Evaluation of PKU Explore

NCT03168399 | Completed

• 2 other identifiers: MCT-PKUE-2016-12-05219119
• Study Type: interventional
• Target: 22
• Locations: 1 country
• Sites: 3

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.

Conditions

Phenylketonuria; Inborn Errors of Metabolism

Keywords

Phenylketonuria; PKU; Metabolism; Metabolic; Control; Acceptability

Outcome Measures

Primary Outcomes (4)

• Product compliance daily diary

  Quantitative assessments from subject questionnaires that allow evaluation of compliance with the study product, i.e. actual versus prescribed intake.

  Days 1-28

• GI tolerance daily diary assessing change

  Qualitative assessments from subject questionnaires that allow evaluation of any change in the gastro-intestinal tolerance of the study product.

  Days 1-7 and 22-28

• Ease of use questionnaire

  Qualitative assessment from subject questionnaire that allows evaluation of the ease of use of the study product.

  Day 29

• Weekly phenylalanine control

  Collection of quantitative data regarding phenylalanine control using routine biochemical testing

  Days 1-28

Study Arms (1)

Consumption of PKU Explore

EXPERIMENTAL

Daily feed, substituting the participant's normal phe-free protein substitute for PKU Explore.

Dietary Supplement: PKU Explore

Interventions

PKU Explore DIETARY_SUPPLEMENT

PKU Explore is an unflavoured, powdered, phenylalanine-free, protein substitute, containing essential and non-essential amino acids, carbohydrate, sugar, vitamins, minerals, trace elements and the long chain polyunsaturated fatty acids (LCPs); arachidonic acid (AA) and docosahexaenoic acid (DHA).

Consumption of PKU Explore

Eligibility Criteria

• Age: 6 Months - 5 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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).

You may not qualify if:

• 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).

Sponsors & Collaborators

• Vitaflo International, Ltd: lead
• Birmingham Women's and Children's NHS Foundation Trust: collaborator

Study Sites (3)

NHS Greater Glasgow and Clyde

Glasgow, Lanarkshire, G51 4TF, United Kingdom

Location

Birmingham Women's and Children's NHS Foundation Trust

Burringham, West Midlands, B4 6NH, United Kingdom

Location

Bradford Teaching Hospitals NHS Foundation Trust

Bradford, West Yorkshire, BD5 0NA, United Kingdom

Location

MeSH Terms

Conditions

Phenylketonurias; Metabolism, Inborn Errors

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Amino Acid Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Anita MacDonald

  Birmingham Women's and Children's NHS Foundation Trust

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 24, 2017
• First Posted: May 30, 2017
• Study Start: June 8, 2017
• Primary Completion: March 15, 2018
• Study Completion: March 15, 2018
• Last Updated: February 16, 2024

Record last verified: 2024-02

Data Sharing

• IPD Sharing: Will not share

Locations

GB (3)