Time frame: at 8:00 pm(±15 min) on the second test day (T2; T4) with SoC

  8. 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

    Time frame: at 8:00 pm(±15 min) on the second test day (T2; T4) with Golike Plus

  9. Tyr 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.

    Time frame: at 8:00 am(±15 min) on the day following the second test day (T2; T4) with SoC

  10. Tyr 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.

    Time frame: at 8:00 am(±15 min) on the day following the second test day (T2; T4) with Golike Plus

07

Results

Posted Mar 11, 2026

Participant flow

Participants were recruited at San Paolo Hospital (Milan-Italy) The First Patient First Visit (FPFV) - defined as the first patient enrolled who signed the Informed Consent Form - took place on 30 October 2023.The last patient completed the study on 27 November 2024, corresponding to the final visit of the last subject (Last Patient Last Visit, LPLV)

1st Period 3 Daily Administrations 2 Day
Participant flow — 1st Period 3 Daily Administrations 2 Day
MilestoneAB =1st Usual SoC, 2nd PKU GOLIKEBA =1st PKU GOLIKE 2nd Usual SoC
Started1011
Completed67
Not completed44
Withdrew: Protocol violation21
Withdrew: Withdrawal by subject23
2nd Period 3 Daily Administrations 2 Day
Participant flow — 2nd Period 3 Daily Administrations 2 Day
MilestoneAB =1st Usual SoC, 2nd PKU GOLIKEBA =1st PKU GOLIKE 2nd Usual SoC
Started1011
Completed57
Not completed54
Withdrew: Withdrawal by subject33
Withdrew: Protocol violation21

Outcome measures

PrimaryPhe 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

Measurement of blood phenylalanine (Phe) levels

Time frame:
at 8:00 am(±15 min) on the second test day (T2; T4) with SoC
Reported as:
Mean · μmol/L
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
μmol/LSoC at T2SoC at T4
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 Intake620.2 ± 154.56554.2 ± 202.28
PrimaryPhe 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
Time frame:
at 8:00 am(±15 min) on the second test day (T2; T4) with Golike Plus
Reported as:
Mean · μmol/L
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
μmol/LPKU Golike at T2PKU Golike at T4
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 Intake553.4 ± 230.99537.4 ± 147.87
PrimaryPhe 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
Time frame:
at 12:00 am (±15 min) on the second test day (T2; T4)with SoC
Reported as:
Mean · μmol/L
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
μmol/LSoC at T2SoC at T4
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 Intake574.9 ± 127.60533.3 ± 206.68
PrimaryPhe 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
Time frame:
at 12:00 am (±15 min) on the second test day (T2; T4) with Golike Plus
Reported as:
Mean · μmol/L
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
μmol/LPKU Golike at T2PKU Golike at T4
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 Intake523.2 ± 216.90527.9 ± 195.50
PrimaryPhe Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With Standard of Care Before Food Intake
Time frame:
At 4:00 pm (±15 min) on the second test (T2;T4) day with SoC
Reported as:
Mean · μmol/L
Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With Standard of Care Before Food Intake
μmol/LSoC at T2SoC at T4
Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With Standard of Care Before Food Intake559.9 ± 141.58522.8 ± 162.32
PrimaryPhe Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With GOLIKE PLUS Before Food Intake
Time frame:
At 4:00 pm (±15 min) on the second test day (T2; T4) with Golike Plus
Reported as:
Mean · μmol/L
Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With GOLIKE PLUS Before Food Intake
μmol/LPKU Golike at T2PKU Golike at T4
Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With GOLIKE PLUS Before Food Intake505.5 ± 244.91504.7 ± 176.47
PrimaryPhe 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
Time frame:
at 8:00 pm (±15 min) on the second test day (T2; T4) with SoC
Reported as:
Mean · μmol/L
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
μmol/LSoC at T2SoC at T4
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 Intake589.8 ± 144.38532.5 ± 198.72
PrimaryPhe 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
Time frame:
at 8:00 pm (±15 min) on the second test day (T2; T4) with Golike Plus
Reported as:
Mean · μmol/L
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
μmol/LPKU Golike at T2PKU Golike at T4
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 Intake509.7 ± 229.24519.9 ± 187.42
PrimaryPhe 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.
Time frame:
at 8:00 am (±15 min) on the day following the second test day (T2; T4) with SoC
Reported as:
Mean · μmol/L
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.
μmol/LSoC at T2SoC at T4
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.585.1 ± 158.88532.0 ± 205.12
PrimaryPhe 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.
Time frame:
at 8:00 am (±15 min) on the day following the second test day (T2; T4) with Golike Plus
Reported as:
Mean · μmol/L
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.
μmol/LPKU Golike at T2PKU Golike at T4
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.506.1 ± 222.73531.4 ± 194.77
SecondaryTyr 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

