Evaluation of PKU Explore

Phenylketonuria Clinical Trial

— 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.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03168399
• Other study ID #: MCT-PKUE-2016-12-05
• Secondary ID: 219119
• Status: Completed
• Phase: N/A
• Start date: June 8, 2017
• Est. completion date: March 15, 2018

Study information

• Verified date: February 2024
• Source: Vitaflo International, Ltd
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 22
• Est. completion date: March 15, 2018
• Est. primary completion date: March 15, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Months to 5 Years

Eligibility

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).

Related Conditions & MeSH terms

• Inborn Errors of Metabolism
• Metabolism, Inborn Errors
• Phenylketonuria
• Phenylketonurias

Intervention

Dietary Supplement:
• PKU Explore
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).

Locations

Country	Name	City	State
United Kingdom	Bradford Teaching Hospitals NHS Foundation Trust	Bradford	West Yorkshire
United Kingdom	Birmingham Women's and Children's NHS Foundation Trust	Burringham	West Midlands
United Kingdom	NHS Greater Glasgow and Clyde	Glasgow	Lanarkshire

Sponsors (2)

Lead Sponsor	Collaborator
Vitaflo International, Ltd	Birmingham Women's and Children's NHS Foundation Trust

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Product compliance daily diary	Quantitative assessments from subject questionnaires that allow evaluation of compliance with the study product, i.e. actual versus prescribed intake.	Days 1-28
Primary	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.	Days 1-7 and 22-28
Primary	Ease of use questionnaire	Qualitative assessment from subject questionnaire that allows evaluation of the ease of use of the study product.	Day 29
Primary	Weekly phenylalanine control	Collection of quantitative data regarding phenylalanine control using routine biochemical testing	Days 1-28