Clinical trial NCT03168399: Evaluation of PKU Explore
Official title: A Study to Evaluate the Acceptability of PKU Explore, a Renovated Phenylalanine Free Second Stage Protein Substitute for Use in the Dietary Management of Phenylketonuria in Infants From 6 Months to 5 Years of Age With Regard to Product Tolerance and Adherence.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Phenylketonuria
Inborn Errors of Metabolism
Keywords: Phenylketonuria
PKU
Metabolism
Metabolic
Control
Acceptability
Lead sponsor: Vitaflo International, Ltd
Sponsor class: INDUSTRY
Collaborators: name: Birmingham Women's and Children's NHS Foundation Trust
First posted: 2017-05-30
Enrollment: 22
Sex: ALL
Minimum age (years): 6
Maximum age (years): 5
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: To evaluate the acceptability, tolerance and effect on metabolic control of PKU Explore, a renovated Phe free protein substitute for the dietary management of PKU in children from 6 months to 5 years.

Detailed description: This is a prospective, acceptability study to evaluate the gastrointestinal tolerance, palatability and adherence of PKU explore in a four-week period for 15 participants aged between 6 months and 5 years for the dietary management of PKU.

The outcome of this assessment will be used in a submission to the regulatory authorities, Advisory Committee on Borderline Substances (ACBS), for PKU Start to become reimbursable on prescription in the UK.

The participant's dietitian will advise on an appropriate amount of PKU Explore based on individual requirements. Parents/carers will be asked to substitute their usual Phe-free protein substitute with PKU Explore for one (1) month. The sponsor will supply PKU Explore free of charge.

Parents/guardians will be asked to record information about the following:

Daily PKU Explore intake Stools Vomiting and Spit-up Feed / Fluid Intake Compliance Phenylalanine Levels (from their routine blood spot testing)

Final Evaluation about the presentation of the product, ease of preparation and use.

Eligibility criteria: Inclusion Criteria:

i. A diagnosis of PKU on new-born screening requiring a low protein diet and Phe-free L-amino acid supplements.

ii. Aged between 6 months and 5 years.

iii. Already taking a second stage concentrated protein substitute as part of their PKU management OR is at the stage in their PKU management when a second stage concentrated protein substitute is recommended to commence.

iv. Willingly given, written, informed consent from parent/guardian.

v. Willingly given, written assent (if appropriate).

Exclusion Criteria:

i. Diagnosis of persistent hyperphenylalaninaemia, or mild PKU not requiring dietary treatment with a low protein diet and Phe-free L-amino acid supplements.

ii. Diagnosis of a concurrent condition which may adversely affect developmental progression and feeding ability.

iii. Participation in any other clinical trial/acceptability study.

iv. Any serious medical precluding the study intervention.

v. Use of additional macro/micronutrient supplements during the study period, unless clinically indicated and prescribed by the investigator (must be recorded in patient case record file).

Primary outcomes:
measure: Product compliance daily diary
timeFrame: Days 1-28
measure: GI tolerance daily diary assessing change
timeFrame: Days 1-7 and 22-28
measure: Ease of use questionnaire
timeFrame: Day 29
measure: Weekly phenylalanine control
timeFrame: Days 1-28
Secondary outcomes:
Other outcomes:
Locations:
city: Glasgow
country: United Kingdom
facility: NHS Greater Glasgow and Clyde
state: Lanarkshire
city: Burringham
country: United Kingdom
facility: Birmingham Women's and Children's NHS Foundation Trust
state: West Midlands
city: Bradford
country: United Kingdom
facility: Bradford Teaching Hospitals NHS Foundation Trust
state: West Yorkshire
Study officials:
affiliation: Birmingham Women's and Children's NHS Foundation Trust
name: Anita MacDonald
role: PRINCIPAL_INVESTIGATOR
References: