Trial Outcomes & Findings for Evaluation of Phe Fluctuation in PKU Pts Treated With PKU GOLIKE Versus Standard Amino Acid Protein Substitute. (NCT NCT05487378)
NCT ID: NCT05487378
Last Updated: 2026-02-23
Results Overview
Measurement of blood phenylalanine (Phe) levels
Recruitment status
COMPLETED
Study phase
NA
Target enrollment
16 participants
Primary outcome timeframe
Mean value of Blood Sample after 7 treatment days (days 7 and 28 together depending on the period)
Results posted on
2026-02-23
Participant Flow
Participants were recruited at Birmingham Hospital from June 2023 to February 2024. The first participant was enrolled on 30 June 2023 (date of informed consent signature), and the last participant was enrolled in February 2024.
16 patients are recruited in the study and treated according to the study design: randomised two-periods crossover. 3 patients are excluded from analyses due to protocol deviations. So 16 patients are included in the ITT analysis and only 13 in the PP analysis.
Participant milestones
| Measure |
1st PKU GOLIKE, 2nd AA Protein Substitute
For the study All Patients are randomised to receive:
* The PKU GOLIKE treatment for 7 days as their last dose of protein substitute for the day in an amount equivalent to their usual protein substitute Proteine Equivalent; or
* An amino acid protein substitute (AA treatment) for all daily doses for 7 days.
Each the above mentioned treatments were followed by a 2-week washout period on their usual protein substitute, and then 7 days of the other study arm.
All participants were randomized to receive both Golike and AA treatments
|
1st AA Protein Substitute 2nd PKU GOLIKE
For the study All Patients are randomised to receive:
* The PKU GOLIKE treatment for 7 days as their last dose of protein substitute for the day in an amount equivalent to their usual protein substitute Proteine Equivalent; or
* An amino acid protein substitute (AA treatment) for all daily doses for 7 days.
Each the above mentioned treatments were followed by a 2-week washout period on their usual protein substitute, and then 7 days of the other study arm.
All participants were randomized to receive both Golike and AA treatments
|
|---|---|---|
|
1st Intervention (1week)
STARTED
|
9
|
7
|
|
1st Intervention (1week)
COMPLETED
|
7
|
7
|
|
1st Intervention (1week)
NOT COMPLETED
|
2
|
0
|
|
2 Weeks Washout
STARTED
|
9
|
7
|
|
2 Weeks Washout
COMPLETED
|
9
|
7
|
|
2 Weeks Washout
NOT COMPLETED
|
0
|
0
|
|
2nd Intervention (1 Week)
STARTED
|
9
|
7
|
|
2nd Intervention (1 Week)
COMPLETED
|
9
|
6
|
|
2nd Intervention (1 Week)
NOT COMPLETED
|
0
|
1
|
Reasons for withdrawal
| Measure |
1st PKU GOLIKE, 2nd AA Protein Substitute
For the study All Patients are randomised to receive:
* The PKU GOLIKE treatment for 7 days as their last dose of protein substitute for the day in an amount equivalent to their usual protein substitute Proteine Equivalent; or
* An amino acid protein substitute (AA treatment) for all daily doses for 7 days.
Each the above mentioned treatments were followed by a 2-week washout period on their usual protein substitute, and then 7 days of the other study arm.
All participants were randomized to receive both Golike and AA treatments
|
1st AA Protein Substitute 2nd PKU GOLIKE
For the study All Patients are randomised to receive:
* The PKU GOLIKE treatment for 7 days as their last dose of protein substitute for the day in an amount equivalent to their usual protein substitute Proteine Equivalent; or
* An amino acid protein substitute (AA treatment) for all daily doses for 7 days.
Each the above mentioned treatments were followed by a 2-week washout period on their usual protein substitute, and then 7 days of the other study arm.
All participants were randomized to receive both Golike and AA treatments
|
|---|---|---|
|
1st Intervention (1week)
Withdrawal by Subject
|
2
|
0
|
|
2nd Intervention (1 Week)
Withdrawal by Subject
|
0
|
1
|
Baseline Characteristics
Evaluation of Phe Fluctuation in PKU Pts Treated With PKU GOLIKE Versus Standard Amino Acid Protein Substitute.
Baseline characteristics by cohort
| Measure |
All Study Participants (ITT)
n=16 Participants
For the study All Patients are randomised to receive:
* The PKU GOLIKE treatment for 7 days as their last dose of protein substitute for the day in an amount equivalent to their usual protein substitute Proteine Equivalent; or
* An amino acid protein substitute (AA treatment) for all daily doses for 7 days.
Each the above mentioned treatments were followed by a 2-week washout period on their usual protein substitute, and then 7 days of the other study arm.
All participants were randomized to receive both Golike and AA treatments
|
|---|---|
|
Age, Continuous
|
11.19 years
STANDARD_DEVIATION 3.29 • n=58 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=58 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=58 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=58 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=58 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=58 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=58 Participants
|
|
Race (NIH/OMB)
White
|
14 Participants
n=58 Participants
|
|
Race (NIH/OMB)
More than one race
|
2 Participants
n=58 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=58 Participants
|
|
Region of Enrollment
United Kingdom
|
16 participants
n=58 Participants
|
PRIMARY outcome
Timeframe: Mean value of Blood Sample after 7 treatment days (days 7 and 28 together depending on the period)Population: In this cross-over study, the analysis was conducted by grouping patients data from both arms based on the type of treatment (Golike or AA) used for 7 days.
