Status:

ACTIVE_NOT_RECRUITING

Evaluation of PKU UP

Lead Sponsor:

Vitaflo International, Ltd

Collaborating Sponsors:

Birmingham Women's and Children's NHS Foundation Trust

Conditions:

PKU

Eligibility:

All Genders

1-10 years

Phase:

NA

Brief Summary

PKU UP is a prospective, single-arm, open-label, 26-week acceptability study to evaluate PKU UP for the dietary management of participants with phenylketonuria (PKU). Up to 15 participants aged 1 - 10...

Detailed Description

PKU is a rare inborn error of metabolism with a prevalence of 1 in 10,000 in the UK population. The mainstay of treatment for most individuals is dietary therapy with a strict protein-restricted diet....

Eligibility Criteria

Inclusion

  • 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

  • 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).

Key Trial Info

Start Date :

January 11 2024

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

May 31 2026

Estimated Enrollment :

16 Patients enrolled

Trial Details

Trial ID

NCT05995717

Start Date

January 11 2024

End Date

May 31 2026

Last Update

November 28 2025

Active Locations (7)

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Page 1 of 2 (7 locations)

1

Royal Belfast Hospital for Sick Children

Belfast, United Kingdom, BT12 6BE

2

Birmingham Children's Hospital

Birmingham, United Kingdom, B4 6NH

3

St. Luke's Hospital

Bradford, United Kingdom, BD5 0NA

4

University Hospitals Bristol and Weston NHS Foundation Trust

Bristol, United Kingdom, BS2 8BJ

Evaluation of PKU UP | DecenTrialz