PhenylketonuriasNCT03167697
Evaluating the Efficacy of PKU Synergy in Patients Expressing Phenylketonuria or Hyperphenylalaninemia
A study testing Synergy and Routine in people with Phenylketonurias and Hyperphenylalaninaemia, Type I, run by Nutricia UK Ltd at 6 sites in United Kingdom.
Trial summary
This study centres around a new one-a-day phenylalanine-free protein substitute for phenylketonuria patients. Fifty eligible adults (≥ 16 years) with proven phenylketonuria or hyperphenylalaninemia will be recruited and randomly allocated to one of two intervention arms (n = 25 per arm). Read more about this study
- Who is it for?
- Ages 16 to 100 years
- Conditions the study is for
- Phenylketonurias, Hyperphenylalaninaemia, Type I
- What is being tested?
- Synergy and Routine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 6 sitesUnited Kingdom
- Study dates
- 1 May 2017to 17 Dec 2018
- 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 16 to 100 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male or female
- Over 16 years of age
- Diagnosed with proven PKU or hyperphenylalaninemia with an increased phenylalanine-tolerance/intake
- Currently taking a maximum of 1 protein substitute per day (equal to 20g protein equivalent)
- Have a minimum blood phenylalanine level of ≥ 600 umol/L (for PKU patients)
- Have relaxed (if not stopped) their dietary and protein substitute regimen for at least 1 month prior to trial commencement
- Have Written informed consent from patient
Who cannot join
- Pregnant or lactating
- Requiring nutritional support (including enteral and 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 to comply with protocol requirements
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United Kingdom6 sites
Queen Elizabeth Hospital
Birmingham
Royal Hospital for Sick Children
Edinburgh
Queen Elizabeth University Hospital
Glasgow
Guys & St Thomas' Hospital
London
Royal Victoria Infirmary
Newcastle upon Tyne
Southampton General Hospital
Southampton
About this study
This study centres around a new one-a-day phenylalanine-free protein substitute for phenylketonuria patients. Fifty eligible adults (≥ 16 years) with proven phenylketonuria or hyperphenylalaninemia will be recruited and randomly allocated to one of two intervention arms (n = 25 per arm). Following a 3-day baseline period, and in addition to routine nutritional management, patients will receive either one sachet of the new protein substitute daily (intervention) or continue their usual dietary and/or protein substitute regimen (maximum of 1 protein substitute per day (equal to 20g protein equivalent) control) for 28 days.
Read the detailed description
This study centres around a new one-a-day phenylalanine-free protein substitute for phenylketonuria patients. In particular, this randomised controlled trial aims to evaluate the efficacy (changes relating to nutritional status and metabolic control) of this new protein substitute, while also capturing data pertaining to tolerance, compliance, safety and acceptability. Designed for poorly compliant adult patients with proven phenylketonuria or hyperphenylalaninemia, this new protein substitute is composed of an adapted mixture of other essential and non-essential amino acids, carbohydrates, vitamins and selected minerals and trace elements and enriched with docosahexaenoic acid (DHA). Fifty eligible adults (≥ 16 years) will be recruited and randomly allocated to one of two intervention arms (n = 25 per arm). Following a 3-day baseline period, and in addition to routine nutritional management, patients will receive either one sachet of the new protein substitute daily (intervention) or continue their usual dietary and/or protein substitute regimen (maximum of 1 protein substitute per day (equal to 20g protein equivalent) control) for 28 days.
- Conditions
- Phenylketonurias, Hyperphenylalaninaemia, Type I
- Being tested
- Synergy, Routine
- Keywords
- Phenylketonuria, Hyperphenylalaninaemia, Inherited Metabolic Disorders
- Medical subject headings
- Phenylketonurias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SynergyDietary supplement
Small serving (x1 33 g serving daily) of the new phenylalanine-free protein substitute made up with 100mL of water daily (28 days).
