PhenylketonuriasNCT05487378
Evaluation of Phe Fluctuation in PKU Pts Treated With PKU GOLIKE Versus Standard Amino Acid Protein Substitute.
A study testing PKU GOLIKE in people with Phenylketonurias, run by APR Applied Pharma Research s.a. at 1 site in United Kingdom.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 5 to 16 years
- 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
- 16 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 30 Jun 2023to 26 May 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 5 to 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Male and female PKU patients ≥5 years and ≤16 years of age.
- Patients diagnosed with PKU via new born screening.
- Taking a Phe free/low Phe protein substitute
- On a low phenylalanine diet .
- Absence of neurological deficiencies.
- Adherence with dietary management and protein substitute.
- Able to understand and comply with the requirements of the investigation and sign the Informed Consent Form/Assent form.
Who cannot join
- Age <5 years old and >16 years old.
- Patients with mild PKU or HPA.
- On sapropterin therapy.
- Patients with late diagnosis of PKU and neurological problems.
- History of hypersensitivity to any excipients/components of the investigational product.
- Pregnancy or breastfeeding during the study.
Show all 9Show fewer
- Any moderate to severe acute illness which in the opinion of the Investigator would interfere with the study procedures or study outcome.
- History of poor co-operation, non-adherence with dietary management, or poor adherence to investigation procedures.
- Participation in any other studies involving investigational or marketed products concomitantly or within two weeks prior to entry into the study.
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site
Birmingham Children's Hospital Steelhouse Lane
Birmingham, B4 6NH
About this study
Official title: Randomised Investigation to Evaluate Phe Fluctuation in PKU Patients Treated With PKU GOLIKE Versus Standard Amino Acid Protein Substitute.
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:
Read more
- 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
- 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.
Read the detailed description
16 children with PKU who currently take a Phe-free/low Phe protein substitute (3 or 4 doses/day) will be recruited. Subjects will replace the last daily dose of their usual protein substitute with the study product for 7 days of the 28 day trial (either days 1-7 or days 22-28 based on random allocation). On the remaining study days, subjects will take an amino acid based protein substitute for all daily doses. There will be a 2 week washout period between study arms where subjects will take their usual protein substitute. The amount of study product prescribed will be calculated to provide the same amount of protein as their usual protein substitute. The last protein substitute (PS) dose of the day (amino acids or study product) will need to be taken between 7-9pm to allow an 8-10 hour fasting period overnight. Three finger prick blood spots will be collected and analysed for phenylalanine, tyrosine and BCAA at 5am, 6am and 7am on days -1, 0, 6, 7, 20, 21, 27 and 28. For all subjects, a second void urine sample will be collected on days 0, 7, 21 and 28 for analysis of urea and creatinine. A quality of sleep questionnaire will be completed by subjects or their carers on days 0, 7, 21 and 28 and a 24 hour food diary on days -1, 0, 6, 7, 20, 21, 27 and 28. A palatability questionnaire will be completed by subjects or their carers on days 7 or 28 (at the end of the period with PKU GOLIKE, if Bars or Krunches are used). Subject visits will be on days -2 (enrolment), 0, 7, 21 and 28 where the research dietitian will collect urine samples, blood spots, questionnaires and diaries.
- Conditions
- Phenylketonurias
- Being tested
- PKU GOLIKE
- 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
Also called PKU GOLIKE is a food for special medicinal purposes (FSMP) for the dietary management of PKU.
AA protein Substitute for the dietary management of PKU, PKU GOLIKE is a food for special medicinal purposes (FSMP) for the dietary management of PKU.
| Study group | Role | What they receive |
|---|---|---|
| 1st PKU GOLIKE 2nd AA protein substitute | Other | 7 days with usual protein substitute and PKU GOLIKE as their last dose of protein substitute for the day (at least one sachet with15g PE) in an amount equivalent to their usual protein substitute PE followed by a 2-week washout period on their usual protein substitute, and then 7 days with AA protein substitute for all daily doses. Receives: Dietary Supplement: PKU GOLIKE |
| 1st AA protein substitute 2nd PKU GOLIKE | Other | 7 days with AA protein substitute for all daily doses followed by a 2-week washout period on their usual protein substitute, and then 7 days with usual protein substitute and PKU GOLIKE as their last dose of protein substitute for the day (at least one sachet with15g PE) in an amount equivalent to their usual protein substitute PE Receives: Dietary Supplement: PKU GOLIKE |
What is being measured
The primary outcome is the main question the study is designed to answer.
Blood Phe After 7 Days of Each Treatment
Measurement of blood phenylalanine (Phe) levels
Measured: Mean value of Blood Sample after 7 treatment days (days 7 and 28 together depending on the period)
Secondary outcomes (1)
Dosage of Tyr in Blood (Umol/L) With Dried Blood Spots Before Breakfast
Measurement of blood tyrosine (Tyr) levels
Measured: Mean value of Blood Sample after 7 treatment days (days 7 and 28 together depending on the period)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Aug 2022
- Study start30 Jun 2023
- Primary completion26 May 2024
- Study completion26 May 2024
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
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- APR Applied Pharma Research s.a.
