Evaluation of Phe Fluctuation in PKU Pts Treated With PKU GOLIKE Versus Standard Amino Acid Protein Substitute.

NCT05487378 | Completed Results

• 1 other identifier: GLK-UK-2021
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 1

Brief Summary

This is a 2 arm, randomised, controlled, cross-over study in 16 children with PKU. Subjects who are currently taking a Phe free/low Phe protein substitute will be recruited for a 31-day trial. Patients will be randomised to receive:

• Collect 3 finger prick blood spots on days -1, 0, 6, 7, 20, 21, 27 and 28.
• Collect urine sample, second void of the day on days 0, 7, 21 and 28.
• Complete a questionnaire on sleep quality on day 0, 7, 21 and 28.
• Complete a 24 hour food diary on days -1, 0, 6, 7, 20, 21, 27 and 28. APR will supply the study product for participants free of charge.

Conditions

Phenylketonurias

Outcome Measures

Primary Outcomes (1)

• Blood Phe After 7 Days of Each Treatment

  Measurement of blood phenylalanine (Phe) levels

  Mean value of Blood Sample after 7 treatment days (days 7 and 28 together depending on the period)

Secondary Outcomes (1)

• Dosage of Tyr in Blood (Umol/L) With Dried Blood Spots Before Breakfast

  Mean value of Blood Sample after 7 treatment days (days 7 and 28 together depending on the period)

Study Arms (2)

1st PKU GOLIKE 2nd AA protein substitute

OTHER

7 days with usual protein substitute and PKU GOLIKE as their last dose of protein substitute for the day (at least one sachet with15g PE) in an amount equivalent to their usual protein substitute PE followed by a 2-week washout period on their usual protein substitute, and then 7 days with AA protein substitute for all daily doses.

Dietary Supplement: PKU GOLIKE

1st AA protein substitute 2nd PKU GOLIKE

OTHER

7 days with AA protein substitute for all daily doses followed by a 2-week washout period on their usual protein substitute, and then 7 days with usual protein substitute and PKU GOLIKE as their last dose of protein substitute for the day (at least one sachet with15g PE) in an amount equivalent to their usual protein substitute PE

Dietary Supplement: PKU GOLIKE

Interventions

PKU GOLIKE DIETARY_SUPPLEMENT

AA protein Substitute for the dietary management of PKU, PKU GOLIKE is a food for special medicinal purposes (FSMP) for the dietary management of PKU.

Also known as: PKU GOLIKE is a food for special medicinal purposes (FSMP) for the dietary management of PKU.

1st AA protein substitute 2nd PKU GOLIKE; 1st PKU GOLIKE 2nd AA protein substitute

Eligibility Criteria

• Age: 5 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Male and female PKU patients ≥5 years and ≤16 years of age.
• Patients diagnosed with PKU via new born screening.
• Taking a Phe free/low Phe protein substitute
• On a low phenylalanine diet .
• Absence of neurological deficiencies.
• Adherence with dietary management and protein substitute.
• Able to understand and comply with the requirements of the investigation and sign the Informed Consent Form/Assent form.

You may not qualify if:

• Age \<5 years old and \>16 years old.
• Patients with mild PKU or HPA.
• On sapropterin therapy.
• Patients with late diagnosis of PKU and neurological problems.
• History of hypersensitivity to any excipients/components of the investigational product.
• Pregnancy or breastfeeding during the study.
• Any moderate to severe acute illness which in the opinion of the Investigator would interfere with the study procedures or study outcome.
• History of poor co-operation, non-adherence with dietary management, or poor adherence to investigation procedures.
• Participation in any other studies involving investigational or marketed products concomitantly or within two weeks prior to entry into the study.

Sponsors & Collaborators

• APR Applied Pharma Research s.a.: lead

Study Sites (1)

Birmingham Children's Hospital Steelhouse Lane

Birmingham, B4 6NH, United Kingdom

Location

MeSH Terms

Conditions

Phenylketonurias

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

Results Point of Contact

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

giorgio.reiner@apr.ch

+41.91.6957020

Study Officials

• Anita MacDonald, Pr.

  Birmingham Children's Hospital, Steelhouse Lane, Birmingham B4 6NH

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• 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

Study Record Dates

• First Submitted: June 17, 2022
• First Posted: August 4, 2022
• Study Start: June 30, 2023
• Primary Completion: May 26, 2024
• Study Completion: May 26, 2024
• Last Updated: February 23, 2026
• Results First Posted: June 8, 2025

Record last verified: 2023-10

Locations

GB (1)