Evaluation of PKU UP

NCT05995717 | Active Not Recruiting

• 3 other identifiers: PKU-PAED-LIQ-2022-10-0732764123/NW/0185
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 7

Brief Summary

PKU UP is a prospective, single-arm, open-label, 26-week acceptability study to evaluate PKU UP for the dietary management of participants with phenylketonuria (PKU). Up to 15 participants aged 1 - 10 years old will be recruited and it is anticipated the study will open in at least three sites in the United Kingdom (UK) to recruit the target number of participants in the required timeframe.

Conditions

PKU

Keywords

Phenylketonuria; PKU; Glyco-Macro-Peptide; GMP; amino acid; AA; protein substitute; PS; United Kingdom; UK; children; PKU UP; Glycomacropeptide

Outcome Measures

Primary Outcomes (11)

• Change in phe level between baseline and visit 3

  Change in phe level as measured by dried blood spot testing

  26 weeks

• Change in tyrosine level between baseline and visit 3

  Change in tyrosine level as measured by dried blood spot testing

  26 weeks

• Change in PI impression of metabolic control

  Assessment of the participant's metabolic control in the opinion of the investigator

  Week 0, week 6, week 12, week 26

• Change in the growth of participants as measured by Height (length in under 2 years of age), Head circumference in under 2 years of age and Weight.

  Assessed via anthropometry measurements. Participants will be weighed and measured for growth monitoring.

  Week 0, week 12, week 26

• Change in GI tolerance (participant)

  Gastrointestinal tolerance assessed using the PedsQL Gastrointestinal Symptoms Scale completed by the participants

  Week 0, week 6, week 12, week 26

• Change in PI impression of GI tolerance

  Assessment of the participant's GI tolerance in the opinion of the investigator

  Week 0, week 6, week 12, week 26

• Change in compliance with study product consumption

  Assessed via participant diary regarding study product intake

  Week 0, week 6, week 12, week 26

• Change in PI impression of study product intake

  Assessment of the participant's compliance in the opinion of the investigator

  Week 0, week 6, week 12, week 26

• Change in product acceptability

  Assessed via a product acceptability questionnaire completed by the participant/parent

  Week 0, week 12, week 26

• Change in dietary intake

  Assessed via 24-hour recall and low protein food frequency questionnaire

  Week 0, week 12, week 26

• Change in nutritional biochemistry

  Assessed via venous blood sample of approximately 10ml

  Week 0, week 26

Study Arms (1)

PKU UP

EXPERIMENTAL

All participants will be assessed by their dietitian and prescribed an appropriate amount of the study product, PKU UP, to manage their phenylketonuria.

Dietary Supplement: PKU UP

Interventions

PKU UP DIETARY_SUPPLEMENT

PKU UP is a food for special medical purposes (FSMP). This product is for use in the dietary management of Phenylketonuria (PKU) in children aged from one (1) to ten (10) years. PKU UP is a neutral-flavoured, ready-to-drink, low-phenylalanine protein substitute containing a blend of glycomacropeptide (GMP) isolate, essential and non-essential amino acids, fat, carbohydrate, vitamins, minerals and docosahexaenoic acid (DHA).

PKU UP

Eligibility Criteria

• Age: 1 Year - 10 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Diagnosis of PKU requiring a low-protein diet and protein substitute.
• Participants who are adherent with their blood phenylalanine (Phe) testing, in the opinion of the investigator.
• Participants aged 1-10 years.
• Participants who in the opinion of the investigator are anticipated to be able to take at least half of their daily protein equivalent requirement from PKU UP (in order to assess this, a taste test (or similar) may be conducted at investigator's discretion as part of the screening process).
• Willingly given, written, informed consent from the parent(s)/guardian(s).
• Willingly given, written assent by the participant (if appropriate).

You may not qualify if:

• Inability to comply with the study protocol, in the opinion of the investigator.
• Any co-morbidity, which, in the opinion of the investigator, would preclude participation in the study.
• Diagnosis of persistent hyperphenylalaninemia, or mild PKU not requiring a low protein diet and protein substitute.
• Allergy or intolerance to milk.
• Participants who are currently participating in, plan to participate in or have participated in an interventional investigational drug, food or medical device study within 30 days prior to the screening visit.
• Use of additional micronutrient supplements during the evaluation period, unless clinically indicated and prescribed by the Investigator (must be recorded in both the medical records and case report form).

Sponsors & Collaborators

• Vitaflo International, Ltd: lead
• Birmingham Women's and Children's NHS Foundation Trust: collaborator

Study Sites (7)

Royal Belfast Hospital for Sick Children

Belfast, BT12 6BE, United Kingdom

Location

Birmingham Children's Hospital

Birmingham, B4 6NH, United Kingdom

Location

St. Luke's Hospital

Bradford, BD5 0NA, United Kingdom

Location

University Hospitals Bristol and Weston NHS Foundation Trust

Bristol, BS2 8BJ, United Kingdom

Location

Great Ormond Street Hospital for Children NHS Foundation Trust

London, WC1N 3JH, United Kingdom

Location

Nottingham Children's Hospital

Nottingham, NG7 2UH, United Kingdom

Location

Sheffield Children's Hospital

Sheffield, S10 2TH, 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

Study Officials

• Anita MacDonald

  Birmingham Women's and Children's NHS Foundation Trust

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 3, 2023
• First Posted: August 16, 2023
• Study Start: January 11, 2024
• Primary Completion (Estimated): May 31, 2026
• Study Completion (Estimated): May 31, 2026
• Last Updated: February 19, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

GB (7)