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

Not Applicable
Completed
Conditions
PKU
Interventions
Other: B2970 Metabolic Texture Modified Protein Substitute
Registration Number
NCT03913156
Lead Sponsor
Nutricia Research
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.

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.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
46
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 & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Part 2B2970 Metabolic Texture Modified Protein SubstitutePart 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.
Part 1B2970 Metabolic Texture Modified Protein SubstitutePart 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.
Primary Outcome Measures
NameTimeMethod
Intake of energy and nutritients: Study productup to 2 years

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

Feeding patterns- product intakeup to 2 years

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

Feeding patterns - intake other than study productup to 2 years

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

Feeding practices and difficulties by questionnaire; positionup to 2 years

Feeding practices entered by parents in diaries: the way the child has been fed will be assessed : categories \[sitting position e.g on lap, in chair etc\]

Feeding practices and difficulties by questionnaire; self feedingup to 2 years

Feeding practices entered by parents in diaries; the way the child has been fed will be assessed : \[self-feeding; parent feeding\]; \[type of self feeding: bottle, finger; spoon, fork\]

Occurence of Adverse Events [ Safety and tolerability]up to 2 years

Occurence of Adverse Events

Gastro-Intestinal symptomsup to 2 years

Occurence of GI symptoms \[yes/no\] for:

* Mouth Ulcers

* Tummy upset

* Harder poos

* Constipation

* Softer poos

* Vomiting

* Colic

* Other (free entry)

Product Acceptability yes/noup 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)

Blood Phenylalanine control: Phe levelsup to 2 years

Phe levels \[umol/L\]

Blood Tyrosine levelsup to 2 years

Tyrosine levels \[umol/L\]

Intake of energy and nutritients: Weaningup to 2 years

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

Anthropometry: weightup to 2 years

Weight \[g\]

Anthropometry: lengthup to 2 years

Length \[cm\]

Secondary Outcome Measures
NameTimeMethod
Weaning per group (with our without protein substitute) - descriptiveup 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 \[parent questionnaires\]

Anxiety levels of parents - Beck's Anxiety inventoryup to 2 years

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

Coping mechanisms of parents by questionnaireup to 2 years

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

Parent experience of Feeding via questionnaireup to 2 years

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

Trial Locations

Locations (3)

Birmingham Children's Hospital

🇬🇧

Birmingham, United Kingdom

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

🇬🇧

Bradford, United Kingdom

JB Russell House, Gartnavel Royal Hospital

🇬🇧

Glasgow, United Kingdom

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