PhenylketonuriasNCT05096988
Evaluation of PKU Sphere Liquid
A study testing PKU sphere liquid in people with Phenylketonurias, run by Vitaflo International, Ltd at 3 sites in United Kingdom.
Trial summary
PKU Sphere Liquid is a prospective, open-label, acceptability study to evaluate PKU sphere liquid in up to 15 participants aged 3 and above for the dietary management of PKU over 31 days. Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- Phenylketonurias
- What is being tested?
- PKU sphere liquid
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- 3 sitesUnited Kingdom
- Study dates
- 23 Nov 2021to 28 Feb 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 3 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of PKU requiring a low protein diet and protein substitute.
- Aged three (3) years and over
- Already taking a minimum of one GMP based protein substitute per day or have previously taken one
- Able to comply with the study protocol and take the study product, according to the opinion of the PI.
- Willingly given, written, informed consent from patient or parent/guardian.
- Willingly given, written assent (if appropriate).
Who cannot join
- Inability to comply with the study protocol, in the opinion of the investigator.
- Any co-morbidity, which, in the opinion of the investigator, would preclude participation in the study.
- Diagnosis of persistent hyperphenylalaninaemia, or mild PKU not requiring dietary intervention with a low protein diet and protein supplement.
- Unwilling/unable to take a GMP based protein substitute.
- Women who are pregnant / breastfeeding at the start of the study or planning to become pregnant during the study period.
- N.B.: Women who become pregnant unexpectedly during this study will be immediately withdrawn from the study.
Trial locations and contacts
3 sites in 1 country.
United Kingdom3 sites
Royal Victoria Hospital
Belfast, BT12 6BA
Birmingham Children's Hospital
Birmingham, B4 6NH
Northern General Hospital
Sheffield, S5 7AU
About this study
Official title: A Study to Evaluate the Acceptability, Tolerance and Adherence of Children and Adults Consuming PKU Sphere Liquid, a Food for Special Medical Purposes (FSMP), for the Dietary Management of Phenylketonuria (PKU)
PKU Sphere Liquid is a prospective, open-label, acceptability study to evaluate PKU sphere liquid in up to 15 participants aged 3 and above for the dietary management of PKU over 31 days.
Read the detailed description
PKU Sphere Liquid is a newly-developed Food for Special Medical Purposes (FSMP) designed for the dietary management of patients with phenylketonuria (PKU).
PKU sphere liquid is a vanilla flavoured, ready-to-drink, low phenylalanine protein substitute containing a blend of casein glycomacropeptide (GMP) isolate, essential and non-essential amino acids, carbohydrate, fat, vitamins, minerals, and Docosachexaenoic acid (DHA).
This is a feasibility study designed to evaluate the acceptability, tolerance and adherence of children and adults consuming PKU sphere liquid.
Participants will be given a four-week supply of PKU Sphere Liquid and they will be asked to complete a daily diary and short questionnaire for four weeks to record information on: adherence, gastrointestinal tolerance, palatability and how the product is used.
Each participant will be on the trial for 31 days. This includes a 3-day baseline period and 28 days taking the study product. If deemed appropriate in each individual circumstance, the sponsor of the trial, Vitaflo International Ltd, will continue to supply the product free of charge at the end of the trial until available on prescription.
- Conditions
- Phenylketonurias
- Being tested
- PKU sphere liquid
- Keywords
- PKU, Sphere, Liquid
- Medical subject headings
- Phenylketonurias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PKU sphere liquidDietary supplement
PKU sphere liquid will be prescribed by the study dietitian based on the patient's individual requirements.
| Study group | Role | What they receive |
|---|---|---|
| PKU sphere liquid | Experimental | PKU sphere liquid will be prescribed by the study dietitian based on the patient's individual requirements. Receives: Dietary Supplement: PKU sphere liquid |
What is being measured
The primary outcome is the main question the study is designed to answer.