Measurement of blood phenylalanine (Phe) levels
Outcome measures
| Measure |
All Study Participants
n=13 Participants
For the study All Patients are randomised to receive:
* The PKU GOLIKE treatment for 7 days as their last dose of protein substitute for the day in an amount equivalent to their usual protein substitute Proteine Equivalent; or
* An amino acid protein substitute (AA treatment) for all daily doses for 7 days.
Each the above mentioned treatments were followed by a 2-week washout period on their usual protein substitute, and then 7 days of the other study arm.
All participants were randomized to receive both Golike and AA treatments
|
|---|---|
|
Blood Phe After 7 Days of Each Treatment
Phe level Before All study partecipants from both arms start AA Treatment
|
346.8 µmol/L
Standard Deviation 143.72
|
|
Blood Phe After 7 Days of Each Treatment
Phe level After All study Partcipants from both arms are 7 days treated with AA Treatment
|
442.4 µmol/L
Standard Deviation 177.58
|
|
Blood Phe After 7 Days of Each Treatment
Phe level Before All study partecipants from both arms start GolikeTreatment
|
357.5 µmol/L
Standard Deviation 155.58
|
|
Blood Phe After 7 Days of Each Treatment
Phe level After All study Partcipants from both arms are 7 days treated with Golike Treatment
|
294.0 µmol/L
Standard Deviation 149.93
|
SECONDARY outcome
Timeframe: Mean value of Blood Sample after 7 treatment days (days 7 and 28 together depending on the period)Measurement of blood tyrosine (Tyr) levels
Outcome measures
| Measure |
All Study Participants
n=13 Participants
For the study All Patients are randomised to receive:
* The PKU GOLIKE treatment for 7 days as their last dose of protein substitute for the day in an amount equivalent to their usual protein substitute Proteine Equivalent; or
* An amino acid protein substitute (AA treatment) for all daily doses for 7 days.
Each the above mentioned treatments were followed by a 2-week washout period on their usual protein substitute, and then 7 days of the other study arm.
All participants were randomized to receive both Golike and AA treatments
|
|---|---|
|
Dosage of Tyr in Blood (Umol/L) With Dried Blood Spots Before Breakfast
Tyr level Before All study partecipants from both arms start GolikeTreatment
|
46.4 µmol/L
Standard Deviation 15.76
|
|
Dosage of Tyr in Blood (Umol/L) With Dried Blood Spots Before Breakfast
Tyr level after All study partecipants from both arms are 7 days treated with GolikeTreatment
|
62.1 µmol/L
Standard Deviation 23.43
|
|
Dosage of Tyr in Blood (Umol/L) With Dried Blood Spots Before Breakfast
Tyr level Before All study partecipants from both arms start AA Treatment
|
49.5 µmol/L
Standard Deviation 11.93
|
|
Dosage of Tyr in Blood (Umol/L) With Dried Blood Spots Before Breakfast
Tyr level after All study partecipants from both arms are 7 days treated with AA Treatment
|
51.6 µmol/L
Standard Deviation 22.02
|
Adverse Events
PKU GOLIKE TREATMENT
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
AA TREATMENT
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
PKU GOLIKE TREATMENT
n=16 participants at risk
For the study Patients are randomised to receive:
* The PKU GOLIKE treatment for 7 days as their last dose of protein substitute for the day in an amount equivalent to their usual protein substitute Proteine Equivalent; or
* An amino acid protein substitute (AA treatment) for all daily doses for 7 days.
Each the above mentioned treatments were followed by a 2-week washout period on their usual protein substitute, and then 7 days of the other study arm.
In this Arm/Group, for PKU GOLIKE treatment we consider the data of all patients after the 7 days with PKU GOLIKE as their last dose of protein substitute for the day.
|
AA TREATMENT
n=16 participants at risk
For the study Patients are randomised to receive:
* The PKU GOLIKE treatment for 7 days as their last dose of protein substitute for the day in an amount equivalent to their usual protein substitute Proteine Equivalent; or
* An amino acid protein substitute (AA treatment) for all daily doses for 7 days.
Each the above mentioned treatment were followed by a 2-week washout period on their usual protein substitute, and then 7 days of the other study arm.
In this Arm/Group, for AA treatment we consider the data of all patients after the 7 days with AA treatment.
|
|---|---|---|
|
Infections and infestations
viral infections
|
12.5%
2/16 • Safety information collected from the signature of the ICF (V0) to the end of study (about 5 weeks).
|
6.2%
1/16 • Safety information collected from the signature of the ICF (V0) to the end of study (about 5 weeks).
|
|
Infections and infestations
vomiting
|
12.5%
2/16 • Safety information collected from the signature of the ICF (V0) to the end of study (about 5 weeks).
|
0.00%
0/16 • Safety information collected from the signature of the ICF (V0) to the end of study (about 5 weeks).
|
Additional Information
Chief Scientific Officer
APR Applied Pharma Research s.a.
Phone: +41.91.6957020
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee The material for public dissemination will be submitted to the Sponsor for review at least sixty (60) days (or the time specified in the Protocol if longer) prior to submission for publication, public dissemination, or review by a publication committee. All reasonable comments made by the Sponsor in relation to a proposed publication will be incorporated by the Participating Organisation and/or the Principal Investigator into the publication.
- Publication restrictions are in place
Restriction type: OTHER