PKUNCT04318509
Market Research - Acceptability Study for a New PKU Protein Substitute
A study testing GMPOWER in people with PKU and Phenylketonurias, run by Dr. Schär AG / SPA at 2 sites in United Kingdom.
Trial summary
The aim of this study is to demonstrate that a new protein substitute is acceptable and well tolerated in children with PKU. Read more about this study
- Who is it for?
- Ages 3 to 16 years
- Conditions the study is for
- PKU, Phenylketonurias, Inborn Errors of Metabolism
- What is being tested?
- GMPOWER
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 2 sitesUnited Kingdom
- Study dates
- 4 Feb 2019to 19 May 2019
- 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 3 to 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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).
Who cannot join
- 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.
Trial locations and contacts
2 sites in 1 country.
United Kingdom2 sites
Birmingham Children's Hospital
Birmingham, B4 6NH
Bristol Royal Hospital for Children
Bristol, BS2 8BJ
About this study
Official title: Market Research - Acceptability Study for a New PKU Glycomacropeptide Based Protein Substitute (Dr Schär- Mevalia Amino Acids) Mevalia PKU GMPower - Vanilla
The aim of this study is to demonstrate that a new protein substitute is acceptable and well tolerated in children with PKU.
- Conditions
- PKU, Phenylketonurias, Inborn Errors of Metabolism, Metabolic Disease
- Being tested
- GMPOWER
- Medical subject headings
- Phenylketonurias, Metabolism, Inborn Errors, Metabolic Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GMPOWERDietary supplement
(casein) glycomacropeptide protein substitute for the dietary management of PKU from the age of 3 years
| Study group | Role | What they receive |
|---|---|---|
| PKU GMPOWER | Experimental | (casein) glycomacropeptide protein substitute for the dietary management of PKU from the age of 3 years Receives: Dietary Supplement: GMPOWER |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 7 days
Product compliance
Daily questionnaire on amounts offered and amounts actually consumed, compared to recommended amount.
Measured: 7 days
Product palatability
Questionnaire data captured to evaluate taste
Measured: 7 days
Product acceptability
Brief tick-box questionnaire on overall liking and acceptability of product
Measured: 7 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Mar 2020
- Study start4 Feb 2019
- Primary completion19 May 2019
- Study completion19 May 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PKUStudies 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
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Dr. Schär AG / SPA
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04318509
- Sponsor's protocol ID
- GMPower1
- Status last verified
- Mar 2020
- First submitted
- 20 Mar 2020
- Last updated
- 24 Mar 2020
- 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 24 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Market Research - Acceptability Study for a New PKU Glycomacropeptide Based Protein Substitute (Dr Schär- Mevalia Amino Acids) Mevalia PKU GMPower - Vanilla
- ClinicalTrials.gov ID
- NCT04318509
- Acronym / sponsor ID
- GMPower1
- Conditions
- PKU, Phenylketonurias, Inborn Errors of Metabolism, Metabolic Disease
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Dr. Schär AG / SPAIndustry (pharma, biotech, devices)
- Study start
- 4 Feb 2019 (actual)
- Primary completion
- 19 May 2019 (actual)
- Study completion
- 19 May 2019 (actual)
- First posted
- 24 Mar 2020 (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.
The aim of this study is to demonstrate that a new protein substitute is acceptable and well tolerated in children with PKU.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PKU GMPOWER | Experimental | Dietary Supplement: GMPOWER(casein) glycomacropeptide protein substitute for the dietary management of PKU from the age of 3 years |
Who may qualify
- Ages
- 3 to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- 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).
Key exclusion criteria (5 listed)
- 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.
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 |
|---|---|---|
| GMPOWER(casein) glycomacropeptide protein substitute for the dietary management of PKU from the age of 3 years | Dietary supplement | PKU GMPOWER |
Primary outcome
Gastrointestinal tolerance
Time frame: 7 days
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
Time frame: 7 days
Daily questionnaire on amounts offered and amounts actually consumed, compared to recommended amount.
Product palatability
Time frame: 7 days
Questionnaire data captured to evaluate taste
Product acceptability
Time frame: 7 days
Brief tick-box questionnaire on overall liking and acceptability of product
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 24 Mar 2020.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing GMPOWER in people with PKU and Phenylketonurias, run by Dr. Schär AG / SPA at 2 sites in United Kingdom.
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 4 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 24 Mar 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United Kingdom. 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?
Gastrointestinal tolerance (time frame: 7 days). The study lists 4 primary outcomes in all. 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 24 Mar 2020.