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.

NCT05827536 | Terminated Results

• 1 other identifier: GLK-IT-2023
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 1

Brief Summary

This is an open-label, randomized, 2-way crossover, monocentric controlled study in patients (≥ 16 years old) with phenylketonuria (PKU). The comparison will be between the test product (PKU GOLIKE, a prolonged-release amino-acids (AAs) mixture) and standard of care.

Conditions

Phenylketonurias

Outcome Measures

Primary Outcomes (10)

• 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

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

• Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With GOLIKE PLUS Right Before Product Self-administration and Before Any Food Intake

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

• Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Day Following the Second Test Day With Standard of Care on the Following Morning, Before Any Food Intake.

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

• Phe Blood Concentration With Dried Blood Spots (Umol/L) on the Day Following the Second Test Day With GOLIKE PLUS on the Following Morning, Before Any Food Intake.

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

Secondary Outcomes (10)

• Tyr Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With Standard of Care Right Before Product Self-administration and Before Any Food Intake

  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

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

• Tyr Blood Concentration With Dried Blood Spots (Umol/L)on the Second Test Day With Standard of Care Right Before Product Self-administration and Before Any Food Intake

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

• Tyr Blood Concentration With Dried Blood Spots (Umol/L) on the Second Test Day With GOLIKE PLUS Right Before Product Self-administration and Before Any Food Intake

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

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

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

Study Arms (2)

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

Interventions

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; BA where B=PKU GOLIKE and A=standard of care

Standard of Care DIETARY_SUPPLEMENT

Protein substitute

AB where A=standard of care and B=PKU GOLIKE; BA where B=PKU GOLIKE and A=standard of care

Eligibility Criteria

• Age: 16 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Signed, informed consent obtained by the patient prior to beingenrolled into the study and prior to starting any data collection. For legally minor patients, signed written consent shall be obtained also by the parents/legal guardian
• Male or female, aged ≥16 years.
• Patients with a registered diagnosis of PKU
• Ability and willingness to comply with all study procedures and availability for the duration of the study.
• Patient taking free-AA and/or GMP as usual amino-acidssupplementation.

You may not qualify if:

• Known or suspected hypersensitivity to any excipients/components of PKU GOLIKE.
• Treatment with any drug therapy for PKU
• Patient taking PKU GOLIKE as usual amino-acids supplementation
• Patient taking LNAA as usual amino-acids supplementation
• Any moderate to severe medical condition, which in the opinion of the Investigator would interfere with the study procedures or study outcome (reason to be provided)
• Pregnancy or lactation.

Sponsors & Collaborators

• APR Applied Pharma Research s.a.: lead

Study Sites (1)

ASST Santi Paolo e Carlo, Presidio Ospedale San Paolo, Via Antonio di Rudinì 8

Milan, 20142, Italy

Location

MeSH Terms

Conditions

Phenylketonurias

Interventions

Standard of Care

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Quality Indicators, Health Care; Quality of Health Care; Health Services Administration; Health Care Quality, Access, and Evaluation

Results Point of Contact

• Title: Chief Scientific Officer
• Organization: APR Applied Pharma Research s.a.

giorgio.reiner@apr.ch

+41.91.6957020

Study Officials

• Valentina Rovelli, MD

  ASST Santi Paolo e Carlo, Presidio Ospedale San Paolo, Via Antonio di Rudinì 8 - 20142, Milano (Italy)

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: The study products will be self-administered at home according to the assigned randomization sequences, AB or BA where A=standard of care and B=PKU GOLIKE. Patients will be randomized in a 1:1 ratio to one of the following two sequences: * AB: patients will receive 3 daily self-administrations of their usual standard of care for 2 consecutive days in the first period followed by 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the second period. * BA: patients will receive 3 daily self-administrations of PKU GOLIKE for 2 consecutive days in the first period followed by 3 daily self-administrations of their usual standard of care for 2 consecutive days in the second period. Patients that are not compliant or need to be excluded for other clinical reasons from the treatment schedule will be replaced in order to ensure 20 subjects completing both treatment sequences.

Study Record Dates

• First Submitted: March 24, 2023
• First Posted: April 25, 2023
• Study Start: October 30, 2023
• Primary Completion: November 27, 2024
• Study Completion: November 27, 2024
• Last Updated: March 11, 2026
• Results First Posted: March 11, 2026

Record last verified: 2026-03

Locations

IT (1)