Back to resultsMarket Research - Acceptability Study for a New PKU Protein Substitute
Primary Purpose
PKU, Phenylketonurias, Inborn Errors of Metabolism
Status
Completed
Phase
Not Applicable
Locations
United Kingdom
Study Type
Interventional
Intervention
GMPOWER
Sponsored by
About this trial
This is an interventional other trial for PKU
Eligibility Criteria
Inclusion Criteria:
- Diagnosis of PKU or PKU variant requiring a protein substitute.
- Subjects who were already taking a phenylalanine-free/low phenylalanine protein substitute and were willing to try the study product for 7 days.
- Patients aged 3 years of age to adulthood.
- Written informed consent obtained from participant or parental caregiver (as appropriate).
Exclusion Criteria:
- Presence of serious concurrent illness
- Investigator's uncertainty about the willingness or ability of the patient to comply with the protocol requirements
- Participation in any other studies involving investigational or marketed products concomitantly or within two weeks prior to entry into the study.
- Any patients having taken antibiotics over the previous 2 weeks leading up to the study.
- Patients less than 3 years of age.
Sites / Locations
- Birmingham Children's Hospital
- Bristol Royal Hospital for Children
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
PKU GMPOWER
Arm Description
(casein) glycomacropeptide protein substitute for the dietary management of PKU from the age of 3 years
Outcomes
Primary Outcome Measures
Gastrointestinal tolerance
Questionnaire detailing any GI symptoms, severity and change from usual
• The collection of daily data about the gastro-intestinal tolerance of the protein substitute.
Product compliance
Daily questionnaire on amounts offered and amounts actually consumed, compared to recommended amount.
Product palatability
Questionnaire data captured to evaluate taste
Product acceptability
Brief tick-box questionnaire on overall liking and acceptability of product
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04318509
Brief Title
Market Research - Acceptability Study for a New PKU Protein Substitute
Official Title
Market Research - Acceptability Study for a New PKU Glycomacropeptide Based Protein Substitute (Dr Schär- Mevalia Amino Acids) Mevalia PKU GMPower - Vanilla
Study Type
Interventional
2. Study Status
Record Verification Date
March 2020
Overall Recruitment Status
Completed
Study Start Date
February 4, 2019 (Actual)
Primary Completion Date
May 19, 2019 (Actual)
Study Completion Date
May 19, 2019 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Dr. Schär AG / SPA
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
The aim of this study is to demonstrate that a new protein substitute is acceptable and well tolerated in children with PKU.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
PKU, Phenylketonurias, Inborn Errors of Metabolism, Metabolic Disease
7. Study Design
Primary Purpose
Other
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
8 (Actual)
8. Arms, Groups, and Interventions
Arm Title
PKU GMPOWER
Arm Type
Experimental
Arm Description
(casein) glycomacropeptide protein substitute for the dietary management of PKU from the age of 3 years
Intervention Type
Dietary Supplement
Intervention Name(s)
GMPOWER
Intervention Description
(casein) glycomacropeptide protein substitute for the dietary management of PKU from the age of 3 years
Primary Outcome Measure Information:
Title
Gastrointestinal tolerance
Description
Questionnaire detailing any GI symptoms, severity and change from usual
• The collection of daily data about the gastro-intestinal tolerance of the protein substitute.
Time Frame
7 days
Title
Product compliance
Description
Daily questionnaire on amounts offered and amounts actually consumed, compared to recommended amount.
Time Frame
7 days
Title
Product palatability
Description
Questionnaire data captured to evaluate taste
Time Frame
7 days
Title
Product acceptability
Description
Brief tick-box questionnaire on overall liking and acceptability of product
Time Frame
7 days
10. Eligibility
Sex
All
Minimum Age & Unit of Time
3 Years
Maximum Age & Unit of Time
16 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Diagnosis of PKU or PKU variant requiring a protein substitute.
Subjects who were already taking a phenylalanine-free/low phenylalanine protein substitute and were willing to try the study product for 7 days.
Patients aged 3 years of age to adulthood.
Written informed consent obtained from participant or parental caregiver (as appropriate).
Exclusion Criteria:
Presence of serious concurrent illness
Investigator's uncertainty about the willingness or ability of the patient to comply with the protocol requirements
Participation in any other studies involving investigational or marketed products concomitantly or within two weeks prior to entry into the study.
Any patients having taken antibiotics over the previous 2 weeks leading up to the study.
Patients less than 3 years of age.
Facility Information:
Facility Name
Birmingham Children's Hospital
City
Birmingham
ZIP/Postal Code
B4 6NH
Country
United Kingdom
Facility Name
Bristol Royal Hospital for Children
City
Bristol
ZIP/Postal Code
BS2 8BJ
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Market Research - Acceptability Study for a New PKU Protein Substitute
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