Evaluating a new dietary product for managing phenylketonuria in children
An Acceptability Study to Evaluate the Compliance, Gastrointestinal Tolerance, Palatability and Metabolic Control of Children With Phenylketonuria (PKU) When Using PKU UP (a Food for Special Medical Purposes) as Part of Dietary Management.
NA · Vitaflo International, Ltd · NCT05995717
This study is testing a new dietary product called PKU UP to see if it helps children with phenylketonuria manage their condition better.
Quick facts
| Phase | NA |
|---|---|
| Study type | Interventional |
| Enrollment | 15 (estimated) |
| Ages | 1 Year to 10 Years |
| Sex | All |
| Sponsor | Vitaflo International, Ltd (industry) |
| Locations | 6 sites (Belfast and 5 other locations) |
| Trial ID | NCT05995717 on ClinicalTrials.gov |
What this trial studies
This study evaluates the acceptability of PKU UP, a food for special medical purposes, for dietary management in children aged 1-10 years with phenylketonuria (PKU). It is a prospective, single-arm, open-label study lasting 26 weeks, divided into two parts: a 12-week introduction phase and a 14-week continuation phase. Participants will be monitored for adherence, gastrointestinal symptoms, and overall nutritional status while using PKU UP as a protein substitute. Data will be collected through questionnaires and case report forms completed by investigators and parents/guardians.
Who should consider this trial
Good fit: Ideal candidates are children aged 1-10 years diagnosed with PKU who require a low-protein diet.
Not a fit: Patients with mild PKU not requiring a low-protein diet or those unable to comply with the study protocol may not benefit.
Why it matters
Potential benefit: If successful, this study could improve dietary management and quality of life for children with PKU.
How similar studies have performed: While dietary management for PKU is established, this specific approach with PKU UP is novel and has not been extensively tested in prior studies.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Diagnosis of PKU requiring a low-protein diet and protein substitute. * Participants who are adherent with their blood phenylalanine (Phe) testing, in the opinion of the investigator. * Participants aged 1-10 years. * Participants who in the opinion of the investigator are anticipated to be able to take at least half of their daily protein equivalent requirement from PKU UP (in order to assess this, a taste test (or similar) may be conducted at investigator's discretion as part of the screening process). * Willingly given, written, informed consent from the parent(s)/guardian(s). * Willingly given, written assent by the participant (if appropriate). Exclusion Criteria: * Inability to comply with the study protocol, in the opinion of the investigator. * Any co-morbidity, which, in the opinion of the investigator, would preclude participation in the study. * Diagnosis of persistent hyperphenylalaninemia, or mild PKU not requiring a low protein diet and protein substitute. * Allergy or intolerance to milk. * Participants who are currently participating in, plan to participate in or have participated in an interventional investigational drug, food or medical device study within 30 days prior to the screening visit. * Use of additional micronutrient supplements during the evaluation period, unless clinically indicated and prescribed by the Investigator (must be recorded in both the medical records and case report form).
Where this trial is running
Belfast and 5 other locations
- Royal Belfast Hospital for Sick Children — Belfast, United Kingdom (RECRUITING)
- Birmingham Children's Hospital — Birmingham, United Kingdom (RECRUITING)
- St. Luke's Hospital — Bradford, United Kingdom (NOT_YET_RECRUITING)
- University Hospitals Bristol and Weston NHS Foundation Trust — Bristol, United Kingdom (RECRUITING)
- Great Ormond Street Hospital for Children NHS Foundation Trust — London, United Kingdom (RECRUITING)
- Nottingham Children's Hospital — Nottingham, United Kingdom (RECRUITING)
Study contacts
- Principal investigator: Anita MacDonald — Birmingham Women's and Children's NHS Foundation Trust
- Study coordinator: Clinical Trials
- Email: ClinicalTrialsTeam@Vitaflo.co.uk
- Phone: 0151 709 9020
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: PKU, Phenylketonuria, Glyco-Macro-Peptide, GMP, amino acid, AA, protein substitute, PS