Clinical Trial: Evaluation of PKU Start

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Study to Evaluate the Acceptability of a New Phenylalanine Free Infant Formula for Use in the Dietary Management of Phenylketonuria in Infants From Birth to 2 Year of Age With Regard to Product Tole

Brief Summary: To evaluate the acceptability, tolerance and effect on metabolic control of PKU Start, a new Phe free protein substitute for the dietary management of PKU in infants from birth.

Detailed Summary:

This is an assessment of ten (10) infants who require a protein restricted diet that is low in Phe. Infants who routinely use a Phe free infant formula as part of their dietary therapy will be recruited for a 28-day assessment of PKU Start, to evaluate tolerance and acceptability.

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 Start based on individual requirements. Parents/carers will be asked to substitute their usual Phe-free infant formula with PKU Start for one (1) month. The sponsor will supply PKU Start free of charge.

Prior to starting PKU Start, parents/carers will be asked to record information about the infant's usual GI tolerance and feeding pattern for a period of three (3) days, to allow for comparison between their existing formula and PKU Start.

They will be asked to record information about the following:

Stools Vomiting and Spit-up Feed / Fluid Intake and Compliance Phenylalanine Levels Final Evaluation about the presentation of the product, ease of preparation and how PKU Start flowed through the teat of a bottle.


Sponsor: Vitaflo International, Ltd

Current Primary Outcome:

  • Product compliance daily diary [ Time Frame: Days 1-28 ]
    Quantitative assessments from subject questionnaires that allow evaluation of compliance with the study product, i.e. actual versus prescribed intake.
  • GI tolerance daily diary [ Time Frame: Days 1-28 ]
    Qualitative assessments from subject questionnaires that allow evaluation of the gastro-intestinal tolerance of the study product.
  • Ease of use questionnaire [ Time Frame: Day 29 ]
    Qualitative assessment from subject questionnaire that allows evaluation of the ease of use of the study product.
  • Daily phenylalanine control [ Time Frame: Days 1-28 ]
    Collection of quantitative data regarding phenylalanine control using routine biochemical testing


Original Primary Outcome: Same as current

Current Secondary Outcome:

Original Secondary Outcome:

Information By: Vitaflo International, Ltd

Dates:
Date Received: February 16, 2017
Date Started: March 6, 2017
Date Completion: May 2017
Last Updated: April 18, 2017
Last Verified: April 2017