Clinical trial NCT05995717: Evaluation of PKU UP
Official title: 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.
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: PKU
Keywords: Phenylketonuria
PKU
Glyco-Macro-Peptide
GMP
amino acid
AA
protein substitute
PS
United Kingdom
UK
children
PKU UP
Glycomacropeptide
Lead sponsor: Vitaflo International, Ltd
Sponsor class: INDUSTRY
Collaborators: name: Birmingham Women's and Children's NHS Foundation Trust
First posted: 2023-08-16
Enrollment: 15
Sex: ALL
Minimum age (years): 1
Maximum age (years): 10
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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 years old will be recruited and it is anticipated the study will open in at least three sites in the United Kingdom (UK) to recruit the target number of participants in the required timeframe.

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. We propose to recruit children with a diagnosis of PKU aged 1-10 years.

The study product, PKU UP, is a food for special medical purposes (FSMP), as defined by the Delegated Act EU 2016/128, used for the dietary management of Phenylketonuria (PKU).

The study will be investigating the acceptability of PKU UP, as defined by the Advisory Committee on Borderline Substances (ACBS). This includes the following:

* participant adherence to recommended intakes
* gastrointestinal symptoms
* evaluations of palatability.

Each participant will be on the study for 26 weeks. This includes evaluation part 1 and evaluation part 2:

* Part 1 consists of a 12 week period where participants introduce the study product into their diet. Gastrointestinal tolerance, metabolic control, compliance, product acceptability and dietary quality will be evaluated.
* Part 2 consists of a 14 week period where participants continue to take the study product for a longer assessment of growth and nutritional status.

Data collection will be performed using paper and/or electronic Case Report Forms completed by the investigators at the baseline, evaluation periods and end of study visits. There will also be three questionnaires completed by the parents/guardians over the course of the study, in relation to protein substitute intake, gastrointestinal tolerance, and product acceptability.

Eligibility 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).

Primary outcomes:
measure: Change in phe level between baseline and visit 3
timeFrame: 26 weeks
measure: Change in tyrosine level between baseline and visit 3
timeFrame: 26 weeks
measure: Change in PI impression of metabolic control
timeFrame: Week 0, week 6, week 12, week 26
measure: Change in the growth of participants as measured by Height (length in under 2 years of age), Head circumference in under 2 years of age and Weight.
timeFrame: Week 0, week 12, week 26
measure: Change in GI tolerance (participant)
timeFrame: Week 0, week 6, week 12, week 26
measure: Change in PI impression of GI tolerance
timeFrame: Week 0, week 6, week 12, week 26
measure: Change in compliance with study product consumption
timeFrame: Week 0, week 6, week 12, week 26
measure: Change in PI impression of study product intake
timeFrame: Week 0, week 6, week 12, week 26
measure: Change in product acceptability
timeFrame: Week 0, week 12, week 26
measure: Change in dietary intake
timeFrame: Week 0, week 12, week 26
measure: Change in nutritional biochemistry
timeFrame: Week 0, week 26
Secondary outcomes:
Other outcomes:
Locations:
city: Belfast
country: United Kingdom
facility: Royal Belfast Hospital for Sick Children
city: Birmingham
country: United Kingdom
facility: Birmingham Children's Hospital
city: Bradford
country: United Kingdom
facility: St. Luke's Hospital
city: Bristol
country: United Kingdom
facility: University Hospitals Bristol and Weston NHS Foundation Trust
city: London
country: United Kingdom
facility: Great Ormond Street Hospital for Children NHS Foundation Trust
city: Nottingham
country: United Kingdom
facility: Nottingham Children's Hospital
Study officials:
affiliation: Birmingham Women's and Children's NHS Foundation Trust
name: Anita MacDonald
role: PRINCIPAL_INVESTIGATOR
References: