Evaluation of PKU Start (PKU Start)

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.

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.

Study Overview

• Status: Completed
• Conditions: Phenylketonuria; Inborn Errors of Metabolism
• Intervention / Treatment: Dietary supplement: PKU Start

Detailed 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.

• Study Type: Interventional
• Enrollment (Actual): 10
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United Kingdom
  • Lanarkshire
    • Glasgow, Lanarkshire, United Kingdom, G51 4TF
      • NHS Greater Glasgow and Clyde
  • West Midlands
    • Birmingham, West Midlands, United Kingdom, B4 6NH
      • Birmingham Children's Hospital NHS Foundation Trust
  • West Yorkshire
    • Bradford, West Yorkshire, United Kingdom, BD5 0NA
      • Bradford Teaching Hospitals NHS Foundation Trust

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 4 weeks to 2 years (Child)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: Consumption of PKU Start; Daily feed, substituting the participant's normal phe-free formula for PKU Start.

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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
Ease of use questionnaire	Qualitative assessment from subject questionnaire that allows evaluation of the ease of use of the study product.	Day 29
GI tolerance daily diary	Qualitative assessments from subject questionnaires that allow evaluation of the gastro-intestinal tolerance of the study product.	Days 1-28
Daily phenylalanine control	Collection of quantitative data regarding phenylalanine control using routine biochemical testing	Days 1-28

Collaborators and Investigators

• Sponsor: Vitaflo International, Ltd
• Investigators: Principal Investigator: Anita MacDonald, Birmingham Women's and Children's NHS Foundation Trust

Study record dates

Study Major Dates

• Study Start (Actual): March 6, 2017
• Primary Completion (Actual): September 15, 2017
• Study Completion (Actual): September 15, 2017

Study Registration Dates

• First Submitted: February 16, 2017
• First Submitted That Met QC Criteria: February 16, 2017
• First Posted (Actual): February 23, 2017

Study Record Updates

• Last Update Posted (Actual): February 16, 2024
• Last Update Submitted That Met QC Criteria: February 15, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Keywords: Acceptability; Metabolic; Control; Metabolism; Phenylketonuria; PKU
• Additional Relevant MeSH Terms: Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Amino Acid Metabolism, Inborn Errors; Phenylketonurias; Metabolism, Inborn Errors
• Other Study ID Numbers: BCH-PKUSTART-032016-29; 209920 (Registry Identifier: IRAS); 17/NW/0035 (Other Identifier: UK REC)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No