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Evaluation of PKU UP (PKU UP)

Primary Purpose

PKU

Status
Not yet recruiting
Phase
Not Applicable
Locations
United Kingdom
Study Type
Interventional
Intervention
PKU UP
Sponsored by
Vitaflo International, Ltd
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for PKU focused on measuring Phenylketonuria, PKU, Glyco-Macro-Peptide, GMP, amino acid, AA, protein substitute, PS, United Kingdom, UK, children, PKU UP, Glycomacropeptide

Eligibility Criteria

1 Year - 10 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria: 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 are anticipated to be able to take at least half of their daily protein equivalent requirement from PKU UP. Willingly given, written, informed consent from the parent(s)/guardian(s). Willingly given, written assent by the participant (if appropriate). Exclusion Criteria: 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).

Sites / Locations

  • Royal Belfast Hospital for Sick Children
  • Birmingham Children's Hospital
  • University Hospitals Bristol and Weston NHS Foundation Trust
  • Great Ormond Street Hospital for Children NHS Foundation Trust

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

PKU UP

Arm Description

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

Outcomes

Primary Outcome Measures

Change in phe level between baseline and visit 3
Change in phe level as measured by dried blood spot testing
Change in tyrosine level between baseline and visit 3
Change in tyrosine level as measured by dried blood spot testing
Change in PI impression of metabolic control
Assessment of the participant's metabolic control in the opinion of the investigator
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.
Change in GI tolerance (participant)
Gastrointestinal tolerance assessed using the PedsQL Gastrointestinal Symptoms Scale completed by the participants
Change in PI impression of GI tolerance
Assessment of the participant's GI tolerance in the opinion of the investigator
Change in compliance with study product consumption
Assessed via participant diary regarding study product intake
Change in PI impression of study product intake
Assessment of the participant's compliance in the opinion of the investigator
Change in product acceptability
Assessed via a product acceptability questionnaire completed by the participant/parent
Change in dietary intake
Assessed via 24-hour recall and low protein food frequency questionnaire
Change in nutritional biochemistry
Assessed via venous blood sample of approximately 7ml

Secondary Outcome Measures

Full Information

First Posted
August 3, 2023
Last Updated
August 15, 2023
Sponsor
Vitaflo International, Ltd
Collaborators
Birmingham Women's and Children's NHS Foundation Trust
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1. Study Identification

Unique Protocol Identification Number
NCT05995717
Brief Title
Evaluation of PKU UP
Acronym
PKU UP
Official Title
An Acceptability Study to Evaluate the Compliance, Gastrointestinal Tolerance, Palatability and Metabolic Control of Children With Phenylketonuria (PKU) When Using PKU UP (a Food for Special Medical Purposes) as Part of Dietary Management.
Study Type
Interventional

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Not yet recruiting
Study Start Date
October 16, 2023 (Anticipated)
Primary Completion Date
April 16, 2025 (Anticipated)
Study Completion Date
June 16, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vitaflo International, Ltd
Collaborators
Birmingham Women's and Children's NHS Foundation Trust

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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.
Detailed Description
PKU is a rare inborn error of metabolism with a prevalence of 1 in 10,000 in the UK population. The mainstay of treatment for most individuals is dietary therapy with a strict protein-restricted diet. We propose to recruit children with a diagnosis of PKU aged 1-10 years. The study product, PKU UP, is a food for special medical purposes (FSMP), as defined by the Delegated Act EU 2016/128, used for the dietary management of Phenylketonuria (PKU). The study will be investigating the acceptability of PKU UP, as defined by the Advisory Committee on Borderline Substances (ACBS). This includes the following: participant adherence to recommended intakes gastrointestinal symptoms evaluations of palatability. Each participant will be on the study for 26 weeks. This includes evaluation part 1 and evaluation part 2: Part 1 consists of a 12 week period where participants introduce the study product into their diet. Gastrointestinal tolerance, metabolic control, compliance, product acceptability and dietary quality will be evaluated. Part 2 consists of a 14 week period where participants continue to take the study product for a longer assessment of growth and nutritional status. Data collection will be performed using paper and/or electronic Case Report Forms completed by the investigators at the baseline, evaluation periods and end of study visits. There will also be three questionnaires completed by the parents/guardians over the course of the study, in relation to protein substitute intake, gastrointestinal tolerance, and product acceptability.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
PKU
Keywords
Phenylketonuria, PKU, Glyco-Macro-Peptide, GMP, amino acid, AA, protein substitute, PS, United Kingdom, UK, children, PKU UP, Glycomacropeptide

