PhenylketonuriasGMP In PKUNCT03771391
A 16 Week Study Evaluating the Introduction of a GMP Based Protein Substitute in Participants With PKU
A study testing PKU Sphere in people with Phenylketonurias, run by Vitaflo International, Ltd at 1 site in Germany.
Trial 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. Read more about this study
- Who is it for?
- Ages 10 years and older
- Conditions the study is for
- Phenylketonurias
- What is being tested?
- PKU Sphere
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteGermany
- Study dates
- 6 Dec 2018to 31 Jul 2023
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 10 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
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- 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)
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
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.
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.
Read the 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.
- Conditions
- Phenylketonurias
- Being tested
- PKU Sphere
- Keywords
- Phenylketonuria, PKU, Child, Children, Adult, Adults, GMP, Glycomacropeptide, Amino acid mixture, Amino acid, Protein supplement, Protein substitute
- Medical subject headings
- Phenylketonurias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PKU SphereDietary supplement
Also called GMP, Glycomacropeptide
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.
| Study group | Role | What they receive |
|---|---|---|
| 4 Weeks | Experimental | 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. Receives: Dietary Supplement: PKU Sphere |
| 8 Weeks | Experimental | 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. Receives: Dietary Supplement: PKU Sphere |
| 12 Weeks | Experimental | 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. Receives: Dietary Supplement: PKU Sphere |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline (week 0) and weeks 2, 4, 6, 8, 10, 12, 14 and 16.
Secondary outcomes (7)
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.
Measured: Baseline (week 0) and weeks 2, 4, 6, 8, 10, 12, 14 and 16.
Dietary intake
Compliance with a low protein diet - 3-day food diary.
Measured: Weeks 1 and 16.
Protein substitute intake
Compliance with the recommended volume of protein substitute - 3-day protein substitute diary.
Measured: Weeks 1, 4, 8, 12 and 16
Stool patterns
3-day diary noting stool type (Bristol stool chart).
Measured: Weeks 1, 4, 8, 12 and 16
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.
Measured: Weeks 1, 4, 8, 12 and 16
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.
Measured: Weeks 1, 4, 8, 12 and 16
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.
Measured: Weeks 1, 4, 8, 12 and 16
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Dec 2018
- Study start6 Dec 2018
- Primary completion30 Oct 2020
- Study completion31 Jul 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PhenylketonuriasStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- 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
- Single blind
- Who is masked
- Participant
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vitaflo International, Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Kreiskliniken Reutlingen, Johannes Gutenberg University Mainz
- Responsible party
- Sponsor
- Investigators
- Peter Freisinger, Principal investigator, Kreiskliniken Reutlingen GmbH
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03771391
- Sponsor's protocol ID
- MCT-W-PKUSp-2017-06-29
- Other IDs
- F-2018-043
- Acronym
- GMP In PKU
- Status last verified
- Feb 2024
- First submitted
- 7 Dec 2018
- Last updated
- 16 Feb 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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.
- ClinicalTrials.gov ID
- NCT03771391
- Acronym / sponsor ID
- GMP In PKU / MCT-W-PKUSp-2017-06-29
- Conditions
- Phenylketonurias
- Keywords
- Phenylketonuria, PKU, Child, Children, Adult, Adults, GMP, Glycomacropeptide
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Vitaflo International, LtdIndustry (pharma, biotech, devices)
- Collaborators
- Kreiskliniken Reutlingen, Johannes Gutenberg University Mainz
- Study start
- 6 Dec 2018 (actual)
- Primary completion
- 30 Oct 2020 (actual)
- Study completion
- 31 Jul 2023 (actual)
- First posted
- 11 Dec 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Single blind (participant)
- Number of arms
- 3
- 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.
| Arm | Type | Receives |
|---|---|---|
| 4 Weeks | Experimental | Dietary Supplement: PKU SphereAll 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. |
| 8 Weeks | Experimental | Dietary Supplement: PKU SphereAll 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. |
| 12 Weeks | Experimental | Dietary Supplement: PKU SphereAll 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. |
Who may qualify
- Ages
- 10 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- 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
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PKU SphereAlso: GMP, GlycomacropeptidePKU 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. | Dietary supplement | 12 Weeks, 4 Weeks, 8 Weeks |
Primary outcome
Change in blood Phe
Time frame: Baseline (week 0) and weeks 2, 4, 6, 8, 10, 12, 14 and 16.
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.
7 secondary outcomes
Change in blood tyrosine
Time frame: Baseline (week 0) and weeks 2, 4, 6, 8, 10, 12, 14 and 16.
Dietary intake
Time frame: Weeks 1 and 16.
Protein substitute intake
Time frame: Weeks 1, 4, 8, 12 and 16
Stool patterns
Time frame: Weeks 1, 4, 8, 12 and 16
Satiety questionnaire
Time frame: Weeks 1, 4, 8, 12 and 16
Acceptance questionnaire
Time frame: Weeks 1, 4, 8, 12 and 16
Gastrointestinal symptoms questionnaire
Time frame: Weeks 1, 4, 8, 12 and 16
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Feb 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Peter Freisinger principal investigator
Kreiskliniken Reutlingen GmbH
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing PKU Sphere in people with Phenylketonurias, run by Vitaflo International, Ltd at 1 site in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in blood Phe (time frame: Baseline (week 0) and weeks 2, 4, 6, 8, 10, 12, 14 and 16.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Feb 2024.