Phenylketonuriasexpress plusNCT05051657
Evaluation of the Express Plus Range
A study testing PKU express plus in people with Phenylketonurias and PKU, run by Vitaflo International, Ltd at 6 sites in United Kingdom.
Trial summary
A prospective, open label, acceptability study to evaluate PKU, MSUD, HCU, TYR and GA express plus in the dietary management of 40 patients with IEM. The following parameters will be assessed: adherence to prescribed dietary intakes, palatability, usability, gastrointestinal tolerance, clinically relevant routine… Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- Phenylketonurias, PKU, Homocystinuria
- What is being tested?
- PKU express plus
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 28 participants
- Where
- 6 sitesUnited Kingdom
- Study dates
- 14 Jun 2021to 21 Jan 2026
- 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 or associated disorder for example, Tetrahydrobiopterin (BH4) deficiency, requiring a low protein diet and Phe-free L-amino acid supplements (protein substitute).
- Established on PKU express or an equivalent protein substitute
- In the opinion of the study investigator, the participant is able to take the study product and meets at least one of the following criteria:
- taking an age inappropriate protein substitute
- struggling with adherence to current protein substitute
- delayed in transitioning
Show all 9Show fewer
- Aged 3 years and above
- Willingly given, written, informed consent from patient or parent/guardian.
- Willingly given, written assent (if appropriate).
Who cannot join
- Diagnosis of persistent hyperphenylalaninaemia, or mild PKU not requiring dietary intervention with a low protein diet and protein substitute.
- Patients who are tube-fed.
- Individuals, who in the opinion of the investigator, are unable to comply with the requirements of the protocol.
- Any co-morbidity/condition which in the opinion of the Investigator, would preclude participation in the study.
- Patients who are currently participating in, plan to participate in, or have participated in an interventional investigational drug, food or medical device trial within 30 days prior to screening.
- Use of additional macro/micronutrient supplements during the study period, unless clinically indicated and prescribed by the investigator (must be recorded in patient case record file).
- 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 may, in consultation with their doctor, continue on the study's dietary product if they wish but will not have any investigations that would not normally be carried out during pregnancy.)
- Those with allergies to fish, milk or soya.
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United Kingdom6 sites
Royal Belfast Hospital for Sick Children
Belfast, BT12 6BA
Birmingham Women's and Children's Hospital
Birmingham, B15 2TG
Bristol Royal Hospital for Children
Bristol, BS2 8BJ
Greater Glasgow and Clyde NHS Foundation Trust
Glasgow, G12 0XH
Great Ormond Street Hospital for Children
London, WC1N 3JH
Nottingham Children's Hospital
Nottingham, NG7 2UH
About this study
Official title: A Study To Evaluate The Acceptability Of the Express Plus Range, Foods For Special Medical Purposes (FSMP), In Children And Adults With Inborn Errors of Metabolism (IEM)
A prospective, open label, acceptability study to evaluate PKU, MSUD, HCU, TYR and GA express plus in the dietary management of 40 patients with IEM. The following parameters will be assessed: adherence to prescribed dietary intakes, palatability, usability, gastrointestinal tolerance, clinically relevant routine biochemical parameters, timeframe to transition and contribution of the express plus range to overall protein substitute intake over a 28 day period.
Read the detailed description
This is a prospective acceptability study of the express plus range in 40 participants aged at least three years of age for the dietary management of IEM (PKU, MSUD, HCU, TYR and GA ).
The study will involve a Transition Period where patients will build-up to at least one sachet of express plus per day. During the Transition Period, daily intake of express plus and the patient's other PS will be recorded, including the time taken to consume.
The Transition Period will last a maximum of 6 weeks, after which the patient will enter a 28-day Evaluation Period. During the Evaluation Period, patients will continue to record intakes, as well as GI tolerance and a final evaluation of the product's palatability and usability. Some patients, likely adults, will not require a transition from their original protein substitute to at least one sachet of express plus. These patients can enter the Evaluation Period directly.
The end of the 4-week Evaluation Period is defined as the participant's End of Study. Following this, participants will be followed up as per standard of care. Some of this standard of care data may be relayed to the sponsor for a maximum of 26 weeks. This will last for a maximum of 26 weeks and will involve the collection of routine phe and tyrosine levels, as well as amount of PS prescribed and taken. Information will also be collected from patients regarding the reasons for the success or indeed failure of transition to PKU express plus. This data will come from routine clinical contact and will be recorded by the HCP, rather than by the patient.
