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Evaluation of PKU Explore (PKU Explore)

Primary Purpose

Phenylketonuria, Inborn Errors of Metabolism

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

About this trial

This is an interventional treatment trial for Phenylketonuria focused on measuring Phenylketonuria, PKU, Metabolism, Metabolic, Control, Acceptability

Eligibility Criteria

6 Months - 5 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

i. A diagnosis of PKU on new-born screening requiring a low protein diet and Phe-free L-amino acid supplements.

ii. Aged between 6 months and 5 years.

iii. Already taking a second stage concentrated protein substitute as part of their PKU management OR is at the stage in their PKU management when a second stage concentrated protein substitute is recommended to commence.

iv. Willingly given, written, informed consent from parent/guardian.

v. Willingly given, written assent (if appropriate).

Exclusion Criteria:

i. Diagnosis of persistent hyperphenylalaninaemia, or mild PKU not requiring dietary treatment with a low protein diet and Phe-free L-amino acid supplements.

ii. Diagnosis of a concurrent condition which may adversely affect developmental progression and feeding ability.

iii. Participation in any other clinical trial/acceptability study.

iv. Any serious medical precluding the study intervention.

v. 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).

Sites / Locations

  • NHS Greater Glasgow and Clyde
  • Birmingham Women's and Children's NHS Foundation Trust
  • Bradford Teaching Hospitals NHS Foundation Trust

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Consumption of PKU Explore

Arm Description

Daily feed, substituting the participant's normal phe-free protein substitute for PKU Explore.

Outcomes

Primary Outcome Measures

Product compliance daily diary
Quantitative assessments from subject questionnaires that allow evaluation of compliance with the study product, i.e. actual versus prescribed intake.
GI tolerance daily diary assessing change
Qualitative assessments from subject questionnaires that allow evaluation of any change in the gastro-intestinal tolerance of the study product.
Ease of use questionnaire
Qualitative assessment from subject questionnaire that allows evaluation of the ease of use of the study product.
Weekly phenylalanine control
Collection of quantitative data regarding phenylalanine control using routine biochemical testing

Secondary Outcome Measures

Full Information

First Posted
May 24, 2017
Last Updated
April 4, 2018
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
NCT03168399
Brief Title
Evaluation of PKU Explore
Acronym
PKU Explore
Official Title
A Study to Evaluate the Acceptability of PKU Explore, a Renovated Phenylalanine Free Second Stage Protein Substitute for Use in the Dietary Management of Phenylketonuria in Infants From 6 Months to 5 Years of Age With Regard to Product Tolerance and Adherence.
Study Type
Interventional

2. Study Status

Record Verification Date
April 2018
Overall Recruitment Status
Completed
Study Start Date
June 8, 2017 (Actual)
Primary Completion Date
March 15, 2018 (Actual)
Study Completion Date
March 15, 2018 (Actual)

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
To evaluate the acceptability, tolerance and effect on metabolic control of PKU Explore, a renovated Phe free protein substitute for the dietary management of PKU in children from 6 months to 5 years.
Detailed Description
This is a prospective, acceptability study to evaluate the gastrointestinal tolerance, palatability and adherence of PKU explore in a four-week period for 15 participants aged between 6 months and 5 years for the dietary management of PKU. The outcome of this assessment will be used in a submission to the regulatory authorities, Advisory Committee on Borderline Substances (ACBS), for PKU Start to become reimbursable on prescription in the UK. The participant's dietitian will advise on an appropriate amount of PKU Explore based on individual requirements. Parents/carers will be asked to substitute their usual Phe-free protein substitute with PKU Explore for one (1) month. The sponsor will supply PKU Explore free of charge. Parents/guardians will be asked to record information about the following: Daily PKU Explore intake Stools Vomiting and Spit-up Feed / Fluid Intake Compliance Phenylalanine Levels (from their routine blood spot testing) Final Evaluation about the presentation of the product, ease of preparation and use.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Phenylketonuria, Inborn Errors of Metabolism
Keywords
Phenylketonuria, PKU, Metabolism, Metabolic, Control, Acceptability

7. Study Design

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

8. Arms, Groups, and Interventions

Arm Title
Consumption of PKU Explore
Arm Type
Experimental
Arm Description
Daily feed, substituting the participant's normal phe-free protein substitute for PKU Explore.
Intervention Type
Dietary Supplement
Intervention Name(s)
PKU Explore
Intervention Description
PKU Explore is an unflavoured, powdered, phenylalanine-free, protein substitute, containing essential and non-essential amino acids, carbohydrate, sugar, vitamins, minerals, trace elements and the long chain polyunsaturated fatty acids (LCPs); arachidonic acid (AA) and docosahexaenoic acid (DHA).
Primary Outcome Measure Information:
Title
Product compliance daily diary
Description
Quantitative assessments from subject questionnaires that allow evaluation of compliance with the study product, i.e. actual versus prescribed intake.
Time Frame
Days 1-28
Title
GI tolerance daily diary assessing change
Description
Qualitative assessments from subject questionnaires that allow evaluation of any change in the gastro-intestinal tolerance of the study product.
Time Frame
Days 1-7 and 22-28
Title
Ease of use questionnaire
Description
Qualitative assessment from subject questionnaire that allows evaluation of the ease of use of the study product.
Time Frame
Day 29
Title
Weekly phenylalanine control
Description
Collection of quantitative data regarding phenylalanine control using routine biochemical testing
Time Frame
Days 1-28

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Months
Maximum Age & Unit of Time
5 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: i. A diagnosis of PKU on new-born screening requiring a low protein diet and Phe-free L-amino acid supplements. ii. Aged between 6 months and 5 years. iii. Already taking a second stage concentrated protein substitute as part of their PKU management OR is at the stage in their PKU management when a second stage concentrated protein substitute is recommended to commence. iv. Willingly given, written, informed consent from parent/guardian. v. Willingly given, written assent (if appropriate). Exclusion Criteria: i. Diagnosis of persistent hyperphenylalaninaemia, or mild PKU not requiring dietary treatment with a low protein diet and Phe-free L-amino acid supplements. ii. Diagnosis of a concurrent condition which may adversely affect developmental progression and feeding ability. iii. Participation in any other clinical trial/acceptability study. iv. Any serious medical precluding the study intervention. v. 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).
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
NHS Greater Glasgow and Clyde
City
Glasgow
State/Province
Lanarkshire
ZIP/Postal Code
G51 4TF
Country
United Kingdom
Facility Name
Birmingham Women's and Children's NHS Foundation Trust
City
Burringham
State/Province
West Midlands
ZIP/Postal Code
B4 6NH
Country
United Kingdom
Facility Name
Bradford Teaching Hospitals NHS Foundation Trust
City
Bradford
State/Province
West Yorkshire
ZIP/Postal Code
BD5 0NA
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
Undecided

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Evaluation of PKU Explore

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