Evaluation of the Express Plus Range

Phenylketonurias Clinical Trial

— express plus  

Official title:

A Study To Evaluate The Acceptability Of the Express Plus Range, Foods For Special Medical Purposes (FSMP), In Children And Adults With Inborn Errors of Metabolism (IEM)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05051657
• Other study ID #: MCT-EXPP-2019-05-21
• Secondary ID: 28199520/YH/0248
• Status: Recruiting
• Phase: N/A
• Start date: June 14, 2021
• Est. completion date: April 30, 2025

Study information

• Verified date: February 2024
• Source: Vitaflo International, Ltd
• Contact: Clinical Trials Vitaflo
• Phone: +44 (0) 151 709 9020
• Email: ClinicalTrialsTeam[@]Vitaflo.co.uk
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Description:

This is a prospective acceptability study of the express plus range in 40 participants aged at least three years of age for the dietary management of IEM (PKU, MSUD, HCU, TYR and GA ).

The study will involve a Transition Period where patients will build-up to at least one sachet of express plus per day. During the Transition Period, daily intake of express plus and the patient's other PS will be recorded, including the time taken to consume.

The Transition Period will last a maximum of 6 weeks, after which the patient will enter a 28-day Evaluation Period. During the Evaluation Period, patients will continue to record intakes, as well as GI tolerance and a final evaluation of the product's palatability and usability. Some patients, likely adults, will not require a transition from their original protein substitute to at least one sachet of express plus. These patients can enter the Evaluation Period directly.

The end of the 4-week Evaluation Period is defined as the participant's End of Study. Following this, participants will be followed up as per standard of care. Some of this standard of care data may be relayed to the sponsor for a maximum of 26 weeks. This will last for a maximum of 26 weeks and will involve the collection of routine phe and tyrosine levels, as well as amount of PS prescribed and taken. Information will also be collected from patients regarding the reasons for the success or indeed failure of transition to PKU express plus. This data will come from routine clinical contact and will be recorded by the HCP, rather than by the patient.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 40
• Est. completion date: April 30, 2025
• Est. primary completion date: October 31, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years and older

Eligibility

Inclusion Criteria:

• 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:

1. taking an age inappropriate protein substitute

2. struggling with adherence to current protein substitute

3. delayed in transitioning

• Aged 3 years and above

• Willingly given, written, informed consent from patient or parent/guardian.

• Willingly given, written assent (if appropriate).

Exclusion Criteria:

• 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.

Related Conditions & MeSH terms

• Glutaric Acidemia I
• Homocystinuria
• Maple Syrup Urine Disease
• Phenylketonurias
• PKU
• Tyrosinemias

Intervention

Dietary Supplement:
• PKU express plus
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.

Locations

Country	Name	City	State
United Kingdom	Royal Belfast Hospital for Sick Children	Belfast
United Kingdom	Birmingham Women's and Children's Hospital	Birmingham
United Kingdom	St. Luke's Hospital	Bradford
United Kingdom	Bristol Royal Hospital for Children	Bristol
United Kingdom	Greater Glasgow and Clyde NHS Foundation Trust	Glasgow
United Kingdom	Great Ormond Street Hospital for Children	London
United Kingdom	Nottingham Children's Hospital	Nottingham

Sponsors (1)

Lead Sponsor	Collaborator
Vitaflo International, Ltd

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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)
Primary	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
Primary	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)
Primary	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)
Primary	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)
Primary	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)
Primary	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)