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Completed

NCT Number: NCT03913156

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

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.

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Key information

Conditions

PKU

Age range

Up to 5 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Birmingham Children's Hospital, Birmingham, United Kingdom

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

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.

Other names: PKU gel

Primary outcomes

  1. Feeding patterns- product intake

    Time frame: up to 2 years

    Actual intake of product and prescribed amount of product [# sachets] will be recorded as documented in diaries.

  2. Feeding patterns - intake other than study product

    Time frame: up to 2 years

    Intakes of standard formulas / breast-milk and normal foods [mL] or [g/day] and [frequency] of breastfeeding, as documented in diaries.

  3. Feeding practices and difficulties by questionnaire; position

    Time frame: up to 2 years

    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]

  4. Feeding practices and difficulties by questionnaire; self feeding

    Time frame: up to 2 years

    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]

  5. Occurence of Adverse Events [ Safety and tolerability]

    Time frame: up to 2 years

    Occurence of Adverse Events

  6. Gastro-Intestinal symptoms

    Time frame: up to 2 years

    Occurence of GI symptoms [yes/no] for:

    • Mouth Ulcers
    • Tummy upset
    • Harder poos
    • Constipation
    • Softer poos
    • Vomiting
    • Colic
    • Other (free entry)

  7. Product Acceptability yes/no

    Time frame: up to 2 years

    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)

  8. Blood Phenylalanine control: Phe levels

    Time frame: up to 2 years

    Phe levels [umol/L]

  9. Blood Tyrosine levels

    Time frame: up to 2 years

    Tyrosine levels [umol/L]

  10. Intake of energy and nutritients: Study product

    Time frame: up to 2 years

    Intakes of energy and nutrients from the study product [diet diary; #g product/day]

  11. Intake of energy and nutritients: Weaning

    Time frame: up to 2 years

    Intakes of energy and nutrients from the weaning diet: descriptive information, intakes [g/day]

  12. Intake of energy and nutritients: Weaning

    Time frame: up to 2 years

    Intakes of energy and nutrients from the weaning diet: descriptive information, intakes [# meals or snacks/day]

  13. Anthropometry: weight

    Time frame: up to 2 years

    Weight [g]

  14. Anthropometry: length

    Time frame: up to 2 years

    Length [cm]

Secondary outcomes

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

    Time frame: up to 2 years

    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]

  2. Anxiety levels of parents - Beck's Anxiety inventory

    Time frame: up to 2 years

    The Beck's Anxiety Inventory, scores on domains [0 not at all - 3 severely]

  3. Coping mechanisms of parents by questionnaire

    Time frame: up to 2 years

    Coping Health Inventory for Parents (scores on domains coping behaviour [3 extremely helpful -0 not helpful]

  4. Parent experience of Feeding via questionnaire

    Time frame: up to 2 years

    Feeding time Parent Questionnaire, experience of parents of their child's feeding time, scores on various domains [0-7]

Sponsors and collaborators

Lead sponsor

Nutricia Research

Industry

Registry information

Official study title

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

Acronym: PKU-weaning

Important dates

Study start
2010
Primary completion
2013
Study completion
2016
First posted
Apr 12, 2019
Registry last updated
Jun 19, 2020

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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