PhenylketonuriasNCT03333720
Compliance, Tolerance and Acceptability of a Tablet Protein Substitute for the Dietary Management of Phenylketonuria
A study testing Phenylalanine-free protein substitute tablets in people with Phenylketonurias, run by Nutricia UK Ltd.
Trial summary
Thirty eligible PKU patients (≥ 8 years), identified as tablet protein substitute users, will be recruited. Patients will firstly observe a 7-day baseline period, in which participants will continue with their existing diet, protein substitute and multi vitamin prescription. Read more about this study
- Who is it for?
- Ages 8 to 100 years
- Conditions the study is for
- Phenylketonurias
- What is being tested?
- Phenylalanine-free protein substitute tablets
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 15 Jul 2019to 31 Dec 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 8 to 100 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male or female
- 8 years of age and older
- Diagnosed with proven PKU
- Consume a minimum of 20g protein equivalent in tablet format while enrolled on to the trial
- If routine care and dietary management is not different from normal PKU care and dietary management, preconception patients may be considered
- Have written informed consent from patient or parent/carer carer
Who cannot join
- Requiring nutritional support (including enteral and parenteral nutrition)
- Major hepatic or renal dysfunction
- Maternal PKU patients (if a patient becomes pregnant during the study they will be withdrawn on immediate notice to the Metabolic Dietitian responsible for the patient's care)
- Participation in other studies within 1 month prior to entry of this study
- Investigator concern around willingness/ability of patient to comply with protocol requirements
About this study
Thirty eligible PKU patients (≥ 8 years), identified as tablet protein substitute users, will be recruited. Patients will firstly observe a 7-day baseline period, in which participants will continue with their existing diet, protein substitute and multi vitamin prescription. Patients will then receive the phaenylalanine-free protein substitute tablets daily for 28 days in addition to appropriate nutritional management. The aim of this prospective, single arm trial is to evaluate the acceptability (compliance, tolerance and palatability) of the phenylalanine-free protein substitute tablets in patients with proven PKU. The primary outcome measure is compliance, with secondary outcome measures of gastrointestinal tolerance, acceptability, blood amino acids, dietary intake, anthropometry and safety.
Read the detailed description
Protein substitutes are typically presented in powder format and reconstituted in water to a set volume, but may also be offered in ready-to-drink formats or as a gel. Notwithstanding recent advancements related to the taste, scent and texture (organoleptic properties) of commercially available protein substitutes, a small proportion of PKU patients choose to consume tablet-based protein substitutes. In this sense, anecdotal evidence suggests that in the UK approximately 80-100 PKU patients consume tablet protein substitutes. Despite the need to consume large quantities, protein substitutes in tablet format may help overcome some of the aforementioned limitations compared to powdered or ready-to-drink preparations, but also offer additional benefits including convenience and reduced preparation.
Although usage of tablet-based protein substitutes is low in comparison to powdered preparations, early evidence from MacDonald and colleagues suggests tablet protein substitutes are an equally effective strategy to manage PKU in older children, teenagers and adults. Tablet-based protein substitutes, however, only offer a balanced mixture of essential and non-essential amino acids. Considering the daily burden and discipline required to follow the restrictive low-protein diet, reports suggest achieving full compliance with tablet-based protein substitutes is difficult, and this becomes especially challenging when given alongside multi vitamin supplements. To elaborate, patients solely consuming protein substitutes in tablet format may be required to consume 70-140 tablets per day (average = 75 tablets per day) alongside an additional 5 prescribable vitamin and mineral tablets. In this instance, it may be beneficial if tablet-based protein substitutes combined amino acids with vitamins, minerals and trace elements.
At present, tablets combining amino acids with vitamins, minerals and trace elements are currently unavailable. As such, the sponsor has produced a tablet-based protein substitute designed for individuals aged 8 years and older. Developed specifically for the dietary management of proven PKU, the phenylalanine-free protein substitute tablets comprise an adapted mixture of essential and non-essential amino acids (excluding phenylalanine) and are combined with carbohydrates, DHA, vitamins and selected minerals and trace elements. As such, the aim of this intervention trial is therefore to evaluate the acceptability (compliance, gastrointestinal tolerance and palatability) of the tablets, while also capturing information related to blood amino acid profiles, dietary intake, anthropometry and safety as part of a low phenylalanine dietary regimen in PKU patients. Thirty eligible PKU patients (≥ 8 years), identified as current tablet users, will be recruited. Patients will firstly observe a 7-day baseline period. During baseline, patients will continue with their existing diet and protein substitute prescription before receiving the tablets daily for 28 days in addition to appropriate nutritional management.
- Conditions
- Phenylketonurias
- Being tested
- Phenylalanine-free protein substitute tablets
- Keywords
- Phenylketonuria, 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.
