Evaluation of PKU Start

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

NA

Total Enrollment

10 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-03-06

Study Completion Date

2017-09-15

Brief Summary

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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.

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Detailed Description

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This is an assessment of ten (10) infants who require a protein restricted diet that is low in Phe. Infants who routinely use a Phe free infant formula as part of their dietary therapy will be recruited for a 28-day assessment of PKU Start, to evaluate tolerance and acceptability.

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 Start based on individual requirements. Parents/carers will be asked to substitute their usual Phe-free infant formula with PKU Start for one (1) month. The sponsor will supply PKU Start free of charge.

Prior to starting PKU Start, parents/carers will be asked to record information about the infant's usual GI tolerance and feeding pattern for a period of three (3) days, to allow for comparison between their existing formula and PKU Start.

They will be asked to record information about the following:

Stools Vomiting and Spit-up Feed / Fluid Intake and Compliance Phenylalanine Levels Final Evaluation about the presentation of the product, ease of preparation and how PKU Start flowed through the teat of a bottle.

Conditions

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Phenylketonuria Inborn Errors of Metabolism

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Consumption of PKU Start

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

Group Type EXPERIMENTAL

PKU Start

Intervention Type 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.

Interventions

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PKU Start

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.

Intervention Type DIETARY_SUPPLEMENT

Eligibility Criteria

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Inclusion Criteria

1. 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)
2. Taking a minimum of one (1) feed of a Phe-free infant formula
3. A minimum period of four (4) weeks from the time of diagnosis to initial approach to parents

Exclusion Criteria

1. 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)
2. Diagnosis of a congenital condition

Minimum Eligible Age

4 Weeks

Maximum Eligible Age

2 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No