Back to resultsA 16 Week Study Evaluating the Introduction of a GMP Based Protein Substitute in Participants With PKU (GMP In PKU)
Primary Purpose
Phenylketonurias
Status
Completed
Phase
Not Applicable
Locations
Germany
Study Type
Interventional
Intervention
PKU Sphere
Sponsored by
About this trial
This is an interventional treatment trial for Phenylketonurias focused on measuring Phenylketonuria, PKU, Child, Children, Adult, Adults, GMP, Glycomacropeptide, Amino acid mixture, Amino acid, Protein supplement, Protein substitute
Eligibility Criteria
Inclusion Criteria:
- Diagnosis of PKU made in neonatal period
- Treated by a low-phenylalanine diet and necessity of intake of at least one protein substitute per day
- Aged 10 years and above
- Three blood Phe measurements taken within the preceding six months
- Willing to replace current amino acid based protein substitute with PKU Sphere
- Ability to take at least 50% of protein requirements as PKU Sphere
- Ability to comply with the study protocol, in the opinion of the investigator
- Willingly given, written, informed consent from patient or parent/guardian
- Willingly given, written assent (if appropriate)
Exclusion Criteria:
- Women who are pregnant or planning to become pregnant during the study period
- Participants with atypical PKU (e.g. BH4 deficient)
- Intake of sapropterin dihydrochloride (Kuvan) in the six weeks prior to recruitment in the study
- Patients with soya, milk or fish allergies
- Any other severe disease
Sites / Locations
- Kreiskliniken Reutlingen
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Experimental
Experimental
Experimental
Arm Label
4 Weeks
8 Weeks
12 Weeks
Arm Description
All patients randomised to this arm will start on an amino acid based protein substitute and they will all swtich over to incorprating PKU Sphere after 4 weeks.
All patients randomised to this arm will start on an amino acid based protein substitute and they will all swtich over to incorprating PKU Sphere after 8 weeks.
All patients randomised to this arm will start on an amino acid based protein substitute and they will all swtich over to incorprating PKU Sphere after 12 weeks.
Outcomes
Primary Outcome Measures
Change in blood Phe
Change in blood Phe concentrations when switching from an amino acid based protein substitute to a GMP based one (PKU Sphere). A minimum of five (5) blood samples (including a baseline measurement and one from each treatment arm) will be required for a patient to be included in the final analysis.
Secondary Outcome Measures
Change in blood tyrosine
Impact of a GMP based protein substitute on blood tyrosine levels. A minimum of five (5) blood samples (including a baseline measurement and one from each treatment arm) will be required for a patient to be included in the final analysis.
Dietary intake
Compliance with a low protein diet - 3-day food diary.
Protein substitute intake
Compliance with the recommended volume of protein substitute - 3-day protein substitute diary.
Stool patterns
3-day diary noting stool type (Bristol stool chart).
Satiety questionnaire
Short questionnaire asking participants to consider their satiety levels over the last 7 days, asking them if it has increased, stayed the same or decreased. Questionnaire validated as part of a bachelor thesis.
Acceptance questionnaire
Questionnaire to collect information about taste, odour and practicability of the protein substitutes. The questions are answered on a scale of 1 (bad) to 5 or 6 (good). Questionnaire validated as part of a bachelor thesis.
Gastrointestinal symptoms questionnaire
Short questionnaire asking participants to consider their general gastrointestinal health over the previous 7 days. The occurrence of various GI symptoms are given as never, rare, often or always. Questionnaire validated as part of a bachelor thesis.
Full Information
NCT ID
NCT03771391
First Posted
December 7, 2018
Last Updated
August 10, 2023
Sponsor
Vitaflo International, Ltd
Collaborators
Kreiskliniken Reutlingen, Johannes Gutenberg University Mainz
1. Study Identification
Unique Protocol Identification Number
NCT03771391
Brief Title
A 16 Week Study Evaluating the Introduction of a GMP Based Protein Substitute in Participants With PKU
Acronym
GMP In PKU
Official Title
Multicentre Study Project to Evaluate the New Generation of Protein Supplements With Glycomacropeptide (GMP) in Patients With Phenylketonuria (PKU) Aged 10 Years and Older Over a 16 Week Period.
Study Type
Interventional
2. Study Status
Record Verification Date
August 2023
Overall Recruitment Status
Completed
Study Start Date
December 6, 2018 (Actual)
Primary Completion Date
October 30, 2020 (Actual)
Study Completion Date
July 31, 2023 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vitaflo International, Ltd
Collaborators
Kreiskliniken Reutlingen, Johannes Gutenberg University Mainz
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 aims to measure changes in participants' blood phenylalanine (Phe) levels in participants with PKU when switching from a conventional amino acid based protein substitute to a GMP based protein substitute (PKU Sphere) over a 16 week period.
Detailed Description
This is a stepped wedge, open-label study of PKU Sphere for the dietary management of participants with Phenylketonuria. It will be carried out with 33 participants who will be randomised into one of three clusters. The total study length per patient is 16 weeks. Each cluster transitions from taking their standard amino acid product alone to incorporating PKU Sphere (at least 50% of their protein substitue requirement) at different timepoints: week 5, 9 or 13.
Participants will have a face-to-face clinical review at the time of recruitment (week 0) and at 16 weeks (completion of the study). They will also have an additional telephone review when they switch onto PKU Sphere.
Blood samples will be taken at baseline, and then every two weeks for the duration of the study.