- Conditions
- Phenylketonurias, PKU, Homocystinuria, Maple Syrup Urine Disease, Hereditary Tyrosinemia, Glutaric Acidemia I
- Being tested
- PKU express plus
- Medical subject headings
- Phenylketonurias, Homocystinuria, Maple Syrup Urine Disease, Tyrosinemias, Glutaric Acidemia I
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PKU express plusDietary supplement
PKU express plus is a powdered protein substitute for the dietary management of PKU. It is formulated in a variety of flavours to provide a convenient, low volume, lower calorie, pre-measured protein substitute, containing a full micronutrient profile.
| Study group | Role | What they receive |
|---|---|---|
| express pus range (PKU, MSUD, HCU, TYR and GA) | Experimental | express plus to be transitioned onto over a maximum of 6 weeks and then incorporated into each participants usual diet for 28 days. Amount taken and frequency to be determined by dietitian. Receives: Dietary Supplement: PKU express plus |
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
Participants will record the amount of PKU express taken each day compared to the amount recommended by their dietitian
Measured: Days 1-28
Transition time
Time taken for participants to transition from their current diet to the inclusion of one sachet of PKU express plus per day
Measured: Transition period Weeks 1-6 (maximum)
Change in gastrointestinal tolerance from week 1 to week 4
Participants will self-report any gastrointestinal symptoms experience over the course of the study in daily study diaries
Measured: Days 1-7 and days 21-28
Product acceptability rated on a Likert scale by the patient after 28-day intake
Participants will answer questions relating to PKU express plus' palatability and ease of use following the end of study
Measured: Visit 2 (end of 28 day evaluation period)
Change in general neophobia
Participants to complete general neophobia questionnaire at baseline and end of study to compare results
Measured: Visit 1 (Day 1) and Visit 2 (end of 28 day evaluation period)
Change in food neophobia
Participants to complete food neophobia questionnaire at baseline and end of study to compare results
Measured: Visit 1 (Day 1) and Visit 2 (end of 28 day evaluation period)
Nutritional suitability: change Phenylalanine levels
PKU express plus' nutritional suitability will be assessed by evaluating Phenylalanine levels recorded as part of routine care
Measured: Visit 1 until visit 2 (day 28)
Nutritional suitability: change Tyrosine levels
PKU express plus' nutritional suitability will be assessed by evaluating Tyrosine levels recorded as part of routine care
Measured: Visit 1 until visit 2 (day 28)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Sep 2021
- Study start14 Jun 2021
- Primary completion30 Sep 2025
- Study completion21 Jan 2026
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
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- single group - all patients to receive relevant express plus product
- Masking
- None (open label)
- Enrolment
- 28 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vitaflo International, Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Anita MacDonald, Study director, Birmingham Women's and Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05051657
- Sponsor's protocol ID
- MCT-EXPP-2019-05-21
- Other IDs
- 281995, 20/YH/0248
- Acronym
- express plus
- Status last verified
- May 2026
- First submitted
- 10 Sep 2021
- Last updated
- 18 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: No plans to share IPD
Study at a glance
From the registry record, last updated by the sponsor on 18 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study To Evaluate The Acceptability Of the Express Plus Range, Foods For Special Medical Purposes (FSMP), In Children And Adults With Inborn Errors of Metabolism (IEM)
- ClinicalTrials.gov ID
- NCT05051657
- Acronym / sponsor ID
- express plus / MCT-EXPP-2019-05-21
- Conditions
- Phenylketonurias, PKU, Homocystinuria, Maple Syrup Urine Disease, Hereditary Tyrosinemia, Glutaric Acidemia I
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 28 participants (actual)
- Lead sponsor
- Vitaflo International, LtdIndustry (pharma, biotech, devices)
- Study start
- 14 Jun 2021 (actual)
- Primary completion
- 30 Sep 2025 (actual)
- Study completion
- 21 Jan 2026 (actual)
- First posted
- 21 Sep 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.
A prospective, open label, acceptability study to evaluate PKU, MSUD, HCU, TYR and GA express plus in the dietary management of 40 patients with IEM. The following parameters will be assessed: adherence to prescribed dietary intakes, palatability, usability, gastrointestinal tolerance, clinically relevant routine biochemical parameters, timeframe to transition and contribution of the express plus range to overall protein substitute intake over a 28 day period.
Detailed description
This is a prospective acceptability study of the express plus range in 40 participants aged at least three years of age for the dietary management of IEM (PKU, MSUD, HCU, TYR and GA ).
The study will involve a Transition Period where patients will build-up to at least one sachet of express plus per day. During the Transition Period, daily intake of express plus and the patient's other PS will be recorded, including the time taken to consume.
