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

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

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

Conditions
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PKU

Intervention / Treatment
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* Other: B2970 Metabolic Texture Modified Protein Substitute


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Maximum Age: 5 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

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


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: Part 1<br>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<br>* 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.<br>* Other names: PKU gel;|
| Other: Part 2<br>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<br>* 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.<br>* Other names: PKU gel;|

What is the study measuring?
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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 |