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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

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

NCT ID

NCT03168399

First Posted

May 24, 2017

Last Updated

April 4, 2018

Sponsor

Vitaflo International, Ltd

Collaborators

Birmingham Women's and Children's NHS Foundation Trust

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

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

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

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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