Phenylalanine-free protein substitute tabletsDietary supplement
Thirty eligible PKU patients (≥ 8 years), identified as current tablet users, will be recruited to this study. Patients will firstly observe a 7-day baseline period. During baseline, patients will continue with their existing diet and protein substitute prescription before receiving Phenylalanine-free protein subsitute tablets daily for 28 days in addition to appropriate nutritional management.
| Study group | Role | What they receive |
|---|---|---|
| Intervention | Experimental | Intervention is phenylalanine-free protein substitute. Following a 7 day baseline period, all recruits will receive the new phenylalanine-free protein substitute daily for 28 days in addition to routine nutritional management. The study product prescription will be specified on an individual basis by the metabolic Dietitian responsible for the patient's nutritional management and will be dependent on age, bodyweight and medical condition of the patient, but will wholly replace their currently prescribed tablet protein substitute and multivitamin supplements. Receives: Dietary Supplement: Phenylalanine-free protein substitute tablets |
What is being measured
The primary outcome is the main question the study is designed to answer.
Daily Compliance
Compliance with currently prescribed protein substitute and prescribed multivitamin supplements will be assessed daily from days 1-7 and recorded as a percentage of tablets consumed comapred to prescription (0-100%). Compliance with the study product will subsequently be assessed daily from days 8-35. Compliance throughout the trial will be assessed using standarised questionnaires, where patients document number of med versus number of tablets prescribed. A percentage compliance level based on per patient and total patients will be computed to define overall compliance where a higher percentage represents better overall compliance.
Measured: 35 days
Secondary outcomes (7)
Treatment-Emergent tolerability
Gastrointestinal tolerance (including diarrhoea, constipation, nausea, vomiting, abdominal pain, bloating, flatulence and burping) will be assessed via questionnaire using the scale (absent, mild, moderate, severe).
Measured: 12 days
Patient Acceptability
Acceptability (ease of use and palatability) will be assessed via questionnaire.
Measured: 2 days
Metabolic control
Blood phenylalanine, blood tyrosine and other amino acids. Fingertip-capillary blood samples will be collected in the fasted state at baseline observations (day 1), and on the last day of the intervention period (day 35). 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
Nutritional Intake
To assess habitual dietary intake, 24 hour dietary recalls will be completed during baseline and at the end of the intervention period.
Measured: 2 days
Weight
Measures of weight (kg) will be made during baseline observations and at the end of the intervention period.
Measured: 2 days
Height
Measures of weight (kg) will be made during baseline observations and at the end of the intervention period.
Measured: 2 days
Incidence of study product emergent adverse events [safety].
All adverse events will be recorded throughout the study (as and when they occur). Consequently, there is no specific time frame as the frequency of adverse events can not be predicted.
Measured: Baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Nov 2017
- Study start15 Jul 2019 (estimated)
- Primary completion30 Dec 2019 (estimated)
- Study completion31 Dec 2019 (estimated)
Why it stopped: Study halted prematurely, prior to enrollment of first participant
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
- Masking
- None (open label)
- Enrolment
- 0 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, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03333720
- Sponsor's protocol ID
- PKU.LP2017
- Status last verified
- Mar 2019
- First submitted
- 18 Sep 2017
- Last updated
- 13 Mar 2019
- Results posted
- 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
- Compliance, Tolerance and Acceptability of a Tablet Protein Substitute for the Dietary Management of Phenylketonuria
- ClinicalTrials.gov ID
- NCT03333720
- Acronym / sponsor ID
- PKU.LP2017
- Conditions
- Phenylketonurias
- Keywords
- Phenylketonuria, Inherited Metabolic Disorders
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Study halted prematurely, prior to enrollment of first participant
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Nutricia UK LtdIndustry (pharma, biotech, devices)
- Study start
- 15 Jul 2019 (estimated)
- Primary completion
- 30 Dec 2019 (estimated)
- Study completion
- 31 Dec 2019 (estimated)
- First posted
- 7 Nov 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.
Thirty eligible PKU patients (≥ 8 years), identified as tablet protein substitute users, will be recruited. Patients will firstly observe a 7-day baseline period, in which participants will continue with their existing diet, protein substitute and multi vitamin prescription. Patients will then receive the phaenylalanine-free protein substitute tablets daily for 28 days in addition to appropriate nutritional management. The aim of this prospective, single arm trial is to evaluate the acceptability (compliance, tolerance and palatability) of the phenylalanine-free protein substitute tablets in patients with proven PKU. The primary outcome measure is compliance, with secondary outcome measures of gastrointestinal tolerance, acceptability, blood amino acids, dietary intake, anthropometry and safety.
Detailed description
Protein substitutes are typically presented in powder format and reconstituted in water to a set volume, but may also be offered in ready-to-drink formats or as a gel. Notwithstanding recent advancements related to the taste, scent and texture (organoleptic properties) of commercially available protein substitutes, a small proportion of PKU patients choose to consume tablet-based protein substitutes. In this sense, anecdotal evidence suggests that in the UK approximately 80-100 PKU patients consume tablet protein substitutes. Despite the need to consume large quantities, protein substitutes in tablet format may help overcome some of the aforementioned limitations compared to powdered or ready-to-drink preparations, but also offer additional benefits including convenience and reduced preparation.