In addition to these study visits and procedures, routine clinical care will continue in line with local guidelines.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Phenylketonurias
Keywords
Phenylketonuria, PKU, Child, Children, Adult, Adults, GMP, Glycomacropeptide, Amino acid mixture, Amino acid, Protein supplement, Protein substitute
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Crossover Assignment
Model Description
The study has a stepped wedge design, which is chosen over the alternative cross-over study as it allows that all participants start on the amino acid supplement and avoids the difficulty of the 'wash-out' period being the same as one of the treatment strategies being offered.
Masking
Participant
Masking Description
At the point of randomisation every effort will be made to ensure that participants are unaware of the cluster they are randomised to; this is to help ensure that compliance is not affected by participants knowing when they will start the new treatment.
Allocation
Randomized
Enrollment
15 (Actual)
8. Arms, Groups, and Interventions
Arm Title
4 Weeks
Arm Type
Experimental
Arm Description
All patients randomised to this arm will start on an amino acid based protein substitute and they will all swtich over to incorprating PKU Sphere after 4 weeks.
Arm Title
8 Weeks
Arm Type
Experimental
Arm Description
All patients randomised to this arm will start on an amino acid based protein substitute and they will all swtich over to incorprating PKU Sphere after 8 weeks.
Arm Title
12 Weeks
Arm Type
Experimental
Arm Description
All patients randomised to this arm will start on an amino acid based protein substitute and they will all swtich over to incorprating PKU Sphere after 12 weeks.
Intervention Type
Dietary Supplement
Intervention Name(s)
PKU Sphere
Other Intervention Name(s)
GMP, Glycomacropeptide
Intervention Description
PKU sphere is a powdered, low phenylalanine protein substitute containing a balanced mix of casein glycomacropeptide (GMP) isolate, essential and non-essential amino acids, carbohydrate, vitamins, minerals and docosahexaenoic acid (DHA). With sugar and sweetener.
Primary Outcome Measure Information:
Title
Change in blood Phe
Description
Change in blood Phe concentrations when switching from an amino acid based protein substitute to a GMP based one (PKU Sphere). A minimum of five (5) blood samples (including a baseline measurement and one from each treatment arm) will be required for a patient to be included in the final analysis.
Time Frame
Baseline (week 0) and weeks 2, 4, 6, 8, 10, 12, 14 and 16.
Secondary Outcome Measure Information:
Title
Change in blood tyrosine
Description
Impact of a GMP based protein substitute on blood tyrosine levels. A minimum of five (5) blood samples (including a baseline measurement and one from each treatment arm) will be required for a patient to be included in the final analysis.
Time Frame
Baseline (week 0) and weeks 2, 4, 6, 8, 10, 12, 14 and 16.
Title
Dietary intake
Description
Compliance with a low protein diet - 3-day food diary.
Time Frame
Weeks 1 and 16.
Title
Protein substitute intake
Description
Compliance with the recommended volume of protein substitute - 3-day protein substitute diary.
Time Frame
Weeks 1, 4, 8, 12 and 16
Title
Stool patterns
Description
3-day diary noting stool type (Bristol stool chart).
Time Frame
Weeks 1, 4, 8, 12 and 16
Title
Satiety questionnaire
Description
Short questionnaire asking participants to consider their satiety levels over the last 7 days, asking them if it has increased, stayed the same or decreased. Questionnaire validated as part of a bachelor thesis.
Time Frame
Weeks 1, 4, 8, 12 and 16
Title
Acceptance questionnaire
Description
Questionnaire to collect information about taste, odour and practicability of the protein substitutes. The questions are answered on a scale of 1 (bad) to 5 or 6 (good). Questionnaire validated as part of a bachelor thesis.
Time Frame
Weeks 1, 4, 8, 12 and 16
Title
Gastrointestinal symptoms questionnaire
Description
Short questionnaire asking participants to consider their general gastrointestinal health over the previous 7 days. The occurrence of various GI symptoms are given as never, rare, often or always. Questionnaire validated as part of a bachelor thesis.
Time Frame
Weeks 1, 4, 8, 12 and 16
10. Eligibility
Sex
All
Minimum Age & Unit of Time
10 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Diagnosis of PKU made in neonatal period
Treated by a low-phenylalanine diet and necessity of intake of at least one protein substitute per day
Aged 10 years and above
Three blood Phe measurements taken within the preceding six months
Willing to replace current amino acid based protein substitute with PKU Sphere
Ability to take at least 50% of protein requirements as PKU Sphere
Ability to comply with the study protocol, in the opinion of the investigator
Willingly given, written, informed consent from patient or parent/guardian
Willingly given, written assent (if appropriate)
Exclusion Criteria:
Women who are pregnant or planning to become pregnant during the study period
Participants with atypical PKU (e.g. BH4 deficient)
Intake of sapropterin dihydrochloride (Kuvan) in the six weeks prior to recruitment in the study
Patients with soya, milk or fish allergies
Any other severe disease
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Peter Freisinger
Organizational Affiliation
Kreiskliniken Reutlingen GmbH
Official's Role
Principal Investigator
Facility Information:
Facility Name
Kreiskliniken Reutlingen
City
Reutlingen
State/Province
Baden-Württemberg
ZIP/Postal Code
D-72764
Country
Germany
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
A 16 Week Study Evaluating the Introduction of a GMP Based Protein Substitute in Participants With PKU
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