PhenylketonuriasNCT04272736
PKU Low Calorie Drink Study
A study testing Low calorie protein substitute in people with Phenylketonurias and Hyperphenylalaninaemia, run by Nutricia UK Ltd.
Trial summary
This study will evaluate the compliance, acceptability, gastrointestinal (GI) tolerance and safety of a lower calorie amino acid based liquid protein substitute for patients with Phenylketonuria (PKU) or hyperphenylalaninemia (HPA). Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- Phenylketonurias, Hyperphenylalaninaemia
- What is being tested?
- Low calorie protein substitute
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 1 Dec 2023to 31 Dec 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
- Male or female
- 3 years of age or above
- Diagnosed with classical or variant type phenylketonuria (PKU), or hyperphenylalaninemia (HPA)
- Have been compliant in taking at least one amino acid-based, Phe-free protein substitute, providing at least 20g protein equivalent daily, for at least 1 month prior to study commencement
- Have a prescribed daily Phe allowance
- Written informed consent from patient, or from parent / carer if applicable
Who cannot join
- Pregnant or lactating
- Requiring enteral tube or parenteral nutrition
- Major hepatic or renal dysfunction
- Participation in other studies within 1 month prior to entry to this study
- Allergy to any of the study product ingredients
- Investigator concern around the patient being underweight (e.g.: BMI lower than 18.5kg/m2 for adults) and/or having an eating disorder
- Investigator concern around willingness/ability of patient or parent/carer to comply with protocol requirements
About this study
Official title: Evaluating the Compliance, Acceptability, Safety and Tolerance of a Lower Calorie Protein Substitute for the Dietary Management of Phenylketonuria in Children and Adults - a Pilot Study
This study will evaluate the compliance, acceptability, gastrointestinal (GI) tolerance and safety of a lower calorie amino acid based liquid protein substitute for patients with Phenylketonuria (PKU) or hyperphenylalaninemia (HPA).
- Conditions
- Phenylketonurias, Hyperphenylalaninaemia
- Being tested
- Low calorie 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.
Low calorie protein substituteDietary supplement
The evaluated dietary supplement is a lower calorie amino acid based liquid protein substitute designed for patients with PKU.
| Study group | Role | What they receive |
|---|---|---|
| Low calorie protein substitute | Experimental | Single arm designed, 3 day baseline, 28 day on the lower calorie amino acid based liquid protein substitute. Receives: Dietary Supplement: Low calorie protein substitute |
What is being measured
The primary outcome is the main question the study is designed to answer.
Compliance
Questionnaire on amounts offered and amounts actually consumed, compared to recommended amount. Amounts (values) are recorded
Measured: Daily for 31 days
Secondary outcomes (5)
Acceptability: Tick-box questionnaire
Tick-box questionnaire on overall liking and acceptability of product (ie dislike a lot, dislike moderately, neutral, like moderately, like a lot)
Measured: Recorded at baseline and end of intervetnion (day 31)
Blood phenylalanine and other amino acid levels
Blood spot test.
Measured: Recorded at baselien and end of intervention (day 31)
Gastro-intestinal tolerance
Questionnaire detailing any GI symptoms, severity (none, mild, moderate, severe) and change from usual.
Measured: recorded at baseline, beginning of intervention and end of intervention(12 out of 31 days in total)
Nutrient intake
24h dietary recall.
Measured: Recorded at basleine and end of intervention (day 31)
Anthropometry
Measurements of height (m) and weight (kg) at baseline and end of study.
Measured: Recorded at baseline and end of intervention (day 31)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Feb 2020
- Study start1 Dec 2023
- Primary completion31 Dec 2023
- Study completion31 Dec 2023
Why it stopped: Study not started, not funded
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
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04272736
- Sponsor's protocol ID
- PKULite2018
- Status last verified
- Feb 2025
- First submitted
- 29 Aug 2018
- Last updated
- 26 Feb 2025
- 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 26 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluating the Compliance, Acceptability, Safety and Tolerance of a Lower Calorie Protein Substitute for the Dietary Management of Phenylketonuria in Children and Adults - a Pilot Study
- ClinicalTrials.gov ID
- NCT04272736
- Acronym / sponsor ID
- PKULite2018
- Conditions
- Phenylketonurias, Hyperphenylalaninaemia
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Study not started, not funded
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Nutricia UK LtdIndustry (pharma, biotech, devices)
- Study start
- 1 Dec 2023 (actual)
- Primary completion
- 31 Dec 2023 (actual)
- Study completion
- 31 Dec 2023 (actual)
- First posted
- 17 Feb 2020 (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 will evaluate the compliance, acceptability, gastrointestinal (GI) tolerance and safety of a lower calorie amino acid based liquid protein substitute for patients with Phenylketonuria (PKU) or hyperphenylalaninemia (HPA).
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Low calorie protein substitute | Experimental | Dietary Supplement: Low calorie protein substituteSingle arm designed, 3 day baseline, 28 day on the lower calorie amino acid based liquid protein substitute. |
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
- 3 years of age or above
- Diagnosed with classical or variant type phenylketonuria (PKU), or hyperphenylalaninemia (HPA)
- Have been compliant in taking at least one amino acid-based, Phe-free protein substitute, providing at least 20g protein equivalent daily, for at least 1 month prior to study commencement
- Have a prescribed daily Phe allowance
Key exclusion criteria (7 listed)
- Pregnant or lactating
- Requiring enteral tube or parenteral nutrition
- Major hepatic or renal dysfunction
- Participation in other studies within 1 month prior to entry to this study
- Allergy to any of the study product ingredients
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 |
|---|---|---|
| Low calorie protein substituteThe evaluated dietary supplement is a lower calorie amino acid based liquid protein substitute designed for patients with PKU. | Dietary supplement | Low calorie protein substitute |
Primary outcome
Compliance
Time frame: Daily for 31 days
Questionnaire on amounts offered and amounts actually consumed, compared to recommended amount. Amounts (values) are recorded
5 secondary outcomes
Acceptability: Tick-box questionnaire
Time frame: Recorded at baseline and end of intervetnion (day 31)
Blood phenylalanine and other amino acid levels
Time frame: Recorded at baselien and end of intervention (day 31)
Gastro-intestinal tolerance
Time frame: recorded at baseline, beginning of intervention and end of intervention(12 out of 31 days in total)
Nutrient intake
Time frame: Recorded at basleine and end of intervention (day 31)
Anthropometry
Time frame: Recorded at baseline and end of intervention (day 31)
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 26 Feb 2025.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A study testing Low calorie protein substitute in people with Phenylketonurias and Hyperphenylalaninaemia, 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 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: withdrawn, as of the sponsor's last update on 26 Feb 2025. 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?
Compliance (time frame: Daily for 31 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 26 Feb 2025.