Clinical trial NCT03913156: Feeding Patterns and Practices in Young Children With PKU During the First Two Years of Life
Official 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: PKU
Lead sponsor: Nutricia Research
Sponsor class: INDUSTRY
First posted: 2019-04-12
Enrollment: 46
Sex: ALL
Maximum age (years): 5
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: OTHER

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.

Eligibility criteria: 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

Primary outcomes:
measure: Feeding patterns- product intake
timeFrame: up to 2 years
measure: Feeding patterns - intake other than study product
timeFrame: up to 2 years
measure: Feeding practices and difficulties by questionnaire; position
timeFrame: up to 2 years
measure: Feeding practices and difficulties by questionnaire; self feeding
timeFrame: up to 2 years
measure: Occurence of Adverse Events [ Safety and tolerability]
timeFrame: up to 2 years
measure: Gastro-Intestinal symptoms
timeFrame: up to 2 years
measure: Product Acceptability yes/no
timeFrame: up to 2 years
measure: Blood Phenylalanine control: Phe levels
timeFrame: up to 2 years
measure: Blood Tyrosine levels
timeFrame: up to 2 years
measure: Intake of energy and nutritients: Study product
timeFrame: up to 2 years
measure: Intake of energy and nutritients: Weaning
timeFrame: up to 2 years
measure: Intake of energy and nutritients: Weaning
timeFrame: up to 2 years
measure: Anthropometry: weight
timeFrame: up to 2 years
measure: Anthropometry: length
timeFrame: up to 2 years
Secondary outcomes:
measure: Weaning per group (with our without protein substitute) - descriptive
timeFrame: up to 2 years
measure: Anxiety levels of parents - Beck's Anxiety inventory
timeFrame: up to 2 years
measure: Coping mechanisms of parents by questionnaire
timeFrame: up to 2 years
measure: Parent experience of Feeding via questionnaire
timeFrame: up to 2 years
Other outcomes:
Locations:
city: Birmingham
country: United Kingdom
facility: Birmingham Children's Hospital
city: Bradford
country: United Kingdom
facility: Bradford Teaching Hospitals NHS Foundation Trust, St Luke's Hospital
city: Glasgow
country: United Kingdom
facility: JB Russell House, Gartnavel Royal Hospital
Study officials:
affiliation: Birmingham Children's Hospital
name: Anita MacDonald
role: PRINCIPAL_INVESTIGATOR
References: