Clinical trial NCT05096988: Evaluation of PKU Sphere Liquid
Official title: A Study to Evaluate the Acceptability, Tolerance and Adherence of Children and Adults Consuming PKU Sphere Liquid, a Food for Special Medical Purposes (FSMP), for the Dietary Management of Phenylketonuria (PKU)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Phenylketonurias
Keywords: PKU
Sphere
Liquid
Lead sponsor: Vitaflo International, Ltd
Sponsor class: INDUSTRY
First posted: 2021-10-27
Enrollment: 19
Sex: ALL
Minimum age (years): 3
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: PKU Sphere Liquid is a prospective, open-label, acceptability study to evaluate PKU sphere liquid in up to 15 participants aged 3 and above for the dietary management of PKU over 31 days.

Detailed description: PKU Sphere Liquid is a newly-developed Food for Special Medical Purposes (FSMP) designed for the dietary management of patients with phenylketonuria (PKU).

PKU sphere liquid is a vanilla flavoured, ready-to-drink, low phenylalanine protein substitute containing a blend of casein glycomacropeptide (GMP) isolate, essential and non-essential amino acids, carbohydrate, fat, vitamins, minerals, and Docosachexaenoic acid (DHA).

This is a feasibility study designed to evaluate the acceptability, tolerance and adherence of children and adults consuming PKU sphere liquid.

Participants will be given a four-week supply of PKU Sphere Liquid and they will be asked to complete a daily diary and short questionnaire for four weeks to record information on: adherence, gastrointestinal tolerance, palatability and how the product is used.

Each participant will be on the trial for 31 days. This includes a 3-day baseline period and 28 days taking the study product. If deemed appropriate in each individual circumstance, the sponsor of the trial, Vitaflo International Ltd, will continue to supply the product free of charge at the end of the trial until available on prescription.

Eligibility criteria: Inclusion Criteria:

* Diagnosis of PKU requiring a low protein diet and protein substitute.
* Aged three (3) years and over
* Already taking a minimum of one GMP based protein substitute per day or have previously taken one
* Able to comply with the study protocol and take the study product, according to the opinion of the PI.
* Willingly given, written, informed consent from patient or parent/guardian.
* Willingly given, written assent (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 hyperphenylalaninaemia, or mild PKU not requiring dietary intervention with a low protein diet and protein supplement.
* Unwilling/unable to take a GMP based protein substitute.
* Women who are pregnant / breastfeeding at the start of the study or planning to become pregnant during the study period.

N.B.: Women who become pregnant unexpectedly during this study will be immediately withdrawn from the study.

Primary outcomes:
measure: Questionnaire of self-reported adherence to the prescribed amount of study product
timeFrame: 4 weeks
measure: Product acceptability rated on a Likert scale by the participant after four week intake
timeFrame: 4 weeks
measure: Questionnaire of self-reported changes in gastrointestinal tolerance during four week intake
timeFrame: 4 weeks
Secondary outcomes:
Other outcomes:
Locations:
city: Belfast
country: United Kingdom
facility: Royal Victoria Hospital
city: Birmingham
country: United Kingdom
facility: Birmingham Children's Hospital
city: Sheffield
country: United Kingdom
facility: Northern General Hospital
Study officials:
affiliation: Birmingham Women's and Children's NHS Foundation Trust
name: Anita Macdonald
role: PRINCIPAL_INVESTIGATOR
References: