search
Back to results

PKU Low Calorie Drink Study

Primary Purpose

Phenylketonurias, Hyperphenylalaninaemia

Status
Not yet recruiting
Phase
Not Applicable
Locations
Study Type
Interventional
Intervention
Low calorie protein substitute
Sponsored by
Nutricia UK Ltd
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional supportive care trial for Phenylketonurias

Eligibility Criteria

3 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Male or female
  • 3 years of age or above
  • Diagnosed with classical or variant type phenylketonuria (PKU), or hyperphenylalaninemia (HPA)
  • Have been compliant in taking at least one amino acid-based, Phe-free protein substitute, providing at least 20g protein equivalent daily, for at least 1 month prior to study commencement
  • Have a prescribed daily Phe allowance
  • Written informed consent from patient, or from parent / carer if applicable

Exclusion Criteria:

  • Pregnant or lactating
  • Requiring enteral tube or parenteral nutrition
  • Major hepatic or renal dysfunction
  • Participation in other studies within 1 month prior to entry to this study
  • Allergy to any of the study product ingredients
  • Investigator concern around the patient being underweight (e.g.: BMI lower than 18.5kg/m2 for adults) and/or having an eating disorder
  • Investigator concern around willingness/ability of patient or parent/carer to comply with protocol requirements

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    Low calorie protein substitute

    Arm Description

    Single arm designed, 3 day baseline, 28 day on the lower calorie amino acid based liquid protein substitute.

    Outcomes

    Primary Outcome Measures

    Compliance
    Questionnaire on amounts offered and amounts actually consumed, compared to recommended amount. Amounts (values) are recorded

    Secondary Outcome Measures

    Acceptability: Tick-box questionnaire
    Tick-box questionnaire on overall liking and acceptability of product (ie dislike a lot, dislike moderately, neutral, like moderately, like a lot)
    Blood phenylalanine and other amino acid levels
    Blood spot test.
    Gastro-intestinal tolerance
    Questionnaire detailing any GI symptoms, severity (none, mild, moderate, severe) and change from usual.
    Nutrient intake
    24h dietary recall.
    Anthropometry
    Measurements of height (m) and weight (kg) at baseline and end of study.

    Full Information

    First Posted
    August 29, 2018
    Last Updated
    April 19, 2023
    Sponsor
    Nutricia UK Ltd
    search

    1. Study Identification

    Unique Protocol Identification Number
    NCT04272736
    Brief Title
    PKU Low Calorie Drink Study
    Official Title
    Evaluating the Compliance, Acceptability, Safety and Tolerance of a Lower Calorie Protein Substitute for the Dietary Management of Phenylketonuria in Children and Adults - a Pilot Study
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    April 2023
    Overall Recruitment Status
    Not yet recruiting
    Study Start Date
    December 1, 2023 (Anticipated)
    Primary Completion Date
    December 31, 2023 (Anticipated)
    Study Completion Date
    December 31, 2023 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Nutricia UK Ltd

    4. Oversight

    Studies a U.S. FDA-regulated Drug Product
    No
    Studies a U.S. FDA-regulated Device Product
    No
    Data Monitoring Committee
    No

    5. Study Description

    Brief Summary
    This study will evaluate the compliance, acceptability, gastrointestinal (GI) tolerance and safety of a lower calorie amino acid based liquid protein substitute for patients with Phenylketonuria (PKU) or hyperphenylalaninemia (HPA).

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Phenylketonurias, Hyperphenylalaninaemia

    7. Study Design

    Primary Purpose
    Supportive Care
    Study Phase
    Not Applicable
    Interventional Study Model
    Single Group Assignment
    Masking
    None (Open Label)
    Allocation
    N/A
    Enrollment
    30 (Anticipated)

    8. Arms, Groups, and Interventions

    Arm Title
    Low calorie protein substitute
    Arm Type
    Experimental
    Arm Description
    Single arm designed, 3 day baseline, 28 day on the lower calorie amino acid based liquid protein substitute.
    Intervention Type
    Dietary Supplement
    Intervention Name(s)
    Low calorie protein substitute
    Intervention Description
    The evaluated dietary supplement is a lower calorie amino acid based liquid protein substitute designed for patients with PKU.
    Primary Outcome Measure Information:
    Title
    Compliance
    Description
    Questionnaire on amounts offered and amounts actually consumed, compared to recommended amount. Amounts (values) are recorded
    Time Frame
    Daily for 31 days
    Secondary Outcome Measure Information:
    Title
    Acceptability: Tick-box questionnaire
    Description
    Tick-box questionnaire on overall liking and acceptability of product (ie dislike a lot, dislike moderately, neutral, like moderately, like a lot)
    Time Frame
    Recorded at baseline and end of intervetnion (day 31)
    Title
    Blood phenylalanine and other amino acid levels
    Description
    Blood spot test.
    Time Frame
    Recorded at baselien and end of intervention (day 31)
    Title
    Gastro-intestinal tolerance
    Description
    Questionnaire detailing any GI symptoms, severity (none, mild, moderate, severe) and change from usual.
    Time Frame
    recorded at baseline, beginning of intervention and end of intervention(12 out of 31 days in total)
    Title
    Nutrient intake
    Description
    24h dietary recall.
    Time Frame
    Recorded at basleine and end of intervention (day 31)
    Title
    Anthropometry
    Description
    Measurements of height (m) and weight (kg) at baseline and end of study.
    Time Frame
    Recorded at baseline and end of intervention (day 31)

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    3 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion Criteria: Male or female 3 years of age or above Diagnosed with classical or variant type phenylketonuria (PKU), or hyperphenylalaninemia (HPA) Have been compliant in taking at least one amino acid-based, Phe-free protein substitute, providing at least 20g protein equivalent daily, for at least 1 month prior to study commencement Have a prescribed daily Phe allowance Written informed consent from patient, or from parent / carer if applicable Exclusion Criteria: Pregnant or lactating Requiring enteral tube or parenteral nutrition Major hepatic or renal dysfunction Participation in other studies within 1 month prior to entry to this study Allergy to any of the study product ingredients Investigator concern around the patient being underweight (e.g.: BMI lower than 18.5kg/m2 for adults) and/or having an eating disorder Investigator concern around willingness/ability of patient or parent/carer to comply with protocol requirements
    Central Contact Person:
    First Name & Middle Initial & Last Name or Official Title & Degree
    Gary Hubbard, PhD
    Phone
    07738024720
    Email
    gary.hubbard@nutricia.com

    12. IPD Sharing Statement

    Plan to Share IPD
    No

    Learn more about this trial

    PKU Low Calorie Drink Study

    We'll reach out to this number within 24 hrs