Open-label, Randomized, 2-way Crossover, Monocentric, Con... | NCT05827536

Label	Type	Description	Intervention Names
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.	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.	Dietary Supplement: PKU GOLIKE Dietary Supplement: Standard of Care

Name	Type	Description	Arm Group Labels	Other Names
PKU GOLIKE	Dietary Supplement	PKU GOLIKE is a food for special medicinal purposes (FSMP) for the dietary management of PKU.	AB where A=standard of care and B=PKU GOLIKE

Measure	Description	Time Frame
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	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		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		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		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		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		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

Measure	Description	Time Frame
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	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

Name	Affiliation	Role
Valentina Rovelli, MD	ASST Santi Paolo e Carlo, Presidio Ospedale San Paolo, Via Antonio di Rudinì 8 - 20142, Milano (Italy)	Principal Investigator

Facility	Status	City	State	ZIP	Country	Contacts
ASST Santi Paolo e Carlo, Presidio Ospedale San Paolo, Via Antonio di Rudinì 8		Milan		20142	Italy

ID	Title	Description
FG000	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
FG001

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot_SAP	Yes	Yes	No	Study Protocol and Statistical Analysis Plan	Feb 13, 2024

ID	Title	Description
BG000	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
BG001	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
BG002	Total	Total of all reporting groups

Units	Counts
Participants	BG00010 BG00111 BG00221

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

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

Posted

Mean

Standard Deviation

μmol/L

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

ID	Title	Description
OG000	SoC at T2	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.
OG001	SoC at T4	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.

Units	Counts
Participants	OG0008 OG0018

Title

Denominators

Categories

Title	Measurements
	OG000620.2± 154.56 OG001554.2± 202.28

Primary

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

Posted

Mean

Standard Deviation

μmol/L

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

ID	Title	Description
OG000	PKU Golike at T2	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.
OG001	PKU Golike at T4	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.

Primary

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

Posted

Mean

Standard Deviation

μmol/L

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

ID	Title	Description
OG000	SoC at T2	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.
OG001	SoC at T4	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.

Primary

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

Posted

Mean

Standard Deviation

μmol/L

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

ID	Title	Description
OG000	PKU Golike at T2	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.
OG001	PKU Golike at T4	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.

Primary

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

Data not availale for 1 participant.

Posted

Mean

Standard Deviation

μmol/L

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

ID	Title	Description
OG000	SoC at T2	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.
OG001	SoC at T4	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.

Primary

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

Data not availale for 1 participant.

Posted

Mean

Standard Deviation

μmol/L

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

ID	Title	Description
OG000	PKU Golike at T2	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.
OG001	PKU Golike at T4	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.

Primary

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

Posted

Mean

Standard Deviation

μmol/L

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

ID	Title	Description
OG000	SoC at T2	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.
OG001	SoC at T4	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.

Primary

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

Posted

Mean

Standard Deviation

μmol/L

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

ID	Title	Description
OG000	PKU Golike at T2	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.
OG001	PKU Golike at T4	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.

Primary

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.

Posted

Mean

Standard Deviation

μmol/L

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

ID	Title	Description
OG000	SoC at T2	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.
OG001	SoC at T4	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.

Primary

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.

Posted

Mean

Standard Deviation

μmol/L

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

ID	Title	Description
OG000	PKU Golike at T2	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.
OG001	PKU Golike at T4	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.

Secondary

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

Posted

Mean

Standard Deviation

μmol/L

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

ID	Title	Description
OG000	Soc at T2	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.
OG001	SoC at T4	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.

Secondary

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

Posted

Mean

Standard Deviation

μmol/L

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

ID	Title	Description
OG000	PKU Golike at T2	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.
OG001	PKU Golike at T4	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.

Secondary

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

Posted

Mean

Standard Deviation

μmol/L

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

ID	Title	Description
OG000	SoC at T2	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.
OG001	SoC at T4	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.

Secondary

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

Posted

Mean

Standard Deviation

μmol/L

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

ID	Title	Description
OG000	PKU Golike at T2	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.
OG001	PKU Golike at T4	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.

Secondary

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

Data not availale for 1 participant.

Posted

Mean

Standard Deviation

μmol/L

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

ID	Title	Description
OG000	SoC at T2	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.
OG001	SoC at T4	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.

Secondary

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

Data not availale for 1 participant.

Posted

Mean

Standard Deviation

μmol/L

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

ID	Title	Description
OG000	PKU Golike at T2	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.
OG001	PKU Golike at T4	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.

Secondary

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

Posted

Mean

Standard Deviation

μmol/L

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

ID	Title	Description
OG000	SoC at T2	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.
OG001	SoC at T4	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.

Secondary

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

Posted

Mean

Standard Deviation

μmol/L

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

ID	Title	Description
OG000	PKU Golike at T2	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.
OG001	PKU Golike at T4	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.

Secondary

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.

Posted

Mean

Standard Deviation

μmol/L

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

ID	Title	Description
OG000	SoC at T2	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.
OG001	SoC at T4	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.

Secondary

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.

Posted

Mean

Standard Deviation

μmol/L

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

ID	Title	Description
OG000	PKU Golike at T2	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.
OG001	PKU Golike at T4	Patients were recruited in the study and randomly assigned to one of two treatment sequences: 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). 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.

ID	Title	Description	Deaths (Affected)	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	A Where A=Standard of Care (SoC)	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	0	21	0	21	0	21
EG001	B Where B=PKU GOLIKE	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	0	21	0	21	1	21

ID	Term
D020739	Brain Diseases, Metabolic, Inborn
D001928	Brain Diseases, Metabolic
D001927	Brain Diseases
D002493	Central Nervous System Diseases
D009422	Nervous System Diseases
D000592	Amino Acid Metabolism, Inborn Errors
D008661	Metabolism, Inborn Errors
D030342	Genetic Diseases, Inborn
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D008659	Metabolic Diseases
D009750	Nutritional and Metabolic Diseases

ID	Term
D019984	Quality Indicators, Health Care
D011787	Quality of Health Care
D006298	Health Services Administration
D017530	Health Care Quality, Access, and Evaluation