NCT03168399

Evaluation of PKU Explore

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.

• Status: Completed
• Phase: N/A
• Study type: Interventional
• Enrollment: 22 (actual)
• Sponsor: Vitaflo International, Ltd · Industry
• Sex: All
• Age: 6 Months – 5 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Phenylketonuria
• Inborn Errors of Metabolism

Interventions

Type	Name	Description
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).

Timeline

• Start date: 2017-06-08
• Primary completion: 2018-03-15
• Completion: 2018-03-15
• First posted: 2017-05-30
• Last updated: 2024-02-16

Locations

3 sites across 1 country: United Kingdom