The Transition Period will last a maximum of 6 weeks, after which the patient will enter a 28-day Evaluation Period. During the Evaluation Period, patients will continue to record intakes, as well as GI tolerance and a final evaluation of the product's palatability and usability. Some patients, likely adults, will not require a transition from their original protein substitute to at least one sachet of express plus. These patients can enter the Evaluation Period directly.
The end of the 4-week Evaluation Period is defined as the participant's End of Study. Following this, participants will be followed up as per standard of care. Some of this standard of care data may be relayed to the sponsor for a maximum of 26 weeks. This will last for a maximum of 26 weeks and will involve the collection of routine phe and tyrosine levels, as well as amount of PS prescribed and taken. Information will also be collected from patients regarding the reasons for the success or indeed failure of transition to PKU express plus. This data will come from routine clinical contact and will be recorded by the HCP, rather than by the patient.
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- single group - all patients to receive relevant express plus product
| Arm | Type | Receives |
|---|---|---|
| express pus range (PKU, MSUD, HCU, TYR and GA) | Experimental | Dietary Supplement: PKU express plusexpress plus to be transitioned onto over a maximum of 6 weeks and then incorporated into each participants usual diet for 28 days. Amount taken and frequency to be determined by dietitian. |
Who may qualify
- Ages
- 3 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- Diagnosis of PKU or associated disorder for example, Tetrahydrobiopterin (BH4) deficiency, requiring a low protein diet and Phe-free L-amino acid supplements (protein substitute).
- Established on PKU express or an equivalent protein substitute
- In the opinion of the study investigator, the participant is able to take the study product and meets at least one of the following criteria:
- Aged 3 years and above
- Willingly given, written, informed consent from patient or parent/guardian.
Key exclusion criteria (8 listed)
- Diagnosis of persistent hyperphenylalaninaemia, or mild PKU not requiring dietary intervention with a low protein diet and protein substitute.
- Patients who are tube-fed.
- Individuals, who in the opinion of the investigator, are unable to comply with the requirements of the protocol.
- Any co-morbidity/condition which in the opinion of the Investigator, would preclude participation in the study.
- Patients who are currently participating in, plan to participate in, or have participated in an interventional investigational drug, food or medical device trial within 30 days prior to screening.
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 express plusPKU express plus is a powdered protein substitute for the dietary management of PKU. It is formulated in a variety of flavours to provide a convenient, low volume, lower calorie, pre-measured protein substitute, containing a full micronutrient profile. | Dietary supplement | express pus range (PKU, MSUD, HCU, TYR and GA) |
Primary outcome
Questionnaire of self-reported adherence to the prescribed amount of study product
Time frame: Days 1-28
Participants will record the amount of PKU express taken each day compared to the amount recommended by their dietitian
Transition time
Time frame: Transition period Weeks 1-6 (maximum)
Time taken for participants to transition from their current diet to the inclusion of one sachet of PKU express plus per day
Change in gastrointestinal tolerance from week 1 to week 4
Time frame: Days 1-7 and days 21-28
Participants will self-report any gastrointestinal symptoms experience over the course of the study in daily study diaries
Product acceptability rated on a Likert scale by the patient after 28-day intake
Time frame: Visit 2 (end of 28 day evaluation period)
Participants will answer questions relating to PKU express plus' palatability and ease of use following the end of study
Change in general neophobia
Time frame: Visit 1 (Day 1) and Visit 2 (end of 28 day evaluation period)
Participants to complete general neophobia questionnaire at baseline and end of study to compare results
Change in food neophobia
Time frame: Visit 1 (Day 1) and Visit 2 (end of 28 day evaluation period)
Participants to complete food neophobia questionnaire at baseline and end of study to compare results
Nutritional suitability: change Phenylalanine levels
Time frame: Visit 1 until visit 2 (day 28)
PKU express plus' nutritional suitability will be assessed by evaluating Phenylalanine levels recorded as part of routine care
Nutritional suitability: change Tyrosine levels
Time frame: Visit 1 until visit 2 (day 28)
PKU express plus' nutritional suitability will be assessed by evaluating Tyrosine levels recorded as part of routine care
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 18 May 2026.
- Individual participant dataSharing planned: No. No plans to share IPD
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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 study director
Birmingham Women's and Children's Hospital
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing PKU express plus in people with Phenylketonurias and PKU, run by Vitaflo International, Ltd at 6 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 9 inclusion and 8 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 18 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 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: Days 1-28). The study lists 8 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 18 May 2026.