RoutineOther
Patients will continue their usual dietary and/or protein substitute regimen (28 days).
| Study group | Role | What they receive |
|---|---|---|
| Synergy | Experimental | This group will receive the new phenylalanine-free protein substitute daily for 28 days. Patients in this group intervention will be directed to consume one powder sachet (33 g) of daily made up with 100mL of water. The new substitute delivers 414 kJ, 20g protein equivalent and a combination of essential and non-essential amino acids as well a combination of vitamins and minerals. Receives: Dietary Supplement: Synergy |
| Routine | Active comparator | This group will continue their usual dietary and/or protein substitute regimen (maximum of 1 protein substitute per day (equal to 20g protein equivalent) control) for 28 days. Receives: Other: Routine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Nutritional Status (objective measure)
Blood micro nutrient levels; active Vitamin B12 (holotranscobalamin)
Measured: 2 days
Secondary outcomes (11)
Nutritional Status (Objective measure)
Blood micro nutrient levels; Folate (Folic Acid)
Measured: 2 days
Nutritional Status (Objective measure)
Blood micro nutrient levels; Iron (Ferritin)
Measured: 2 days
Nutritional Status (Objective measure)
Blood micro nutrient levels; Zinc
Measured: 2 days
Nutritional Status (Objective measure)
Blood micro nutrient levels; Vitamin D (25-hydroxyvitamin D)
Measured: 2 days
Nutritional Status (subjective measure)
3 day weighed food diary
Measured: 6 days
Daily compliance with prescribed protein substitute as assessed by standarised questionnaire
Compliance with the recommended intake of the patients previously prescribed protein substitute (during baseline in both groups, and during the study period in the control group) and with the study product during the intervention period will be assessed daily throughout the study. Patients will be asked to record how much of the protein substitute is taken compared to that recommended by their Health Care Professional. The daily amount prescribed by the Health Care Professional managing the patients care will be recorded at the start of the study and any changes to this prescription during the study will be noted. During the intervention period, protein substitute consumption patterns (e.g. timing and amount consumed) will be recorded daily and assessed b y a standardised questionnaire.
Measured: 31 days
Metabolic Control
Blood amino acid levels; Collected samples will be analysed for blood phenylalanine, tyrosine and 16 other proteinogenic amino acids, 2 non-proteinogenic amino acids and 1 amino sulfonic acid.
Measured: 2 days
Gastrointestinal tolerance as assessed by standarised questionnaire
Gastrointestinal tolerance (including diarrhoea, constipation, nausea, vomiting, abdominal pain, bloating, flatulence and burping) will be assessed using a standardised gastrointestinal tolerance questionnaire completed by the patient.
Measured: 12 days
Acceptability as assessed by standarised questionnaire
Acceptability (ease of use and liking) of the patients previously prescribed protein substitute and the study product will be assessed at the end of baseline (day 4) and the end of the intervention period (day 31) using a standardised questionnaire completed by the patient.
Measured: 2 days
Subjective Mood
Profile of mood states questionnaire
Measured: 3 days
Anthropometry
Measures of weight and height will be made during baseline observations (day 1) and at the end of the intervention period (day 31). Weight will be determined to the nearest 0.1 kg, using portable scales shoeless and wearing light weight clothing.
Measured: 2 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 May 2017
- Study start1 May 2017
- Primary completion20 Sep 2018
- Study completion17 Dec 2018
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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nutricia UK Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Rebecca Stratton, PhD, Principal investigator, Nutricia UK
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03167697
- Sponsor's protocol ID
- PKUSyn.2017
- Status last verified
- Mar 2019
- First submitted
- 22 May 2017
- Last updated
- 13 Mar 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: No IPD will be shared with other researchers.
Study at a glance
From the registry record, last updated by the sponsor on 13 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluating the Efficacy of PKU Synergy in Patients Expressing Phenylketonuria or Hyperphenylalaninemia
- ClinicalTrials.gov ID
- NCT03167697
- Acronym / sponsor ID
- PKUSyn.2017
- Conditions
- Phenylketonurias, Hyperphenylalaninaemia, Type I
- Keywords
- Phenylketonuria, Hyperphenylalaninaemia, Inherited Metabolic Disorders
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Nutricia UK LtdIndustry (pharma, biotech, devices)
- Study start
- 1 May 2017 (actual)
- Primary completion
- 20 Sep 2018 (actual)
- Study completion
- 17 Dec 2018 (actual)
- First posted
- 30 May 2017 (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 centres around a new one-a-day phenylalanine-free protein substitute for phenylketonuria patients. Fifty eligible adults (≥ 16 years) with proven phenylketonuria or hyperphenylalaninemia will be recruited and randomly allocated to one of two intervention arms (n = 25 per arm). Following a 3-day baseline period, and in addition to routine nutritional management, patients will receive either one sachet of the new protein substitute daily (intervention) or continue their usual dietary and/or protein substitute regimen (maximum of 1 protein substitute per day (equal to 20g protein equivalent) control) for 28 days.
