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Market 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
Dr. Schär AG / SPA
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional other trial for PKU

Eligibility Criteria

3 Years - 16 Years (Child)All SexesDoes not accept healthy volunteers

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

First Posted
March 20, 2020
Last Updated
March 20, 2020
Sponsor
Dr. Schär AG / SPA
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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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