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Anita MacDonald, Pr., Principal investigator, Birmingham Children's Hospital, Steelhouse Lane, Birmingham B4 6NH
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05487378
- Sponsor's protocol ID
- GLK-UK-2021
- Status last verified
- Oct 2023
- First submitted
- 17 Jun 2022
- Last updated
- 23 Feb 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 23 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomised Investigation to Evaluate Phe Fluctuation in PKU Patients Treated With PKU GOLIKE Versus Standard Amino Acid Protein Substitute.
- ClinicalTrials.gov ID
- NCT05487378
- Acronym / sponsor ID
- GLK-UK-2021
- Conditions
- Phenylketonurias
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- APR Applied Pharma Research s.a.University, hospital or other
- Study start
- 30 Jun 2023 (actual)
- Primary completion
- 26 May 2024 (actual)
- Study completion
- 26 May 2024 (actual)
- First posted
- 4 Aug 2022 (actual)
- Results
- Posted 8 Jun 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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:
- 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
- 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.
Detailed description
16 children with PKU who currently take a Phe-free/low Phe protein substitute (3 or 4 doses/day) will be recruited. Subjects will replace the last daily dose of their usual protein substitute with the study product for 7 days of the 28 day trial (either days 1-7 or days 22-28 based on random allocation). On the remaining study days, subjects will take an amino acid based protein substitute for all daily doses. There will be a 2 week washout period between study arms where subjects will take their usual protein substitute. The amount of study product prescribed will be calculated to provide the same amount of protein as their usual protein substitute. The last protein substitute (PS) dose of the day (amino acids or study product) will need to be taken between 7-9pm to allow an 8-10 hour fasting period overnight. Three finger prick blood spots will be collected and analysed for phenylalanine, tyrosine and BCAA at 5am, 6am and 7am on days -1, 0, 6, 7, 20, 21, 27 and 28. For all subjects, a second void urine sample will be collected on days 0, 7, 21 and 28 for analysis of urea and creatinine. A quality of sleep questionnaire will be completed by subjects or their carers on days 0, 7, 21 and 28 and a 24 hour food diary on days -1, 0, 6, 7, 20, 21, 27 and 28. A palatability questionnaire will be completed by subjects or their carers on days 7 or 28 (at the end of the period with PKU GOLIKE, if Bars or Krunches are used). Subject visits will be on days -2 (enrolment), 0, 7, 21 and 28 where the research dietitian will collect urine samples, blood spots, questionnaires and diaries.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1st PKU GOLIKE 2nd AA protein substitute | Other | Dietary Supplement: PKU GOLIKE7 days with usual protein substitute and PKU GOLIKE as their last dose of protein substitute for the day (at least one sachet with15g PE) in an amount equivalent to their usual protein substitute PE followed by a 2-week washout period on their usual protein substitute, and then 7 days with AA protein substitute for all daily doses. |
| 1st AA protein substitute 2nd PKU GOLIKE | Other | Dietary Supplement: PKU GOLIKE7 days with AA protein substitute for all daily doses followed by a 2-week washout period on their usual protein substitute, and then 7 days with usual protein substitute and PKU GOLIKE as their last dose of protein substitute for the day (at least one sachet with15g PE) in an amount equivalent to their usual protein substitute PE |
Who may qualify
- Ages
- 5 to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- Male and female PKU patients ≥5 years and ≤16 years of age.
- Patients diagnosed with PKU via new born screening.
- Taking a Phe free/low Phe protein substitute
- On a low phenylalanine diet .
- Absence of neurological deficiencies.
Key exclusion criteria (9 listed)
- Age <5 years old and >16 years old.
- Patients with mild PKU or HPA.
- On sapropterin therapy.
- Patients with late diagnosis of PKU and neurological problems.
- History of hypersensitivity to any excipients/components of the investigational product.
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 GOLIKEAlso: PKU GOLIKE is a food for special medicinal purposes (FSMP) for the dietary management of PKU.AA protein Substitute for the dietary management of PKU, PKU GOLIKE is a food for special medicinal purposes (FSMP) for the dietary management of PKU. | Dietary supplement | 1st AA protein substitute 2nd PKU GOLIKE, 1st PKU GOLIKE 2nd AA protein substitute |
Primary outcome
Blood Phe After 7 Days of Each Treatment
Time frame: Mean value of Blood Sample after 7 treatment days (days 7 and 28 together depending on the period)
Measurement of blood phenylalanine (Phe) levels
1 secondary outcome
Dosage of Tyr in Blood (Umol/L) With Dried Blood Spots Before Breakfast
Time frame: Mean value of Blood Sample after 7 treatment days (days 7 and 28 together depending on the period)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Feb 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.
Anita MacDonald, Pr. principal investigator
Birmingham Children's Hospital, Steelhouse Lane, Birmingham B4 6NH
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 United Kingdom.
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 7 inclusion and 9 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: finished, as of the sponsor's last update on 23 Feb 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 United Kingdom. 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 8 Jun 2025). 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?
Blood Phe After 7 Days of Each Treatment (time frame: Mean value of Blood Sample after 7 treatment days (days 7 and 28 together depending on the period)). 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 23 Feb 2026.