Back to resultsGMP Drink for PKU Study
Primary Purpose
Phenylketonuria
Status
Completed
Phase
Not Applicable
Locations
United Kingdom
Study Type
Interventional
Intervention
Glycomacropeptide-based protein substitute
Sponsored by
About this trial
This is an interventional supportive care trial for Phenylketonuria
Eligibility Criteria
Inclusion Criteria:
- Male or female
- Over 3 years of age
- Diagnosed with classical or variant type phenylketonuria
- Have been compliant in taking at least one amino acid-based, Phe-free protein substitute, providing at least 20g protein equivalents, for at least 1 month prior to trial commencement
- Have a prescribed daily Phe allowance
- Written informed consent from patient, or from parent / carer if applicable
Exclusion Criteria:
- Currently prescribed Sapropterin or similar tetrahydrobiopterin based medication
- Pregnant or lactating
- Requiring parenteral nutrition
- Major hepatic or renal dysfunction
- Participation in other studies within 1 month prior to entry of this study
- Allergy to any of the study product ingredients, including milk protein or soya
- Investigator concern around willingness/ability of patient or parent/carer to comply with protocol requirements
Sites / Locations
- Great Ormond Street Hospital
- Guy's and St Thomas' NHS Fondation Trust
- UCLH
- Royal Manchester Children's Hospital
- Sheffield Teaching Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
GMP
Arm Description
Single arm designed, 3 day baseline, 28day on GMP
Outcomes
Primary Outcome Measures
Blood Phenylalanine (and other amino acids)
Blood spot test
Secondary Outcome Measures
Gastro-intestinal tolerance questionaire
Questionaire detailing any GI symptoms, severity and change from usual
Product compliance questionaire
Breif questionaire on amounts offered and amounts actually consumed, compared to recomended amount.
Product acceptability questionnaire
Breif tick-box questionaire on overal liking and acceptability of product
Patient reproted nutrient intake (3 day food diaries)
3 day food diaries, subsequently analysed in dietary software.
Pateint-reported appetite (visual analogue scale)
Measures of overal appetite and satiety via visual analouge scale
Antropometry (hieight and weight)
Measurements of height and weight at baseline and end of study
Safety (Adverse events reporting)
Reporting of adverse events throughout study
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02915510
Brief Title
GMP Drink for PKU Study
Official Title
Evaluating the Tolerance, Safety and Acceptability of PKU GMPro, a Whey Protein Derived Feed for the Dietary Management of Phenylketonuria in Children and Adults - a Pilot Trial
Study Type
Interventional
2. Study Status
Record Verification Date
September 2019
Overall Recruitment Status
Completed
Study Start Date
July 2016 (undefined)
Primary Completion Date
September 27, 2019 (Actual)
Study Completion Date
September 27, 2019 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Nutricia UK Ltd
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
This study will evaluate the safety, gastrointestinal (GI) tolerance, acceptability and compliance of a Glycomacropeptide-based protein substitute for patients with Phenylketonuria (PKU).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Phenylketonuria
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
40 (Actual)
8. Arms, Groups, and Interventions
Arm Title
GMP
Arm Type
Experimental
Arm Description
Single arm designed, 3 day baseline, 28day on GMP
Intervention Type
Dietary Supplement
Intervention Name(s)
Glycomacropeptide-based protein substitute
Primary Outcome Measure Information:
Title
Blood Phenylalanine (and other amino acids)
Description
Blood spot test
Time Frame
3 points throughout 28 days
Secondary Outcome Measure Information:
Title
Gastro-intestinal tolerance questionaire
Description
Questionaire detailing any GI symptoms, severity and change from usual
Time Frame
12/31 days
Title
Product compliance questionaire
Description
Breif questionaire on amounts offered and amounts actually consumed, compared to recomended amount.
Time Frame
31 days
Title
Product acceptability questionnaire
Description
Breif tick-box questionaire on overal liking and acceptability of product
Time Frame
2/31 days
Title
Patient reproted nutrient intake (3 day food diaries)
Description
3 day food diaries, subsequently analysed in dietary software.
Time Frame
6/31 days
Title
Pateint-reported appetite (visual analogue scale)
Description
Measures of overal appetite and satiety via visual analouge scale
Time Frame
6/31 days
Title
Antropometry (hieight and weight)
Description
Measurements of height and weight at baseline and end of study
Time Frame
2/31 days
Title
Safety (Adverse events reporting)
Description
Reporting of adverse events throughout study
Time Frame
31 days
10. Eligibility
Sex
All
Minimum Age & Unit of Time
3 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Male or female
Over 3 years of age
Diagnosed with classical or variant type phenylketonuria
Have been compliant in taking at least one amino acid-based, Phe-free protein substitute, providing at least 20g protein equivalents, for at least 1 month prior to trial commencement
Have a prescribed daily Phe allowance
Written informed consent from patient, or from parent / carer if applicable
Exclusion Criteria:
Currently prescribed Sapropterin or similar tetrahydrobiopterin based medication
Pregnant or lactating
Requiring parenteral nutrition
Major hepatic or renal dysfunction
Participation in other studies within 1 month prior to entry of this study
Allergy to any of the study product ingredients, including milk protein or soya
Investigator concern around willingness/ability of patient or parent/carer to comply with protocol requirements
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Rebecca Stratton, PHd
Organizational Affiliation
Nutricia Ltd UK
Official's Role
Study Director
Facility Information:
Facility Name
Great Ormond Street Hospital
City
London
Country
United Kingdom
Facility Name
Guy's and St Thomas' NHS Fondation Trust
City
London
Country
United Kingdom
Facility Name
UCLH
City
London
Country
United Kingdom
Facility Name
Royal Manchester Children's Hospital
City
Manchester
Country
United Kingdom
Facility Name
Sheffield Teaching Hospital
City
Sheffield
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
No
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GMP Drink for PKU Study
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