PhenylketonuriasNCT05827536
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.
A study testing PKU GOLIKE in people with Phenylketonurias, run by APR Applied Pharma Research s.a. at 1 site in Italy.
Trial 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 more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Phenylketonurias
- What is being tested?
- PKU GOLIKE
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 1 siteItaly
- Study dates
- 30 Oct 2023to 27 Nov 2024
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
- Male or female, aged ≥16 years.
- Patients with a registered diagnosis of PKU
- Ability and willingness to comply with all study procedures and availability for the duration of the study.
- 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).
- Patient taking free-AA and/or GMP as usual amino-acidssupplementation.
Who cannot join
- Known or suspected hypersensitivity to any excipients/components of PKU GOLIKE.
- Treatment with any drug therapy for PKU
- Patient taking PKU GOLIKE as usual amino-acids supplementation
- Patient taking LNAA as usual amino-acids supplementation
- 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)
- Any current participation in another clinical trial involving investigational or marketed products in the 3 months prior to the inclusion in this study.
- Pregnancy or lactation.
Trial locations and contacts
1 site in 1 country.
Italy1 site
ASST Santi Paolo e Carlo, Presidio Ospedale San Paolo, Via Antonio di Rudinì 8
Milan, 20142
About this study
Official title: 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 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.
- Conditions
- Phenylketonurias
- Being tested
- PKU GOLIKE, Standard of Care
- Medical subject headings
- Phenylketonurias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PKU GOLIKEDietary supplement
PKU GOLIKE is a food for special medicinal purposes (FSMP) for the dietary management of PKU.
Standard of CareDietary supplement
Protein substitute
| Study group | Role | What they receive |
|---|---|---|
| AB where A=standard of care and B=PKU GOLIKE | Other | 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. Receives: Dietary Supplement: PKU GOLIKE; Dietary Supplement: Standard of Care |
| BA where B=PKU GOLIKE and A=standard of care | Other | 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. Receives: Dietary Supplement: PKU GOLIKE; Dietary Supplement: Standard of Care |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: at 8:00 am(±15 min) on the second test day (T2; T4) with SoC
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
Measured: at 8:00 am(±15 min) on the second test day (T2; T4) with Golike Plus
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
Measured: at 12:00 am (±15 min) on the second test day (T2; T4)with SoC
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
Measured: at 12:00 am (±15 min) on the second test day (T2; T4) with Golike Plus
Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With Standard of Care Before Food Intake
Measured: At 4:00 pm (±15 min) on the second test (T2;T4) day with SoC
Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With GOLIKE PLUS Before Food Intake
Measured: At 4:00 pm (±15 min) on the second test day (T2; T4) with Golike Plus
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
Measured: at 8:00 pm (±15 min) on the second test day (T2; T4) with SoC
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
Measured: at 8:00 pm (±15 min) on the second test day (T2; T4) with Golike Plus
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.
Measured: at 8:00 am (±15 min) on the day following the second test day (T2; T4) with SoC
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.
Measured: at 8:00 am (±15 min) on the day following the second test day (T2; T4) with Golike Plus
Secondary outcomes (10)
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
Measured: at 8:00 am(±15 min) on the second test day (T2; T4) with SoC
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
Measured: at 8:00 am(±15 min)on the second test day (T2; T4) with Golike Plus
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
Measured: at 12:00 am(±15 min) on the second test day (T2; T4) with SoC
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
Measured: at 12:00 am(±15 min) on the second test day (T2; T4) with Golike Plus
Tyr Blood Concentration With Dried Blood Spots (Umol/L)on the Second Test Day With Standard of Care Before Food Intake
Measured: at 4:00 pm(±15 min)on the second test day (T2; T4) with SoC
Tyr Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With GOLIKE PLUS Before Food Intake
Measured: at 4:00 pm(±15 min)on the second test day (T2; T4) with Golike Plus
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
Measured: at 8:00 pm(±15 min) on the second test day (T2; T4) with SoC
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
Measured: at 8:00 pm(±15 min) on the second test day (T2; T4) with Golike Plus
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.
Measured: at 8:00 am(±15 min) on the day following the second test day (T2; T4) with SoC
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.
