ClinicalTrials.gov record NCT07802366
Market Research of a Slow Release Protein Substitute for the Dietary Management of PKU
XPhe Piccos - Acceptability and Tolerance Market Research
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This small, open-label study evaluates whether children with PKU or HPA tolerate and accept XPhe Piccos, a slow‑release phenylalanine‑free tablet. About 10 children will replace some or all of their usual protein substitute with the study product for 7 days. Caregivers or participants will complete daily questionnaires on use, taste, and gastrointestinal symptoms, and routine finger‑prick blood spots will be compared before and during the trial.
What it is studying. The study is measuring acceptability (taste, texture, ease of use), compliance with taking the product, gastrointestinal tolerability (nausea, diarrhoea, constipation, bloating, etc.), and routine metabolic control using finger‑prick blood spot tests for phenylalanine and tyrosine. It also records any adverse events that occur while participants use the product.
What the researchers hope to learn. To evaluate whether XPhe Piccos is acceptable and well tolerated by children with PKU/HPA, to document compliance and gastrointestinal side effects, to assess patient acceptability ratings (taste, texture, ease of use), and to compare routine phenylalanine and tyrosine blood spot results while using the study product.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
XPhePiccosMR is a prospective, single-arm, open label, observational study that aims to evaluate the acceptability and tolerability of XPhe Piccos, a slow-release, phenylalanine-free protein substitute in the form of tiny tablets, for use in the dietary management of phenylketonuria (PKU) in children aged three years and older.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Dietary supplement
- Slow release, phenylalanine-free protein substitute tablets
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- Diagnosis of PKU or HPA requiring a phenylalanine-free protein substitute.
- Subjects who are already taking a phenylalanine-free protein substitute and are willing to try the study product for 7 days.
- Subjects aged 3 years and above.
- Written informed assent/consent obtained from subject and/or parental caregiver, depending on subject age.
Exclusion criteria
- Presence of serious concurrent illness.
- Lead Dietitian's uncertainty about the willingness or ability of the patient to comply with the protocol requirements
- Participation in any other studies involving investigational or marketed products concomitantly or within two weeks prior to entry into the study.
- Any subject having taken antibiotics over the previous 2 weeks leading up to the study.
- Subjects less than 3 years of age
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Show the detailed description from the registry
The principle treatment for children with Phenylketonuria (PKU) or Hyperphenylalaninemia (HPA) is a low protein diet. Part of this treatment requires the administration of a protein substitute in order to meet basic protein requirements for normal growth and development. Several brands of protein substitutes for PKU/HPA are already available in various presentations. However, compliance with taking protein substitutes continues to be a challenge. As a low protein diet is usually recommended for life, long term compliance is always a major concern. As a result, improving the choice in terms of product type may aid compliance. XPhe Piccos is a slow release, phenylalanine-free protein substitute in a tiny tablet form, enriched with vitamins, minerals and trace elements and designed for children and adults with PKU/HPA. XPhe Piccos is a food for special medical purposes (FSMP), as defined by the Delegated Regulation (EU) 2016/128 of 25 September 2015 supplementing Regulation (EU) No 609/2013 of 12 June 2013, used for the dietary management of PKU/HPA. It is anticipated that XPhe Piccos will be a suitable alternative option for patients with PKU/HPA broadening the variety of protein substitutes they can choose from in order to suit their lifestyle and preferences. This is a prospective, single-arm, open-label, observational tolerance study with 10 children with PKU. Subjects who are currently taking a second stage protein substitute will be recruited for a 7-day trial, taking the new ready-to-use slow release, phenylalanine-free protein substitute tablets to evaluate the tolerability and acceptability of the study product. Therefore, subjects will replace some or all of their usual protein substitute with the new product. During the 7-day trial subjects or caregivers will be asked to complete a daily questionnaire recording information on:
- Usage and compliance
- Ease of use and any issues with administration
- Any gastro-intestinal side-effects.
A questionnaire will also be completed at the beginning and end of the study that will consider perceptions about taste, appearance, smell, ease of administration of the usually used product and the study product; how it is taken; and any other problems or symptoms. The amount of tablets prescribed will be calculated to provide the same amount of protein as their usual protein substitute. Subjects will continue to have weekly finger-prick blood spot tests as is routine in PKU. The results whilst on the study product will be compared with results whilst on their usual protein substitute. 10 children with PKU will be recruited. When an appropriate subject has been identified, a study information sheet will be sent to the subject or parents/ caregivers. They will be invited to request further information about the study if they wish by contacting the Lead Dietitian. Recruitment of each patient will be by written informed consent, which will be completed by the parents/ primary caregivers and taken by the Lead Dietitian. Children will also complete an assent/consent form and will receive an information sheet, if considered appropriate for their level of understanding.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
You (or your child) would take the study's slow‑release, phenylalanine‑free protein substitute tablets (XPhe Piccos) for 7 days, replacing some or all of your usual protein substitute; the tablet amount will be calculated to provide the same protein as your usual product. Caregivers or participants will complete a daily questionnaire about usage, ease of use, taste/acceptability and any gastro‑intestinal symptoms, plus a questionnaire at the start and end of the 7‑day period. Weekly routine finger‑prick blood spot tests for phenylalanine and tyrosine will be continued and the results while using the study product will be compared with results on the usual product; any adverse events will be recorded throughout the study. To join you must already be taking a phenylalanine‑free protein substitute and provide written informed consent/assent; the study is for ages 3 Years to 18 Years, and people with serious concurrent illness, recent antibiotic use (within 2 weeks), or recent participation in another study may be excluded.
How the study is structured. Interventional, single‑group, open‑label study. Around 10 children who normally take a second‑stage phenylalanine‑free protein substitute will use the slow‑release XPhe Piccos tablets for 7 days, replacing some or all of their usual product. Participants or caregivers complete daily standardized questionnaires about usage, acceptability, and any gastrointestinal symptoms; finger‑prick blood spots are taken as per routine and compared between usual product use and the 7‑day trial.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The registry says recruitment has not started. When is it expected to open, and can I register interest now?
- The listed sites are in United Kingdom. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on phenylketonuria
Important dates
- Study start
- Oct 15, 2026 (estimated)
- Primary completion
- May 1, 2027 (estimated)
- Study completion
- Aug 1, 2027 (estimated)
- Target enrollment
- 10 participants (estimated)
- Last updated on registry
- Oct 9, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- metaX Institut fuer Diatetik GmbH
- Collaborators
- Birmingham Women's and Children's NHS Foundation Trust
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Bernhard Hoffmann, PhD 📞 +49 6031 166 72 71 ✉ behoffmann@metax.org
- Yvonne Muecke, PhD 📞 +49 6031 166 72 79 ✉ yvonne.muecke@metax.org
Participating sites (1)
- Birmingham Children´s Hospital
Birmingham, England, B4 6NH · United Kingdom
- Anita MacDonald, Professor · +44 1213338024 · anita.macdonald@nhs.net
Terms used on this page · explanations by MedicalUpdates.ai
- single-arm
- All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
- open label
- No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
- observational study
- A study in which researchers do not assign participants to a treatment or other intervention. They observe or measure what happens, which may include study visits, tests, questionnaires or a review of medical records.
- assent
- A child's or dependent adult's agreement to take part, given alongside the formal consent of a parent or legal representative.
- investigational
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- written informed consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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