Evaluation of PKU Start

NCT03058848 | Completed

• 3 other identifiers: BCH-PKUSTART-032016-2920992017/NW/0035
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 3

Brief Summary

To evaluate the acceptability, tolerance and effect on metabolic control of PKU Start, a new Phe free protein substitute for the dietary management of PKU in infants from birth.

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

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

  Days 1-28

• Ease of use questionnaire

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

  Day 29

• Daily phenylalanine control

  Collection of quantitative data regarding phenylalanine control using routine biochemical testing

  Days 1-28

Study Arms (1)

Consumption of PKU Start

EXPERIMENTAL

Daily feed, substituting the participant's normal phe-free formula for PKU Start.

Dietary Supplement: PKU Start

Interventions

PKU Start DIETARY_SUPPLEMENT

PKU Start is a powdered, phenylalanine-free, infant formula, containing essential and non-essential amino acids, carbohydrate, fat, vitamins, minerals, trace elements and long chain polyunsaturated fatty acids (LCPs); Arachidonic acid (AA) and Docosahexaenoic acid (DHA). It is suitable for use from birth. The recommended amount of the product for each participant will be determined and prescribed by a dietitian.

Consumption of PKU Start

Eligibility Criteria

• Age: 4 Weeks - 2 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• A diagnosis of classical or severe PKU on new-born screening (For the purposes of this study, 'severe' is defined as phe concentrations between 120 and 600 µmol/L at diagnosis)
• Taking a minimum of one (1) feed of a Phe-free infant formula
• A minimum period of four (4) weeks from the time of diagnosis to initial approach to parents

You may not qualify if:

• Diagnosed with mild PKU or hyperphenylalaninaemia (For the purposes of this study, 'mild' is defined as phe concentrations between 120 and 600 µmol/L at diagnosis)
• Diagnosis of a congenital condition

Sponsors & Collaborators

• Vitaflo International, Ltd: lead

Study Sites (3)

NHS Greater Glasgow and Clyde

Glasgow, Lanarkshire, G51 4TF, United Kingdom

Location

Birmingham Children's Hospital NHS Foundation Trust

Birmingham, 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: February 16, 2017
• First Posted: February 23, 2017
• Study Start: March 6, 2017
• Primary Completion: September 15, 2017
• Study Completion: September 15, 2017
• Last Updated: February 16, 2024

Record last verified: 2024-02

Locations

GB (3)