Measured: at 8:00 am(±15 min) on the day following the second test day (T2; T4) with Golike Plus
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Apr 2023
- Study start30 Oct 2023
- Primary completion27 Nov 2024
- Study completion27 Nov 2024
Why it stopped: 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
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for PhenylketonuriasStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- The study products will be self-administered at home according to the assigned randomization sequences, AB or BA where A=standard of care and B=PKU GOLIKE. Patients will be randomized in a 1:1 ratio to one of the following two sequences: * 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 the second 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. Patients that are not compliant or need to be excluded for other clinical reasons from the treatment schedule will be replaced in order to ensure 20 subjects completing both treatment sequences.
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- APR Applied Pharma Research s.a.
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Valentina Rovelli, MD, Principal investigator, ASST Santi Paolo e Carlo, Presidio Ospedale San Paolo, Via Antonio di Rudinì 8 - 20142, Milano (Italy)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05827536
- Sponsor's protocol ID
- GLK-IT-2023
- Status last verified
- Mar 2026
- First submitted
- 24 Mar 2023
- Last updated
- 11 Mar 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT05827536
- Acronym / sponsor ID
- GLK-IT-2023
- Conditions
- Phenylketonurias
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: 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
- Enrolment
- 21 participants (actual)
- Lead sponsor
- APR Applied Pharma Research s.a.University, hospital or other
- Study start
- 30 Oct 2023 (actual)
- Primary completion
- 27 Nov 2024 (actual)
- Study completion
- 27 Nov 2024 (actual)
- First posted
- 25 Apr 2023 (actual)
- Results
- Posted 11 Mar 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- The study products will be self-administered at home according to the assigned randomization sequences, AB or BA where A=standard of care and B=PKU GOLIKE. Patients will be randomized in a 1:1 ratio to one of the following two sequences: * 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 the second 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. Patients that are not compliant or need to be excluded for other clinical reasons from the treatment schedule will be replaced in order to ensure 20 subjects completing both treatment sequences.
| Arm | Type | Receives |
|---|---|---|
| AB where A=standard of care and B=PKU GOLIKE | Other | Dietary Supplement: PKU GOLIKE, Dietary Supplement: Standard of CarePatients 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. |
| BA where B=PKU GOLIKE and A=standard of care | Other | Dietary Supplement: PKU GOLIKE, Dietary Supplement: Standard of CarePatients 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. |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- 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
- Male or female, aged ≥16 years.
- Patients with a registered diagnosis of PKU
- Ability and willingness to comply with all study procedures and availability for the duration of the study.
- 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).
Key exclusion criteria (7 listed)
- Known or suspected hypersensitivity to any excipients/components of PKU GOLIKE.
- Treatment with any drug therapy for PKU
- Patient taking PKU GOLIKE as usual amino-acids supplementation
- Patient taking LNAA as usual amino-acids supplementation
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PKU GOLIKEPKU GOLIKE is a food for special medicinal purposes (FSMP) for the dietary management of PKU. | Dietary supplement | AB where A=standard of care and B=PKU GOLIKE, BA where B=PKU GOLIKE and A=standard of care |
| Standard of CareProtein substitute | Dietary supplement | AB where A=standard of care and B=PKU GOLIKE, BA where B=PKU GOLIKE and A=standard of care |
Primary outcome
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 am(±15 min) on the second test day (T2; T4) with SoC
Measurement of blood phenylalanine (Phe) levels
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
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
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
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
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
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
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
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
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
10 secondary outcomes
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 am(±15 min) on the second test day (T2; T4) with SoC
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
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
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
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
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
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
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
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
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
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Mar 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Valentina Rovelli, MD principal investigator
ASST Santi Paolo e Carlo, Presidio Ospedale San Paolo, Via Antonio di Rudinì 8 - 20142, Milano (Italy)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing PKU GOLIKE in people with Phenylketonurias, run by APR Applied Pharma Research s.a. at 1 site in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 11 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 11 Mar 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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 am(±15 min) on the second test day (T2; T4) with SoC). The study lists 10 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Mar 2026.