Questionnaire of self-reported adherence to the prescribed amount of study product
Assessment of participant's adherence to prescribed amount during the eight week intake of the study product
Measured: 4 weeks
Product acceptability rated on a Likert scale by the participant after four week intake
Assessment of participant's acceptability following four week intake of the study product Scale is from 1 to 5: 1 = Loved it, 5 = Really didn't like it
Measured: 4 weeks
Questionnaire of self-reported changes in gastrointestinal tolerance during four week intake
Assessment of participant's gastrointestinal tolerance during the four week intake of the study product
Measured: 4 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Oct 2021
- Study start23 Nov 2021
- Primary completion21 Jun 2022
- Study completion28 Feb 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vitaflo International, Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Anita Macdonald, Principal investigator, Birmingham Women's and Children's NHS Foundation Trust
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05096988
- Sponsor's protocol ID
- MCT-GMPL-2018-02-21
- Other IDs
- 271801, 20/NW/0273
- Status last verified
- Feb 2024
- First submitted
- 14 Oct 2021
- 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
- A Study to Evaluate the Acceptability, Tolerance and Adherence of Children and Adults Consuming PKU Sphere Liquid, a Food for Special Medical Purposes (FSMP), for the Dietary Management of Phenylketonuria (PKU)
- ClinicalTrials.gov ID
- NCT05096988
- Acronym / sponsor ID
- MCT-GMPL-2018-02-21
- Conditions
- Phenylketonurias
- Keywords
- PKU, Sphere, Liquid
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 19 participants (actual)
- Lead sponsor
- Vitaflo International, LtdIndustry (pharma, biotech, devices)
- Study start
- 23 Nov 2021 (actual)
- Primary completion
- 21 Jun 2022 (actual)
- Study completion
- 28 Feb 2023 (actual)
- First posted
- 27 Oct 2021 (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.
PKU Sphere Liquid is a prospective, open-label, acceptability study to evaluate PKU sphere liquid in up to 15 participants aged 3 and above for the dietary management of PKU over 31 days.
Detailed description
PKU Sphere Liquid is a newly-developed Food for Special Medical Purposes (FSMP) designed for the dietary management of patients with phenylketonuria (PKU).
PKU sphere liquid is a vanilla flavoured, ready-to-drink, low phenylalanine protein substitute containing a blend of casein glycomacropeptide (GMP) isolate, essential and non-essential amino acids, carbohydrate, fat, vitamins, minerals, and Docosachexaenoic acid (DHA).
This is a feasibility study designed to evaluate the acceptability, tolerance and adherence of children and adults consuming PKU sphere liquid.
Participants will be given a four-week supply of PKU Sphere Liquid and they will be asked to complete a daily diary and short questionnaire for four weeks to record information on: adherence, gastrointestinal tolerance, palatability and how the product is used.
Each participant will be on the trial for 31 days. This includes a 3-day baseline period and 28 days taking the study product. If deemed appropriate in each individual circumstance, the sponsor of the trial, Vitaflo International Ltd, will continue to supply the product free of charge at the end of the trial until available on prescription.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PKU sphere liquid | Experimental | Dietary Supplement: PKU sphere liquidPKU sphere liquid will be prescribed by the study dietitian based on the patient's individual requirements. |
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)
- Diagnosis of PKU requiring a low protein diet and protein substitute.
- Aged three (3) years and over
- Already taking a minimum of one GMP based protein substitute per day or have previously taken one
- Able to comply with the study protocol and take the study product, according to the opinion of the PI.
- Willingly given, written, informed consent from patient or parent/guardian.
Key exclusion criteria (6 listed)
- Inability to comply with the study protocol, in the opinion of the investigator.
- Any co-morbidity, which, in the opinion of the investigator, would preclude participation in the study.
- Diagnosis of persistent hyperphenylalaninaemia, or mild PKU not requiring dietary intervention with a low protein diet and protein supplement.
- Unwilling/unable to take a GMP based protein substitute.
- Women who are pregnant / breastfeeding at the start of the study or planning to become pregnant during the study period.
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 sphere liquidPKU sphere liquid will be prescribed by the study dietitian based on the patient's individual requirements. | Dietary supplement | PKU sphere liquid |
Primary outcome
Questionnaire of self-reported adherence to the prescribed amount of study product
Time frame: 4 weeks
Assessment of participant's adherence to prescribed amount during the eight week intake of the study product
Product acceptability rated on a Likert scale by the participant after four week intake
Time frame: 4 weeks
Assessment of participant's acceptability following four week intake of the study product Scale is from 1 to 5: 1 = Loved it, 5 = Really didn't like it
Questionnaire of self-reported changes in gastrointestinal tolerance during four week intake
Time frame: 4 weeks
Assessment of participant's gastrointestinal tolerance during the four week intake of the study 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 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.
Anita Macdonald principal investigator
Birmingham Women's and Children's NHS Foundation Trust
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing PKU sphere liquid in people with Phenylketonurias, run by Vitaflo International, Ltd at 3 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 6 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 3 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?
Questionnaire of self-reported adherence to the prescribed amount of study product (time frame: 4 weeks). The study lists 3 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 16 Feb 2024.