Feeding Patterns and Practices in Young Children With PKU During the First Two Years of Life (PKU-weaning)

Feeding Patterns and Practices in Young Children With PKU During the First Two Years of Life: Acceptability and Efficacy of a New Protein Substitute

In this study the feeding patterns and practices at the introduction of a second stage phe-free protein substitute will be assessed in young children with PKU. This will be compared with the normal weaning process in healthy non-PKU age-matched children. In addition, tolerability and acceptability of the study product will be evaluated in a smaller group of subjects who have already been transferred onto a second stage protein substitute. The study is performed in 3 centers in the United Kingdom.

Study Overview

• Status: Completed
• Conditions: PKU
• Intervention / Treatment: Other: B2970 Metabolic Texture Modified Protein Substitute

Detailed Description

Part 1:

For subjects with PKU, the study product will be incorporated in the diet as the second stage protein substitute until the subjects are 2 years of age.

The healthy non-PKU age-matched subjects will not have the study product incorporated in their diet in order to be able to compare the weaning experience and nutritional intake of PKU subjects with non-PKU subjects. The healthy subjects will also be part of the study until the subjects are 2 years of age.

Part 2:

All PKU subjects will take the study product for 7 days.

• Study Type: Interventional
• Enrollment (Actual): 46
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United Kingdom
  • Birmingham, United Kingdom, B4 6NH
    • Birmingham Children's Hospital
  • Bradford, United Kingdom, BD5 0NA
    • Bradford Teaching Hospitals NHS Foundation Trust, St Luke's Hospital
  • Glasgow, United Kingdom, G12 OXH
    • JB Russell House, Gartnavel Royal Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 5 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Diagnosis of PKU identified on screening during the newborn period and requiring a phenylalanine free protein substitute
• Subjects who are about to commence weaning and require a concentrated phe-free protein substitute to meet their total protein requirements and are aged between 4 and 10 months (Part 1)
• Subjects aged under 5 years who have already been fully transferred onto a concentrated phe-free protein substitute and are willing to try the study product for 7 days (Part 2)
• Written informed consent obtained from subject or parents / caregiver
• May or may not have commenced weaning solids (i.e. using normal foods)

Exclusion Criteria:

• Presence of serious concurrent illness
• Investigator's uncertainty about the willingness or ability of the patient to comply with the protocol requirements
• Any child who has not commenced a second stage protein substitute and is over the age of 10 months
• Participation in any other studies involving investigational or marketed products concomitantly or within two weeks prior to entry into the study
• Any children having taken antibiotics over the previous 2 weeks leading up to the study

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: Part 1; Part 1 of the study will recruit subjects who are about to be introduced to a concentrated phe-free protein substitute (i.e. second stage protein substitute) for the first time.	Other: B2970 Metabolic Texture Modified Protein Substitute; The study product is a powdered phe-free protein substitute supplemented with long chain polyunsaturated fatty acids (LCP's), containing essential and non-essential amino acids, carbohydrate, fat, vitamins, minerals and trace elements and has been designed for use in the management of proven PKU in children aged 6 months to 5 years.; Other Names: PKU gel
Other: Part 2; Part 2 of the study will recruit subjects who have already been introduced to a concentrated phe-free protein substitute (i.e. have already transferred from phe-free infant formula onto a second stage protein substitute). This group will be included to evaluate the acceptability of the study product in patients who have already moved onto a second stage protein substitute.	Other: B2970 Metabolic Texture Modified Protein Substitute; The study product is a powdered phe-free protein substitute supplemented with long chain polyunsaturated fatty acids (LCP's), containing essential and non-essential amino acids, carbohydrate, fat, vitamins, minerals and trace elements and has been designed for use in the management of proven PKU in children aged 6 months to 5 years.; Other Names: PKU gel

