PKU
Conditions
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.
Interventions
OTHERB2970 Metabolic Texture Modified Protein Substitute
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.
Sponsors
Nutricia Research
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
OTHER
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
No minimum to 5 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Anthropometry: length | up to 2 years | Length \[cm\] |
| Blood Tyrosine levels | up to 2 years | Tyrosine levels \[umol/L\] |
| Intake of energy and nutritients: Study product | up to 2 years | Intakes of energy and nutrients from the study product \[diet diary; #g product/day\] |
| Intake of energy and nutritients: Weaning | up to 2 years | Intakes of energy and nutrients from the weaning diet: descriptive information, intakes \[g/day\] |
| Anthropometry: weight | up to 2 years | Weight \[g\] |
| Feeding patterns- product intake | up 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 product | 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. |
| Feeding practices and difficulties by questionnaire; position | up to 2 years | Feeding practices entered by parents in diaries: the way the child has been fed will be assessed : categories \[sitting position e.g on lap, in chair etc\] |
| Feeding practices and difficulties by questionnaire; self feeding | up to 2 years | Feeding practices entered by parents in diaries; the way the child has been fed will be assessed : \[self-feeding; parent feeding\]; \[type of self feeding: bottle, finger; spoon, fork\] |
| Occurence of Adverse Events [ Safety and tolerability] | up to 2 years | Occurence of Adverse Events |
| Gastro-Intestinal symptoms | 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) |
| Product Acceptability yes/no | 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) |
| Blood Phenylalanine control: Phe levels | up to 2 years | Phe levels \[umol/L\] |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Anxiety levels of parents - Beck's Anxiety inventory | up to 2 years | The Beck's Anxiety Inventory, scores on domains \[0 not at all - 3 severely\] |
| Coping mechanisms of parents by questionnaire | up to 2 years | Coping Health Inventory for Parents (scores on domains coping behaviour \[3 extremely helpful -0 not helpful\] |
| Parent experience of Feeding via questionnaire | up to 2 years | Feeding time Parent Questionnaire, experience of parents of their child's feeding time, scores on various domains \[0-7\] |
| Weaning per group (with our without protein substitute) - descriptive | 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 \[parent questionnaires\] |
Countries
United Kingdom
Outcome results
None listed