Detailed description
This study centres around a new one-a-day phenylalanine-free protein substitute for phenylketonuria patients. In particular, this randomised controlled trial aims to evaluate the efficacy (changes relating to nutritional status and metabolic control) of this new protein substitute, while also capturing data pertaining to tolerance, compliance, safety and acceptability. Designed for poorly compliant adult patients with proven phenylketonuria or hyperphenylalaninemia, this new protein substitute is composed of an adapted mixture of other essential and non-essential amino acids, carbohydrates, vitamins and selected minerals and trace elements and enriched with docosahexaenoic acid (DHA). Fifty eligible adults (≥ 16 years) will be recruited and randomly allocated to one of two intervention arms (n = 25 per arm). Following a 3-day baseline period, and in addition to routine nutritional management, patients will receive either one sachet of the new protein substitute daily (intervention) or continue their usual dietary and/or protein substitute regimen (maximum of 1 protein substitute per day (equal to 20g protein equivalent) control) for 28 days.
Study design
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Synergy | Experimental | Dietary Supplement: SynergyThis group will receive the new phenylalanine-free protein substitute daily for 28 days. Patients in this group intervention will be directed to consume one powder sachet (33 g) of daily made up with 100mL of water. The new substitute delivers 414 kJ, 20g protein equivalent and a combination of essential and non-essential amino acids as well a combination of vitamins and minerals. |
| Routine | Active comparator | Other: RoutineThis group will continue their usual dietary and/or protein substitute regimen (maximum of 1 protein substitute per day (equal to 20g protein equivalent) control) for 28 days. |
Who may qualify
- Ages
- 16 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Male or female
- Over 16 years of age
- Diagnosed with proven PKU or hyperphenylalaninemia with an increased phenylalanine-tolerance/intake
- Currently taking a maximum of 1 protein substitute per day (equal to 20g protein equivalent)
- Have a minimum blood phenylalanine level of ≥ 600 umol/L (for PKU patients)
Key exclusion criteria (6 listed)
- Pregnant or lactating
- Requiring nutritional support (including enteral and 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
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 |
|---|---|---|
| SynergySmall serving (x1 33 g serving daily) of the new phenylalanine-free protein substitute made up with 100mL of water daily (28 days). | Dietary supplement | Synergy |
| RoutinePatients will continue their usual dietary and/or protein substitute regimen (28 days). | Other | Routine |
Primary outcome
Nutritional Status (objective measure)
Time frame: 2 days
Blood micro nutrient levels; active Vitamin B12 (holotranscobalamin)
11 secondary outcomes
Nutritional Status (Objective measure)
Time frame: 2 days
Nutritional Status (Objective measure)
Time frame: 2 days
Nutritional Status (Objective measure)
Time frame: 2 days
Nutritional Status (Objective measure)
Time frame: 2 days
Nutritional Status (subjective measure)
Time frame: 6 days
Daily compliance with prescribed protein substitute as assessed by standarised questionnaire
Time frame: 31 days
Metabolic Control
Time frame: 2 days
Gastrointestinal tolerance as assessed by standarised questionnaire
Time frame: 12 days
Acceptability as assessed by standarised questionnaire
Time frame: 2 days
Subjective Mood
Time frame: 3 days
Anthropometry
Time frame: 2 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 13 Mar 2019.
- Individual participant dataSharing planned: No. No IPD will be shared with other researchers.
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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.
Rebecca Stratton, PhD principal investigator
Nutricia UK
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Synergy and Routine in people with Phenylketonurias and Hyperphenylalaninaemia, Type I, run by Nutricia UK 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 7 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 13 Mar 2019. 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?
Nutritional Status (objective measure) (time frame: 2 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 13 Mar 2019.