Trial Outcomes & Findings for 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. (NCT NCT05827536)
NCT ID: NCT05827536
Last Updated: 2026-03-11
Results Overview
Recruitment status
TERMINATED
Study phase
NA
Target enrollment
21 participants
Primary outcome timeframe
at 8:00 am (±15 min) on the day following the second test day (T2; T4) with Golike Plus
Results posted on
2026-03-11
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)
A total of 21 participants signed the informed consent form and were assigned to a study arm (either AB or BA) as per protocol. Four participants did not receive any treatments, and one participant received the first assigned treatment only. These five participants were therefore excluded from the ITT population due to the absence of blood spot samples required for the determination of amino acid levels.
Participant milestones
| Measure |
AB =1st Usual SoC, 2nd PKU GOLIKE
Patients are randomized to 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.
B=PKU GOLIKE. A=Standard of Care
|
BA =1st PKU GOLIKE 2nd Usual SoC
Patients are randomized to 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.
B=PKU GOLIKE. A=Standard of Care
|
|---|---|---|
|
1st Period 3 Daily Administrations 2 Day
STARTED
|
10
|
11
|
|
1st Period 3 Daily Administrations 2 Day
COMPLETED
|
6
|
7
|
|
1st Period 3 Daily Administrations 2 Day
NOT COMPLETED
|
4
|
4
|
|
2nd Period 3 Daily Administrations 2 Day
STARTED
|
10
|
11
|
|
2nd Period 3 Daily Administrations 2 Day
COMPLETED
|
5
|
7
|
|
2nd Period 3 Daily Administrations 2 Day
NOT COMPLETED
|
5
|
4
|
Reasons for withdrawal
| Measure |
AB =1st Usual SoC, 2nd PKU GOLIKE
Patients are randomized to 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.
B=PKU GOLIKE. A=Standard of Care
|
BA =1st PKU GOLIKE 2nd Usual SoC
Patients are randomized to 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.
B=PKU GOLIKE. A=Standard of Care
|
|---|---|---|
|
1st Period 3 Daily Administrations 2 Day
Protocol Violation
|
2
|
1
|
|
1st Period 3 Daily Administrations 2 Day
Withdrawal by Subject
|
2
|
3
|
|
2nd Period 3 Daily Administrations 2 Day
Withdrawal by Subject
|
3
|
3
|
|
2nd Period 3 Daily Administrations 2 Day
Protocol Violation
|
2
|
1
|
Baseline Characteristics
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.
Baseline characteristics by cohort
| Measure |
AB =1st Usual SoC, 2nd PKU GOLIKE
n=10 Participants
Patients are randomized to 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.
B=PKU GOLIKE. A=Standard of Care
|
BA =1st PKU GOLIKE 2nd Usual SoC
n=11 Participants
Patients are randomized to 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.
B=PKU GOLIKE. A=Standard of Care
|
Total
n=21 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
31.5 years
STANDARD_DEVIATION 8.05 • n=9 Participants
|
28.18 years
STANDARD_DEVIATION 9.63 • n=9 Participants
|
29.8 years
STANDARD_DEVIATION 8.85 • n=18 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=9 Participants
|
3 Participants
n=9 Participants
|
9 Participants
n=18 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=9 Participants
|
8 Participants
n=9 Participants
|
12 Participants
n=18 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=9 Participants
|
0 Participants
n=9 Participants
|
0 Participants
n=18 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=9 Participants
|
0 Participants
n=9 Participants
|
0 Participants
n=18 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=9 Participants
|
0 Participants
n=9 Participants
|
0 Participants
n=18 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=9 Participants
|
0 Participants
n=9 Participants
|
0 Participants
n=18 Participants
|
|
Race (NIH/OMB)
White
|
10 Participants
n=9 Participants
|
11 Participants
n=9 Participants
|
21 Participants
n=18 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=9 Participants
|
0 Participants
n=9 Participants
|
0 Participants
n=18 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=9 Participants
|
0 Participants
n=9 Participants
|
0 Participants
n=18 Participants
|
PRIMARY outcome
Timeframe: at 8:00 am(±15 min) on the second test day (T2; T4) with SoCMeasurement of blood phenylalanine (Phe) levels
Outcome measures
| Measure |
SoC at T2
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
SoC at T4
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
|---|---|---|
|
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
|
620.2 μmol/L
Standard Deviation 154.56
|
554.2 μmol/L
Standard Deviation 202.28
|
PRIMARY outcome
Timeframe: at 8:00 am(±15 min) on the second test day (T2; T4) with Golike PlusOutcome measures
| Measure |
SoC at T2
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
SoC at T4
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
|---|---|---|
|
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
|
553.4 μmol/L
Standard Deviation 230.99
|
537.4 μmol/L
Standard Deviation 147.87
|
PRIMARY outcome
Timeframe: at 12:00 am (±15 min) on the second test day (T2; T4)with SoCOutcome measures
| Measure |
SoC at T2
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
SoC at T4
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
|---|---|---|
|
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
|
574.9 μmol/L
Standard Deviation 127.60
|
533.3 μmol/L
Standard Deviation 206.68
|
PRIMARY outcome
Timeframe: at 12:00 am (±15 min) on the second test day (T2; T4) with Golike PlusOutcome measures
| Measure |
SoC at T2
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
SoC at T4
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
|---|---|---|
|
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
|
523.2 μmol/L
Standard Deviation 216.90
|
527.9 μmol/L
Standard Deviation 195.50
|
PRIMARY outcome
Timeframe: At 4:00 pm (±15 min) on the second test (T2;T4) day with SoCPopulation: Data not availale for 1 participant.
