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Evaluation of the Express Plus Range (express plus)

Primary Purpose

Phenylketonurias, PKU, Homocystinuria

Status

Recruiting

Phase

Not Applicable

Locations

United Kingdom

Study Type

Interventional

Intervention

PKU express plus

About this trial

This is an interventional supportive care trial for Phenylketonurias

Eligibility Criteria

3 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Royal Belfast Hospital for Sick ChildrenRecruiting
Birmingham Women's and Children's HospitalRecruiting
Greater Glasgow and Clyde NHS Foundation Trust

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

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

Arm Description

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.

Outcomes

Primary Outcome Measures

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

Transition time

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

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

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

Change in general neophobia

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

Change in food neophobia

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

Nutritional suitability: change Phenylalanine levels

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

Nutritional suitability: change Tyrosine levels

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

Secondary Outcome Measures

Full Information

NCT ID

NCT05051657

First Posted

September 10, 2021

Last Updated

April 25, 2023

Sponsor

Vitaflo International, Ltd

1. Study Identification

Unique Protocol Identification Number

NCT05051657

Brief Title

Evaluation of the Express Plus Range

Acronym

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)

Study Type

Interventional

2. Study Status

Record Verification Date

April 2023

Overall Recruitment Status

Recruiting

Study Start Date

June 14, 2021 (Actual)

Primary Completion Date

October 31, 2024 (Anticipated)

Study Completion Date

April 30, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Vitaflo International, Ltd

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

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.

Detailed 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

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

7. Study Design

Primary Purpose

Supportive Care

Study Phase

Not Applicable

Interventional Study Model

Single Group Assignment

Model Description

single group - all patients to receive relevant express plus product

Masking

None (Open Label)

Masking Description

No masking, product will be given open label

Allocation

N/A

Enrollment

40 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

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

Arm Type

Experimental

Arm Description

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.

Intervention Type

Dietary Supplement

Intervention Name(s)

PKU express plus

Intervention Description

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.

Primary Outcome Measure Information:

Title

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

Description

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

Time Frame

Days 1-28

Title

Transition time

Description

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

Time Frame

Transition period Weeks 1-6 (maximum)

Title

Change in gastrointestinal tolerance from week 1 to week 4

Description

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

Time Frame

Days 1-7 and days 21-28

Title

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

Description

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

Time Frame

Visit 2 (end of 28 day evaluation period)

Title

Change in general neophobia

Description

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

Time Frame

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

Title

Change in food neophobia

Description

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

Time Frame

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

Title

Nutritional suitability: change Phenylalanine levels

Description

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

Time Frame

Visit 1 until visit 2 (day 28)

Title

Nutritional suitability: change Tyrosine levels

Description

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

Time Frame

Visit 1 until visit 2 (day 28)

10. Eligibility

Sex

All

Minimum Age & Unit of Time

3 Years

Accepts Healthy Volunteers

Eligibility Criteria

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: 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). 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.

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Grace Thomas

Phone

01517099020

Grace.Thomas@Vitaflo.co.uk

First Name & Middle Initial & Last Name or Official Title & Degree

Erika Brennan

Phone

01517099020

erika.brennan@vitaflo.co.uk

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Anita MacDonald

Organizational Affiliation

Birmingham Women's and Children's Hospital

Official's Role

Study Director

First Name & Middle Initial & Last Name & Degree

Anne Grimsley

Organizational Affiliation

Royal Belfast Hospital for Sick Children

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Sarah Adam

Organizational Affiliation

Greater Glasgow and Clyde NHS Foundation Trust

Official's Role

Principal Investigator

Facility Information:

Facility Name

Royal Belfast Hospital for Sick Children

City

Belfast

ZIP/Postal Code

BT12 6BA

Country

United Kingdom

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Anne Grimsley

Anne.Grimsley@BelfastTrust.HSCNI.Net

Facility Name

Birmingham Women's and Children's Hospital

City

Birmingham

ZIP/Postal Code

B15 2TG

Country

United Kingdom

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Anita MacDonald

Anita.MacDonald@NHS.Net

Facility Name

Greater Glasgow and Clyde NHS Foundation Trust

City

Glasgow

ZIP/Postal Code

G12 0XH

Country

United Kingdom

Individual Site Status

Completed

12. IPD Sharing Statement

Plan to Share IPD

IPD Sharing Plan Description

No plans to share IPD

Learn more about this trial

Evaluation of the Express Plus Range

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