Although usage of tablet-based protein substitutes is low in comparison to powdered preparations, early evidence from MacDonald and colleagues suggests tablet protein substitutes are an equally effective strategy to manage PKU in older children, teenagers and adults. Tablet-based protein substitutes, however, only offer a balanced mixture of essential and non-essential amino acids. Considering the daily burden and discipline required to follow the restrictive low-protein diet, reports suggest achieving full compliance with tablet-based protein substitutes is difficult, and this becomes especially challenging when given alongside multi vitamin supplements. To elaborate, patients solely consuming protein substitutes in tablet format may be required to consume 70-140 tablets per day (average = 75 tablets per day) alongside an additional 5 prescribable vitamin and mineral tablets. In this instance, it may be beneficial if tablet-based protein substitutes combined amino acids with vitamins, minerals and trace elements.
At present, tablets combining amino acids with vitamins, minerals and trace elements are currently unavailable. As such, the sponsor has produced a tablet-based protein substitute designed for individuals aged 8 years and older. Developed specifically for the dietary management of proven PKU, the phenylalanine-free protein substitute tablets comprise an adapted mixture of essential and non-essential amino acids (excluding phenylalanine) and are combined with carbohydrates, DHA, vitamins and selected minerals and trace elements. As such, the aim of this intervention trial is therefore to evaluate the acceptability (compliance, gastrointestinal tolerance and palatability) of the tablets, while also capturing information related to blood amino acid profiles, dietary intake, anthropometry and safety as part of a low phenylalanine dietary regimen in PKU patients. Thirty eligible PKU patients (≥ 8 years), identified as current tablet users, will be recruited. Patients will firstly observe a 7-day baseline period. During baseline, patients will continue with their existing diet and protein substitute prescription before receiving the tablets daily for 28 days in addition to appropriate nutritional management.
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Intervention | Experimental | Dietary Supplement: Phenylalanine-free protein substitute tabletsIntervention is phenylalanine-free protein substitute. Following a 7 day baseline period, all recruits will receive the new phenylalanine-free protein substitute daily for 28 days in addition to routine nutritional management. The study product prescription will be specified on an individual basis by the metabolic Dietitian responsible for the patient's nutritional management and will be dependent on age, bodyweight and medical condition of the patient, but will wholly replace their currently prescribed tablet protein substitute and multivitamin supplements. |
Who may qualify
- Ages
- 8 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Male or female
- 8 years of age and older
- Diagnosed with proven PKU
- Consume a minimum of 20g protein equivalent in tablet format while enrolled on to the trial
- If routine care and dietary management is not different from normal PKU care and dietary management, preconception patients may be considered
Key exclusion criteria (5 listed)
- Requiring nutritional support (including enteral and parenteral nutrition)
- Major hepatic or renal dysfunction
- Maternal PKU patients (if a patient becomes pregnant during the study they will be withdrawn on immediate notice to the Metabolic Dietitian responsible for the patient's care)
- Participation in other studies within 1 month prior to entry of this study
- Investigator concern around willingness/ability of patient to comply with protocol requirements
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 |
|---|---|---|
| Phenylalanine-free protein substitute tabletsThirty eligible PKU patients (≥ 8 years), identified as current tablet users, will be recruited to this study. Patients will firstly observe a 7-day baseline period. During baseline, patients will continue with their existing diet and protein substitute prescription before receiving Phenylalanine-free protein subsitute tablets daily for 28 days in addition to appropriate nutritional management. | Dietary supplement | Intervention |
Primary outcome
Daily Compliance
Time frame: 35 days
Compliance with currently prescribed protein substitute and prescribed multivitamin supplements will be assessed daily from days 1-7 and recorded as a percentage of tablets consumed comapred to prescription (0-100%). Compliance with the study product will subsequently be assessed daily from days 8-35. Compliance throughout the trial will be assessed using standarised questionnaires, where patients document number of med versus number of tablets prescribed. A percentage compliance level based on per patient and total patients will be computed to define overall compliance where a higher percentage represents better overall compliance.
7 secondary outcomes
Treatment-Emergent tolerability
Time frame: 12 days
Patient Acceptability
Time frame: 2 days
Metabolic control
Time frame: 2 days
Nutritional Intake
Time frame: 2 days
Weight
Time frame: 2 days
Height
Time frame: 2 days
Incidence of study product emergent adverse events [safety].
Time frame: Baseline
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
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 study director
Nutricia, Inc.
Questions about this trial
What is this clinical trial studying?
A study testing Phenylalanine-free protein substitute tablets in people with Phenylketonurias, run by Nutricia UK Ltd.
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 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: withdrawn, 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.
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 was withdrawn before enrolling participants, so there are no results.
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?
Daily Compliance (time frame: 35 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.