Outcome measures
| Measure |
SoC at T2
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
SoC at T4
n=7 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
|---|---|---|
|
Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With Standard of Care Before Food Intake
|
559.9 μmol/L
Standard Deviation 141.58
|
522.8 μmol/L
Standard Deviation 162.32
|
PRIMARY outcome
Timeframe: At 4:00 pm (±15 min) on the second test day (T2; T4) with Golike PlusPopulation: Data not availale for 1 participant.
Outcome measures
| Measure |
SoC at T2
n=7 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
SoC at T4
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
|---|---|---|
|
Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With GOLIKE PLUS Before Food Intake
|
505.5 μmol/L
Standard Deviation 244.91
|
504.7 μmol/L
Standard Deviation 176.47
|
PRIMARY outcome
Timeframe: at 8:00 pm (±15 min) on the second test day (T2; T4) with SoCOutcome measures
| Measure |
SoC at T2
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
SoC at T4
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
|---|---|---|
|
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
|
589.8 μmol/L
Standard Deviation 144.38
|
532.5 μmol/L
Standard Deviation 198.72
|
PRIMARY outcome
Timeframe: at 8:00 pm (±15 min) on the second test day (T2; T4) with Golike PlusOutcome measures
| Measure |
SoC at T2
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
SoC at T4
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
|---|---|---|
|
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
|
509.7 μmol/L
Standard Deviation 229.24
|
519.9 μmol/L
Standard Deviation 187.42
|
PRIMARY outcome
Timeframe: at 8:00 am (±15 min) on the day following the second test day (T2; T4) with SoCOutcome measures
| Measure |
SoC at T2
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
SoC at T4
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
|---|---|---|
|
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 μmol/L
Standard Deviation 158.88
|
532.0 μmol/L
Standard Deviation 205.12
|
PRIMARY outcome
Timeframe: at 8:00 am (±15 min) on the day following the second test day (T2; T4) with Golike PlusOutcome measures
| Measure |
SoC at T2
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
SoC at T4
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
|---|---|---|
|
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 μmol/L
Standard Deviation 222.73
|
531.4 μmol/L
Standard Deviation 194.77
|
SECONDARY outcome
Timeframe: at 8:00 am(±15 min) on the second test day (T2; T4) with SoCMeasurement of blood Tyrosine (Tyr) levels
Outcome measures
| Measure |
SoC at T2
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
SoC at T4
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
|---|---|---|
|
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
|
56.6 μmol/L
Standard Deviation 15.26
|
54.8 μmol/L
Standard Deviation 15.82
|
SECONDARY outcome
Timeframe: at 8:00 am(±15 min)on the second test day (T2; T4) with Golike PlusOutcome measures
| Measure |
SoC at T2
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
SoC at T4
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
|---|---|---|
|
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
|
64.6 μmol/L
Standard Deviation 10.36
|
59.7 μmol/L
Standard Deviation 13.41
|
SECONDARY outcome
Timeframe: at 12:00 am(±15 min) on the second test day (T2; T4) with SoCOutcome measures
| Measure |
SoC at T2
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
SoC at T4
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
|---|---|---|
|
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
|
57.0 μmol/L
Standard Deviation 17.28
|
72.2 μmol/L
Standard Deviation 18.84
|
SECONDARY outcome
Timeframe: at 12:00 am(±15 min) on the second test day (T2; T4) with Golike PlusOutcome measures
| Measure |
SoC at T2
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
SoC at T4
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
|---|---|---|
|
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
|
83.7 μmol/L
Standard Deviation 28.85
|
74.8 μmol/L
Standard Deviation 16.65
|
SECONDARY outcome
Timeframe: at 4:00 pm(±15 min)on the second test day (T2; T4) with SoCPopulation: Data not availale for 1 participant.