Measurement of blood Tyrosine (Tyr) levels

Time frame:
at 8:00 am(±15 min) on the second test day (T2; T4) with SoC
Reported as:
Mean · μmol/L
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
μmol/LSoc at T2SoC at T4
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 Intake56.6 ± 15.2654.8 ± 15.82
SecondaryTyr 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
Time frame:
at 8:00 am(±15 min)on the second test day (T2; T4) with Golike Plus
Reported as:
Mean · μmol/L
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
μmol/LPKU Golike at T2PKU Golike at T4
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 Intake64.6 ± 10.3659.7 ± 13.41
SecondaryTyr 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
Time frame:
at 12:00 am(±15 min) on the second test day (T2; T4) with SoC
Reported as:
Mean · μmol/L
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
μmol/LSoC at T2SoC at T4
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 Intake57.0 ± 17.2872.2 ± 18.84
SecondaryTyr 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
Time frame:
at 12:00 am(±15 min) on the second test day (T2; T4) with Golike Plus
Reported as:
Mean · μmol/L
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
μmol/LPKU Golike at T2PKU Golike at T4
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 Intake83.7 ± 28.8574.8 ± 16.65
SecondaryTyr Blood Concentration With Dried Blood Spots (Umol/L)on the Second Test Day With Standard of Care Before Food Intake
Time frame:
at 4:00 pm(±15 min)on the second test day (T2; T4) with SoC
Reported as:
Mean · μmol/L
Tyr Blood Concentration With Dried Blood Spots (Umol/L)on the Second Test Day With Standard of Care Before Food Intake
μmol/LSoC at T2SoC at T4
Tyr Blood Concentration With Dried Blood Spots (Umol/L)on the Second Test Day With Standard of Care Before Food Intake68.3 ± 20.1172.0 ± 13.89
SecondaryTyr Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With GOLIKE PLUS Before Food Intake
Time frame:
at 4:00 pm(±15 min)on the second test day (T2; T4) with Golike Plus
Reported as:
Mean · μmol/L
Tyr Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With GOLIKE PLUS Before Food Intake
μmol/LPKU Golike at T2PKU Golike at T4
Tyr Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With GOLIKE PLUS Before Food Intake85.6 ± 19.0380.6 ± 28.68
SecondaryTyr 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
Time frame:
at 8:00 pm(±15 min) on the second test day (T2; T4) with SoC
Reported as:
Mean · μmol/L
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
μmol/LSoC at T2SoC at T4
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 Intake59.0 ± 11.6550.9 ± 9.26
SecondaryTyr 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
Time frame:
at 8:00 pm(±15 min) on the second test day (T2; T4) with Golike Plus
Reported as:
Mean · μmol/L
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
μmol/LPKU Golike at T2PKU Golike at T4
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 Intake63.7 ± 13.1868.5 ± 13.19
SecondaryTyr 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.
Time frame:
at 8:00 am(±15 min) on the day following the second test day (T2; T4) with SoC
Reported as:
Mean · μmol/L
Tyr 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.
μmol/LSoC at T2SoC at T4
Tyr 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.52.8 ± 14.2554.1 ± 11.13
SecondaryTyr 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.
Time frame:
at 8:00 am(±15 min) on the day following the second test day (T2; T4) with Golike Plus
Reported as:
Mean · μmol/L
Tyr 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.
μmol/LPKU Golike at T2PKU Golike at T4
Tyr 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.65.1 ± 18.7057.2 ± 20.96

Adverse events

Collected over From the signature of the informed Consent form (V0) to the completion of the study (end of study visit) this means about 4 weeks.. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
A Where A=Standard of Care (SoC)0/21 (0%)0/21 (0%)0/21 (0%)
B Where B=PKU GOLIKE0/21 (0%)0/21 (0%)1/21 (4.8%)
Most frequent other events
Most frequent other events
EventA Where A=Standard of Care (SoC)B Where B=PKU GOLIKE
ASTHENIAGeneral disorders0/211/21

Baseline characteristics

Age distribution of all randomized patients (N = 21): The mean (SD) age was 29.8 (8.85) years. The median age was 31 years.

Age, Continuous
Age, Continuous(years)AB =1st Usual SoC, 2nd PKU GOLIKEBA =1st PKU GOLIKE 2nd Usual SoCTotal
Mean31.5 ± 8.0528.18 ± 9.6329.8 ± 8.85
Sex: Female, Male
Sex: Female, Male(Participants)AB =1st Usual SoC, 2nd PKU GOLIKEBA =1st PKU GOLIKE 2nd Usual SoCTotal
Female639
Male4812
Race (NIH/OMB)
Race (NIH/OMB)(Participants)AB =1st Usual SoC, 2nd PKU GOLIKEBA =1st PKU GOLIKE 2nd Usual SoCTotal
American Indian or Alaska Native000
Asian000
Native Hawaiian or Other Pacific Islander000
Black or African American000
White101121
More than one race000
Unknown or Not Reported000
08

Study locations

1 site
  • ASST Santi Paolo e Carlo, Presidio Ospedale San Paolo, Via Antonio di Rudinì 8
    Milan, 20142, Italy
09

References and documents

Study documents

  • Protocol and statistical analysis plan · Feb 13, 2024

Documents are hosted by the registry — open the source record to download them.

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Mar 11, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT05827536
Lead sponsor
APR Applied Pharma Research s.a.
Responsible party
Sponsor
First posted
Apr 25, 2023
Start date
Oct 30, 2023
Primary completion
Nov 27, 2024
Completion
Nov 27, 2024
Results posted
Mar 11, 2026
Last update
Mar 11, 2026

Study contacts

Valentina Rovelli, MD
principal investigator · ASST Santi Paolo e Carlo, Presidio Ospedale San Paolo, Via Antonio di Rudinì 8 - 20142, Milano (Italy)

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is terminated, as verified in Mar 2026. You cannot join it, but the record below documents what was studied.

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Discussion

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