Evaluation of PKU Start

Phenylketonuria Clinical Trial

— PKU Start  

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 Tolerance and Adherence.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03058848
• Other study ID #: BCH-PKUSTART-032016-29
• Secondary ID: 20992017/NW/0035
• Status: Completed
• Phase: N/A
• Start date: March 6, 2017
• Est. completion date: September 15, 2017

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 Start, a new Phe free protein substitute for the dietary management of PKU in infants from birth.

Description:

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 10
• Est. completion date: September 15, 2017
• Est. primary completion date: September 15, 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Weeks to 2 Years

Eligibility

Inclusion Criteria:

1. A diagnosis of classical or severe PKU on new-born screening (For the purposes of this study, 'severe' is defined as phe concentrations between 120 and 600 µmol/L at diagnosis)

2. Taking a minimum of one (1) feed of a Phe-free infant formula

3. A minimum period of four (4) weeks from the time of diagnosis to initial approach to parents

Exclusion Criteria:

1. Diagnosed with mild PKU or hyperphenylalaninaemia (For the purposes of this study, 'mild' is defined as phe concentrations between 120 and 600 µmol/L at diagnosis)

2. Diagnosis of a congenital condition

Related Conditions & MeSH terms

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

Intervention

Dietary Supplement:
• PKU Start
PKU Start is a powdered, phenylalanine-free, infant formula, containing essential and non-essential amino acids, carbohydrate, fat, vitamins, minerals, trace elements and long chain polyunsaturated fatty acids (LCPs); Arachidonic acid (AA) and Docosahexaenoic acid (DHA). It is suitable for use from birth. The recommended amount of the product for each participant will be determined and prescribed by a dietitian.

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Vitaflo International, Ltd

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	Qualitative assessments from subject questionnaires that allow evaluation of the gastro-intestinal tolerance of the study product.	Days 1-28
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	Daily phenylalanine control	Collection of quantitative data regarding phenylalanine control using routine biochemical testing	Days 1-28