PKUPKU-weaningNCT03913156
Feeding Patterns and Practices in Young Children With PKU During the First Two Years of Life
A study testing B2970 Metabolic Texture Modified Protein Substitute in people with PKU, run by Nutricia Research at 3 sites in United Kingdom.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 5 years
- Conditions the study is for
- PKU
- What is being tested?
- B2970 Metabolic Texture Modified Protein Substitute
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 46 participants
- Where
- 3 sitesUnited Kingdom
- Study dates
- 15 Mar 2010to Dec 2016
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 5 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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)
Who cannot join
- 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
Trial locations and contacts
3 sites in 1 country.
United Kingdom3 sites
Birmingham Children's Hospital
Birmingham, B4 6NH
Bradford Teaching Hospitals NHS Foundation Trust, St Luke's Hospital
Bradford, BD5 0NA
JB Russell House, Gartnavel Royal Hospital
Glasgow, G12 OXH
About this study
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
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.
Read the 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.
- Conditions
- PKU
- Being tested
- B2970 Metabolic Texture Modified Protein Substitute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
B2970 Metabolic Texture Modified Protein SubstituteOther
Also called PKU gel
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Other: B2970 Metabolic Texture Modified Protein Substitute |
What is being measured
The primary outcome is the main question the study is designed to answer.
Feeding patterns- product intake
Actual intake of product and prescribed amount of product [# sachets] will be recorded as documented in diaries.
Measured: 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.
Measured: 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]
Measured: 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]
Measured: up to 2 years
Occurence of Adverse Events [ Safety and tolerability]
Occurence of Adverse Events
Measured: 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)
Measured: 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)
Measured: up to 2 years
Blood Phenylalanine control: Phe levels
Phe levels [umol/L]
Measured: up to 2 years
Blood Tyrosine levels
Tyrosine levels [umol/L]
Measured: 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]
Measured: up to 2 years
Intake of energy and nutritients: Weaning
Intakes of energy and nutrients from the weaning diet: descriptive information, intakes [g/day]
Measured: 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]
Measured: up to 2 years
Anthropometry: weight
Weight [g]
Measured: up to 2 years
Anthropometry: length
Length [cm]
Measured: up to 2 years
Secondary outcomes (4)
Weaning per group (with our without protein substitute) - descriptive
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]
Measured: up to 2 years
Anxiety levels of parents - Beck's Anxiety inventory
The Beck's Anxiety Inventory, scores on domains [0 not at all - 3 severely]
Measured: up to 2 years
Coping mechanisms of parents by questionnaire
Coping Health Inventory for Parents (scores on domains coping behaviour [3 extremely helpful -0 not helpful]
Measured: up to 2 years
Parent experience of Feeding via questionnaire
Feeding time Parent Questionnaire, experience of parents of their child's feeding time, scores on various domains [0-7]
Measured: up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Apr 2019
- Study start15 Mar 2010
- Primary completionApr 2013
- Study completionDec 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PKUStudies of the same condition, open and under way.
- More trials run by Nutricia ResearchThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 46 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nutricia Research
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Anita MacDonald, Principal investigator, Birmingham Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03913156
- Sponsor's protocol ID
- B2970
- Acronym
- PKU-weaning
- Status last verified
- Jun 2020
- First submitted
- 28 Aug 2018
- Last updated
- 19 Jun 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Jun 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03913156
- Acronym / sponsor ID
- PKU-weaning / B2970
- Conditions
- PKU
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 46 participants (actual)
- Lead sponsor
- Nutricia ResearchIndustry (pharma, biotech, devices)
- Study start
- 15 Mar 2010 (actual)
- Primary completion
- Apr 2013 (actual)
- Study completion
- Dec 2016 (actual)
- First posted
- 12 Apr 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Part 1 | Other | Other: B2970 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. |
| Part 2 | Other | Other: B2970 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. |
Who may qualify
- Ages
- Up to 5 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- 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)
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| B2970 Metabolic Texture Modified Protein SubstituteAlso: PKU gelThe 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 | Part 1, Part 2 |
Primary outcome
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.
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.
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]
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]
Occurence of Adverse Events [ Safety and tolerability]
Time frame: up to 2 years
Occurence of Adverse Events
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)
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)
Blood Phenylalanine control: Phe levels
Time frame: up to 2 years
Phe levels [umol/L]
Blood Tyrosine levels
Time frame: up to 2 years
Tyrosine levels [umol/L]
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]
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]
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]
Anthropometry: weight
Time frame: up to 2 years
Weight [g]
Anthropometry: length
Time frame: up to 2 years
Length [cm]
4 secondary outcomes
Weaning per group (with our without protein substitute) - descriptive
Time frame: up to 2 years
Anxiety levels of parents - Beck's Anxiety inventory
Time frame: up to 2 years
Coping mechanisms of parents by questionnaire
Time frame: up to 2 years
Parent experience of Feeding via questionnaire
Time frame: up to 2 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Jun 2020.
- 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;Link provided by the sponsor.
- How Does Feeding Development and Progression onto Solid Foods in PKU Compare with Non-PKU Children During Weaning?; Nutrients 2019, 11(3), 529Link provided by the sponsor.
- Mealtime Anxiety and Coping Behaviour in Parents and Children During Weaning in PKU: A Case-Control Study; Nutrients. 2019 Dec; 11(12): 2857Link provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Anita MacDonald principal investigator
Birmingham Children's Hospital
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing B2970 Metabolic Texture Modified Protein Substitute in people with PKU, run by Nutricia Research at 3 sites in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Jun 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Feeding patterns- product intake (time frame: up to 2 years). The study lists 14 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Jun 2020.