PhenylketonuriaNCT02915510
GMP Drink for PKU Study
A study testing Glycomacropeptide-based protein substitute in people with Phenylketonuria, run by Nutricia UK Ltd at 5 sites in United Kingdom.
Trial summary
This study will evaluate the safety, gastrointestinal (GI) tolerance, acceptability and compliance of a Glycomacropeptide-based protein substitute for patients with Phenylketonuria (PKU). Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- Phenylketonuria
- What is being tested?
- Glycomacropeptide-based protein substitute
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 5 sitesUnited Kingdom
- Study dates
- Jul 2016to 27 Sep 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 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male or female
- Over 3 years of age
- Diagnosed with classical or variant type phenylketonuria
- Have been compliant in taking at least one amino acid-based, Phe-free protein substitute, providing at least 20g protein equivalents, for at least 1 month prior to trial commencement
- Have a prescribed daily Phe allowance
- Written informed consent from patient, or from parent / carer if applicable
Who cannot join
- Currently prescribed Sapropterin or similar tetrahydrobiopterin based medication
- Pregnant or lactating
- Requiring parenteral nutrition
- Major hepatic or renal dysfunction
- Participation in other studies within 1 month prior to entry of this study
- Allergy to any of the study product ingredients, including milk protein or soya
- Investigator concern around willingness/ability of patient or parent/carer to comply with protocol requirements
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United Kingdom5 sites
Great Ormond Street Hospital
London
Guy's and St Thomas' NHS Fondation Trust
London
UCLH
London
Royal Manchester Children's Hospital
Manchester
Sheffield Teaching Hospital
Sheffield
About this study
Official title: Evaluating the Tolerance, Safety and Acceptability of PKU GMPro, a Whey Protein Derived Feed for the Dietary Management of Phenylketonuria in Children and Adults - a Pilot Trial
This study will evaluate the safety, gastrointestinal (GI) tolerance, acceptability and compliance of a Glycomacropeptide-based protein substitute for patients with Phenylketonuria (PKU).
- Conditions
- Phenylketonuria
- Being tested
- Glycomacropeptide-based 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.
Glycomacropeptide-based protein substituteDietary supplement
| Study group | Role | What they receive |
|---|---|---|
| GMP | Experimental | Single arm designed, 3 day baseline, 28day on GMP Receives: Dietary Supplement: Glycomacropeptide-based protein substitute |
What is being measured
The primary outcome is the main question the study is designed to answer.
Blood Phenylalanine (and other amino acids)
Blood spot test
Measured: 3 points throughout 28 days
Secondary outcomes (7)
Gastro-intestinal tolerance questionaire
Questionaire detailing any GI symptoms, severity and change from usual
Measured: 12/31 days
Product compliance questionaire
Breif questionaire on amounts offered and amounts actually consumed, compared to recomended amount.
Measured: 31 days
Product acceptability questionnaire
Breif tick-box questionaire on overal liking and acceptability of product
Measured: 2/31 days
Patient reproted nutrient intake (3 day food diaries)
3 day food diaries, subsequently analysed in dietary software.
Measured: 6/31 days
Pateint-reported appetite (visual analogue scale)
Measures of overal appetite and satiety via visual analouge scale
Measured: 6/31 days
Antropometry (hieight and weight)
Measurements of height and weight at baseline and end of study
Measured: 2/31 days
Safety (Adverse events reporting)
Reporting of adverse events throughout study
Measured: 31 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Sep 2016 (estimated)
- Study startJul 2016
- Primary completion27 Sep 2019
- Study completion27 Sep 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PhenylketonuriaStudies 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
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nutricia UK Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Rebecca Stratton, PHd, Study director, Nutricia Ltd UK
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02915510
- Sponsor's protocol ID
- GMP2016
- Status last verified
- Sep 2019
- First submitted
- 8 Aug 2016
- Last updated
- 1 Oct 2019
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Oct 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluating the Tolerance, Safety and Acceptability of PKU GMPro, a Whey Protein Derived Feed for the Dietary Management of Phenylketonuria in Children and Adults - a Pilot Trial
- ClinicalTrials.gov ID
- NCT02915510
- Acronym / sponsor ID
- GMP2016
- Conditions
- Phenylketonuria
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (actual)
- Lead sponsor
- Nutricia UK LtdIndustry (pharma, biotech, devices)
- Study start
- Jul 2016
- Primary completion
- 27 Sep 2019 (actual)
- Study completion
- 27 Sep 2019 (actual)
- First posted
- 27 Sep 2016 (estimated)
- 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 will evaluate the safety, gastrointestinal (GI) tolerance, acceptability and compliance of a Glycomacropeptide-based protein substitute for patients with Phenylketonuria (PKU).
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| GMP | Experimental | Dietary Supplement: Glycomacropeptide-based protein substituteSingle arm designed, 3 day baseline, 28day on GMP |
Who may qualify
- Ages
- 3 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Male or female
- Over 3 years of age
- Diagnosed with classical or variant type phenylketonuria
- Have been compliant in taking at least one amino acid-based, Phe-free protein substitute, providing at least 20g protein equivalents, for at least 1 month prior to trial commencement
- Have a prescribed daily Phe allowance
Key exclusion criteria (7 listed)
- Currently prescribed Sapropterin or similar tetrahydrobiopterin based medication
- Pregnant or lactating
- Requiring parenteral nutrition
- Major hepatic or renal dysfunction
- Participation in other studies within 1 month prior to entry of this study
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 |
|---|---|---|
| Glycomacropeptide-based protein substitute | Dietary supplement | GMP |
Primary outcome
Blood Phenylalanine (and other amino acids)
Time frame: 3 points throughout 28 days
Blood spot test
7 secondary outcomes
Gastro-intestinal tolerance questionaire
Time frame: 12/31 days
Product compliance questionaire
Time frame: 31 days
Product acceptability questionnaire
Time frame: 2/31 days
Patient reproted nutrient intake (3 day food diaries)
Time frame: 6/31 days
Pateint-reported appetite (visual analogue scale)
Time frame: 6/31 days
Antropometry (hieight and weight)
Time frame: 2/31 days
Safety (Adverse events reporting)
Time frame: 31 days
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 1 Oct 2019.
- Individual participant dataSharing planned: No
Related trials for Phenylketonuria
RecruitingPhase 3
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
Phenylketonuria
RecruitingPhase 2
A Study of MZE782 in Adults With PKU
Phenylketonuria (PKU)
Recruiting
Impact of Phenylalanine Elevations on Brain and Cognition in Adult PKU Carriers
Carrier of Phenylketonuria, Healthy
RecruitingPhase 3
A Study of JNT-517 in Participants With Phenylketonuria (PKU)
Phenylketonuria
Not yet recruiting
Market Research of a Slow Release Protein Substitute for the Dietary Management of PKU
Phenylketonuria (PKU) and Hyperphenylalaninemia, Brain Diseases, Metabolic, Inborn
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.
Rebecca Stratton, PHd study director
Nutricia Ltd UK
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Glycomacropeptide-based protein substitute in people with Phenylketonuria, run by Nutricia UK Ltd at 5 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 6 inclusion and 7 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 1 Oct 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 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?
Blood Phenylalanine (and other amino acids) (time frame: 3 points throughout 28 days). 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 1 Oct 2019.