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Feeding patterns- product intake	Actual intake of product and prescribed amount of product [# sachets] will be recorded as documented in diaries.	up to 2 years
Feeding patterns - intake other than study product	Intakes of standard formulas / breast-milk and normal foods [mL] or [g/day] and [frequency] of breastfeeding, as documented in diaries.	up to 2 years
Feeding practices and difficulties by questionnaire; position	Feeding practices entered by parents in diaries: the way the child has been fed will be assessed : categories [sitting position e.g on lap, in chair etc]	up to 2 years
Feeding practices and difficulties by questionnaire; self feeding	Feeding practices entered by parents in diaries; the way the child has been fed will be assessed : [self-feeding; parent feeding]; [type of self feeding: bottle, finger; spoon, fork]	up to 2 years
Occurence of Adverse Events [ Safety and tolerability]	Occurence of Adverse Events	up to 2 years
Gastro-Intestinal symptoms	Occurence of GI symptoms [yes/no] for: Mouth Ulcers; Tummy upset; Harder poos; Constipation; Softer poos; Vomiting; Colic; Other (free entry)	up to 2 years
Product Acceptability yes/no	Acceptability questions [yes/no] for: Refuses more than takes; Refuses as much as takes; Takes more than refuses; Spits out product; Puts spoon away; Holds in mouth but won't swallow; Closes mouth on feeding; Cries at beginning of feed; Cries at end of feed; Deliberately spills feed; Turns head away; Other (free entry)	up to 2 years
Blood Phenylalanine control: Phe levels	Phe levels [umol/L]	up to 2 years
Blood Tyrosine levels	Tyrosine levels [umol/L]	up to 2 years
Intake of energy and nutritients: Study product	Intakes of energy and nutrients from the study product [diet diary; #g product/day]	up to 2 years
Intake of energy and nutritients: Weaning	Intakes of energy and nutrients from the weaning diet: descriptive information, intakes [g/day]	up to 2 years
Intake of energy and nutritients: Weaning	Intakes of energy and nutrients from the weaning diet: descriptive information, intakes [# meals or snacks/day]	up to 2 years
Anthropometry: weight	Weight [g]	up to 2 years
Anthropometry: length	Length [cm]	up to 2 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Weaning per group (with our without protein substitute) - descriptive	Compare the weaning , nutritional intakes [g/day] and metabolic control of infants taking the study product, with historical controls of the same age and weaning stage whose diet did not incorporate a semi-solid second stage protein substitute during the same time period [parent questionnaires]	up to 2 years
Anxiety levels of parents - Beck's Anxiety inventory	The Beck's Anxiety Inventory, scores on domains [0 not at all - 3 severely]	up to 2 years
Coping mechanisms of parents by questionnaire	Coping Health Inventory for Parents (scores on domains coping behaviour [3 extremely helpful -0 not helpful]	up to 2 years
Parent experience of Feeding via questionnaire	Feeding time Parent Questionnaire, experience of parents of their child's feeding time, scores on various domains [0-7]	up to 2 years

Collaborators and Investigators

• Sponsor: Nutricia Research
• Investigators: Principal Investigator: Anita MacDonald, Birmingham Children's Hospital

Publications and helpful links

Helpful Links

• Growth, protein, and energy intake in children with PKU taking a weaning protein substitute in the first 2 years of life: a case-control study; Nutrients 2019, 11(3), 552;
• How Does Feeding Development and Progression onto Solid Foods in PKU Compare with Non-PKU Children During Weaning?; Nutrients 2019, 11(3), 529
• Mealtime Anxiety and Coping Behaviour in Parents and Children During Weaning in PKU: A Case-Control Study; Nutrients. 2019 Dec; 11(12): 2857

Study record dates

Study Major Dates

• Study Start (Actual): March 15, 2010
• Primary Completion (Actual): April 1, 2013
• Study Completion (Actual): December 1, 2016

Study Registration Dates

• First Submitted: August 28, 2018
• First Submitted That Met QC Criteria: April 10, 2019
• First Posted (Actual): April 12, 2019

Study Record Updates

• Last Update Posted (Actual): June 19, 2020
• Last Update Submitted That Met QC Criteria: June 17, 2020
• Last Verified: June 1, 2020

More Information

Terms related to this study

• Other Study ID Numbers: B2970

Drug and device information, study documents

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