Outcome measures
| Measure |
SoC at T2
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
SoC at T4
n=7 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
|---|---|---|
|
Tyr Blood Concentration With Dried Blood Spots (Umol/L)on the Second Test Day With Standard of Care Before Food Intake
|
68.3 μmol/L
Standard Deviation 20.11
|
72.0 μmol/L
Standard Deviation 13.89
|
SECONDARY outcome
Timeframe: at 4:00 pm(±15 min)on the second test day (T2; T4) with Golike PlusPopulation: Data not availale for 1 participant.
Outcome measures
| Measure |
SoC at T2
n=7 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
SoC at T4
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
|---|---|---|
|
Tyr Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With GOLIKE PLUS Before Food Intake
|
85.6 μmol/L
Standard Deviation 19.03
|
80.6 μmol/L
Standard Deviation 28.68
|
SECONDARY outcome
Timeframe: at 8:00 pm(±15 min) on the second test day (T2; T4) with SoCOutcome measures
| Measure |
SoC at T2
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
SoC at T4
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
|---|---|---|
|
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
|
59.0 μmol/L
Standard Deviation 11.65
|
50.9 μmol/L
Standard Deviation 9.26
|
SECONDARY outcome
Timeframe: at 8:00 pm(±15 min) on the second test day (T2; T4) with Golike PlusOutcome measures
| Measure |
SoC at T2
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
SoC at T4
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
|---|---|---|
|
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
|
63.7 μmol/L
Standard Deviation 13.18
|
68.5 μmol/L
Standard Deviation 13.19
|
SECONDARY outcome
Timeframe: at 8:00 am(±15 min) on the day following the second test day (T2; T4) with SoCOutcome measures
| Measure |
SoC at T2
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
SoC at T4
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
|---|---|---|
|
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 μmol/L
Standard Deviation 14.25
|
54.1 μmol/L
Standard Deviation 11.13
|
SECONDARY outcome
Timeframe: at 8:00 am(±15 min) on the day following the second test day (T2; T4) with Golike PlusOutcome measures
| Measure |
SoC at T2
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
SoC at T4
n=8 Participants
Patients were recruited in the study and randomly assigned to one of two treatment sequences:
1. AB: Patients received 3 daily self-administrations of their usual standard of care for 2 consecutive days (T1-T2) in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period (T3-T4).
2. BA: Patients received 3 daily self-administrations of PKU GOLIKE for 2 consecutive days (T1-T2) in the first period followed by 3 daily self- administrations of their usual standard of care for 2 consecutive days (T3-T4) in the second period.
|
|---|---|---|
|
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 μmol/L
Standard Deviation 18.70
|
57.2 μmol/L
Standard Deviation 20.96
|
Adverse Events
A Where A=Standard of Care (SoC)
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
B Where B=PKU GOLIKE
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 |
A Where A=Standard of Care (SoC)
n=21 participants at risk
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.
PKU GOLIKE: PKU GOLIKE is a food for special medicinal purposes (FSMP) for the dietary management of PKU.
Standard of Care: Protein substitute
|
B Where B=PKU GOLIKE
n=21 participants at risk
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.
PKU GOLIKE: PKU GOLIKE is a food for special medicinal purposes (FSMP) for the dietary management of PKU.
Standard of Care: Protein substitute
|
|---|---|---|
|
General disorders
ASTHENIA
|
0.00%
0/21 • From the signature of the informed Consent form (V0) to the completion of the study (end of study visit) this means about 4 weeks.
|
4.8%
1/21 • Number of events 1 • From the signature of the informed Consent form (V0) to the completion of the study (end of study visit) this means about 4 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
- Publication restrictions are in place
Restriction type: LTE60