Evaluation of the Express Plus Range

NCT05051657 | Completed

• 3 other identifiers: MCT-EXPP-2019-05-2128199520/YH/0248
• Study Type: interventional
• Target: 28
• Locations: 1 country
• Sites: 6

Brief Summary

A prospective, open label, acceptability study to evaluate PKU, MSUD, HCU, TYR and GA express plus in the dietary management of 40 patients with IEM. The following parameters will be assessed: adherence to prescribed dietary intakes, palatability, usability, gastrointestinal tolerance, clinically relevant routine biochemical parameters, timeframe to transition and contribution of the express plus range to overall protein substitute intake over a 28 day period.

Conditions

Phenylketonurias; PKU; Homocystinuria; Maple Syrup Urine Disease; Hereditary Tyrosinemia; Glutaric Acidemia I

Outcome Measures

Primary Outcomes (8)

• Questionnaire of self-reported adherence to the prescribed amount of study product

  Participants will record the amount of PKU express taken each day compared to the amount recommended by their dietitian

  Days 1-28

• Transition time

  Time taken for participants to transition from their current diet to the inclusion of one sachet of PKU express plus per day

  Transition period Weeks 1-6 (maximum)

• Change in gastrointestinal tolerance from week 1 to week 4

  Participants will self-report any gastrointestinal symptoms experience over the course of the study in daily study diaries

  Days 1-7 and days 21-28

• Product acceptability rated on a Likert scale by the patient after 28-day intake

  Participants will answer questions relating to PKU express plus' palatability and ease of use following the end of study

  Visit 2 (end of 28 day evaluation period)

• Change in general neophobia

  Participants to complete general neophobia questionnaire at baseline and end of study to compare results

  Visit 1 (Day 1) and Visit 2 (end of 28 day evaluation period)

• Change in food neophobia

  Participants to complete food neophobia questionnaire at baseline and end of study to compare results

  Visit 1 (Day 1) and Visit 2 (end of 28 day evaluation period)

• Nutritional suitability: change Phenylalanine levels

  PKU express plus' nutritional suitability will be assessed by evaluating Phenylalanine levels recorded as part of routine care

  Visit 1 until visit 2 (day 28)

• Nutritional suitability: change Tyrosine levels

  PKU express plus' nutritional suitability will be assessed by evaluating Tyrosine levels recorded as part of routine care

  Visit 1 until visit 2 (day 28)

Study Arms (1)

express pus range (PKU, MSUD, HCU, TYR and GA)

EXPERIMENTAL

express plus to be transitioned onto over a maximum of 6 weeks and then incorporated into each participants usual diet for 28 days. Amount taken and frequency to be determined by dietitian.

Dietary Supplement: PKU express plus

Interventions

PKU express plus DIETARY_SUPPLEMENT

PKU express plus is a powdered protein substitute for the dietary management of PKU. It is formulated in a variety of flavours to provide a convenient, low volume, lower calorie, pre-measured protein substitute, containing a full micronutrient profile.

express pus range (PKU, MSUD, HCU, TYR and GA)

Eligibility Criteria

• Age: 3 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of PKU or associated disorder for example, Tetrahydrobiopterin (BH4) deficiency, requiring a low protein diet and Phe-free L-amino acid supplements (protein substitute).
• Established on PKU express or an equivalent protein substitute
• In the opinion of the study investigator, the participant is able to take the study product and meets at least one of the following criteria:
• taking an age inappropriate protein substitute
• struggling with adherence to current protein substitute
• delayed in transitioning
• Aged 3 years and above
• Willingly given, written, informed consent from patient or parent/guardian.
• Willingly given, written assent (if appropriate).

You may not qualify if:

• Diagnosis of persistent hyperphenylalaninaemia, or mild PKU not requiring dietary intervention with a low protein diet and protein substitute.
• Patients who are tube-fed.
• Individuals, who in the opinion of the investigator, are unable to comply with the requirements of the protocol.
• Any co-morbidity/condition which in the opinion of the Investigator, would preclude participation in the study.
• Patients who are currently participating in, plan to participate in, or have participated in an interventional investigational drug, food or medical device trial within 30 days prior to screening.
• Use of additional macro/micronutrient supplements during the study period, unless clinically indicated and prescribed by the investigator (must be recorded in patient case record file).
• Women who are pregnant / breastfeeding at the start of the study or planning to become pregnant during the study period. (N.B.: Women who become pregnant unexpectedly during this study may, in consultation with their doctor, continue on the study's dietary product if they wish but will not have any investigations that would not normally be carried out during pregnancy.)
• Those with allergies to fish, milk or soya.

Sponsors & Collaborators

• Vitaflo International, Ltd: lead

Study Sites (6)

Royal Belfast Hospital for Sick Children

Belfast, BT12 6BA, United Kingdom

Location

Birmingham Women's and Children's Hospital

Birmingham, B15 2TG, United Kingdom

Location

Bristol Royal Hospital for Children

Bristol, BS2 8BJ, United Kingdom

Location

Greater Glasgow and Clyde NHS Foundation Trust

Glasgow, G12 0XH, United Kingdom

Location

Great Ormond Street Hospital for Children

London, WC1N 3JH, United Kingdom

Location

Nottingham Children's Hospital

Nottingham, NG7 2UH, United Kingdom

Location

MeSH Terms

Conditions

Phenylketonurias; Homocystinuria; Maple Syrup Urine Disease; Tyrosinemias; Glutaric Acidemia I

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; Hyperhomocysteinemia; Connective Tissue Diseases; Skin and Connective Tissue Diseases

Study Officials

• Anita MacDonald

  Birmingham Women's and Children's Hospital

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Masking Details: No masking, product will be given open label
• Purpose: SUPPORTIVE CARE
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: single group - all patients to receive relevant express plus product

Study Record Dates

• First Submitted: September 10, 2021
• First Posted: September 21, 2021
• Study Start: June 14, 2021
• Primary Completion: September 30, 2025
• Study Completion: January 21, 2026
• Last Updated: February 19, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

GB (6)