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

NCT03913156 | Completed

• 1 other identifier: B2970
• Study Type: interventional
• Target: 46
• Locations: 1 country
• Sites: 3

Brief Summary

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.

Conditions

PKU

Outcome Measures

Primary Outcomes (14)

• 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 Outcomes (4)

• Weaning per group (with our without protein substitute) - descriptive

  up to 2 years

• Anxiety levels of parents - Beck's Anxiety inventory

  up to 2 years

• Coping mechanisms of parents by questionnaire

  up to 2 years

• Parent experience of Feeding via questionnaire

  up to 2 years

Study Arms (2)

Part 1

OTHER

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

Part 2

OTHER

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

Interventions

B2970 Metabolic Texture Modified Protein Substitute OTHER

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.

Also known as: PKU gel

Part 1; Part 2

Eligibility Criteria

• Age: Up to 5 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Nutricia Research: lead

Study Sites (3)

Birmingham Children's Hospital

Birmingham, B4 6NH, United Kingdom

Location

Bradford Teaching Hospitals NHS Foundation Trust, St Luke's Hospital

Bradford, BD5 0NA, United Kingdom

Location

JB Russell House, Gartnavel Royal Hospital

Glasgow, G12 OXH, United Kingdom

Location

Related 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 Officials

• Anita MacDonald

  Birmingham Children's Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 28, 2018
• First Posted: April 12, 2019
• Study Start: March 15, 2010
• Primary Completion: April 1, 2013
• Study Completion: December 1, 2016
• Last Updated: June 19, 2020

Record last verified: 2020-06

Locations

GB (3)