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
15 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
PKU UP
Arm Type
Experimental
Arm Description
All participants will be assessed by their dietitian and prescribed an appropriate amount of the study product, PKU UP, to manage their phenylketonuria.
Intervention Type
Dietary Supplement
Intervention Name(s)
PKU UP
Intervention Description
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).
Primary Outcome Measure Information:
Title
Change in phe level between baseline and visit 3
Description
Change in phe level as measured by dried blood spot testing
Time Frame
26 weeks
Title
Change in tyrosine level between baseline and visit 3
Description
Change in tyrosine level as measured by dried blood spot testing
Time Frame
26 weeks
Title
Change in PI impression of metabolic control
Description
Assessment of the participant's metabolic control in the opinion of the investigator
Time Frame
Week 0, week 6, week 12, week 26
Title
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.
Description
Assessed via anthropometry measurements. Participants will be weighed and measured for growth monitoring.
Time Frame
Week 0, week 12, week 26
Title
Change in GI tolerance (participant)
Description
Gastrointestinal tolerance assessed using the PedsQL Gastrointestinal Symptoms Scale completed by the participants
Time Frame
Week 0, week 6, week 12, week 26
Title
Change in PI impression of GI tolerance
Description
Assessment of the participant's GI tolerance in the opinion of the investigator
Time Frame
Week 0, week 6, week 12, week 26
Title
Change in compliance with study product consumption
Description
Assessed via participant diary regarding study product intake
Time Frame
Week 0, week 6, week 12, week 26
Title
Change in PI impression of study product intake
Description
Assessment of the participant's compliance in the opinion of the investigator
Time Frame
Week 0, week 6, week 12, week 26
Title
Change in product acceptability
Description
Assessed via a product acceptability questionnaire completed by the participant/parent
Time Frame
Week 0, week 12, week 26
Title
Change in dietary intake
Description
Assessed via 24-hour recall and low protein food frequency questionnaire
Time Frame
Week 0, week 12, week 26
Title
Change in nutritional biochemistry
Description
Assessed via venous blood sample of approximately 7ml
Time Frame
Week 0, week 26

10. Eligibility

Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
10 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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 are anticipated to be able to take at least half of their daily protein equivalent requirement from PKU UP. Willingly given, written, informed consent from the parent(s)/guardian(s). Willingly given, written assent by the participant (if appropriate). Exclusion Criteria: 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).
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Binyam Tesfaye
Phone
0151 709 9020
Email
Binyam.Tesfaye@Vitaflo.co.uk
First Name & Middle Initial & Last Name or Official Title & Degree
Erika Brennan
Phone
0151 709 9020
Email
ClinicalTrialsTeam@Vitaflo.co.uk
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Anita MacDonald
Organizational Affiliation
Birmingham Women's and Children's NHS Foundation Trust
Official's Role
Principal Investigator
Facility Information:
Facility Name
Royal Belfast Hospital for Sick Children
City
Belfast
ZIP/Postal Code
BT12 6BE
Country
United Kingdom
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Anne Grimsley
Phone
07936 877 113
Email
Anne.Grimsley@BelfastTrust.HSCNI.net
Facility Name
Birmingham Children's Hospital
City
Birmingham
ZIP/Postal Code
B4 6NH
Country
United Kingdom
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Anita MacDonald
Phone
0121 333 8024
Email
Anita.MacDonald@NHS.net
Facility Name
University Hospitals Bristol and Weston NHS Foundation Trust
City
Bristol
ZIP/Postal Code
BS2 8BJ
Country
United Kingdom
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Camille Newby
Phone
0117 342 8011
Email
Camille.Newby@UHBW.NHS.UK
Facility Name
Great Ormond Street Hospital for Children NHS Foundation Trust
City
London
ZIP/Postal Code
WC1N 3JH
Country
United Kingdom
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Louise van Dorp
Phone
0207 813 8549
Email
Louise.VanDorp@GOSH.NHS.UK

12. IPD Sharing Statement

Plan to Share IPD
No

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Evaluation of PKU UP

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