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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03913156
Recruitment Status : Completed
First Posted : April 12, 2019
Last Update Posted : August 21, 2019
Sponsor:
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
PKU Other: B2970 Metabolic Texture Modified Protein Substitute Not Applicable

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 46 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Other
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
Actual Study Start Date : March 15, 2010
Actual Primary Completion Date : April 2013
Actual Study Completion Date : December 2016

Arm Intervention/treatment
Part 1
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.
Other: B2970 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.
Other Name: PKU gel

Part 2
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.
Other: B2970 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.
Other Name: PKU gel




Primary Outcome Measures :
  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 Outcome Measures :
  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]



Information from the National Library of Medicine

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Ages Eligible for Study:   up to 5 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03913156


Locations
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United Kingdom
Birmingham Children's Hospital
Birmingham, United Kingdom, B4 6NH
Bradford Teaching Hospitals NHS Foundation Trust, St Luke's Hospital
Bradford, United Kingdom, BD5 0NA
JB Russell House, Gartnavel Royal Hospital
Glasgow, United Kingdom, G12 OXH
Sponsors and Collaborators
Nutricia Research
Investigators
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Principal Investigator: Anita MacDonald Birmingham Children’s Hospital

Additional Information:
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Responsible Party: Nutricia Research
ClinicalTrials.gov Identifier: NCT03913156    
Other Study ID Numbers: B2970
First Posted: April 12, 2019    Key Record Dates
Last Update Posted: August 21, 2019
Last Verified: August 2019

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No