Evaluating the Efficacy of PKU Synergy in Patients Expressing Phenylketonuria or Hyperphenylalaninemia

NCT ID: NCT03167697

Last Updated: 2019-03-13

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 14 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2017-05-01
• Study Completion Date: 2018-12-17

Brief Summary

This study centres around a new one-a-day phenylalanine-free protein substitute for phenylketonuria patients. Fifty eligible adults (≥ 16 years) with proven phenylketonuria or hyperphenylalaninemia will be recruited and randomly allocated to one of two intervention arms (n = 25 per arm). Following a 3-day baseline period, and in addition to routine nutritional management, patients will receive either one sachet of the new protein substitute daily (intervention) or continue their usual dietary and/or protein substitute regimen (maximum of 1 protein substitute per day (equal to 20g protein equivalent) control) for 28 days.

Detailed Description

This study centres around a new one-a-day phenylalanine-free protein substitute for phenylketonuria patients. In particular, this randomised controlled trial aims to evaluate the efficacy (changes relating to nutritional status and metabolic control) of this new protein substitute, while also capturing data pertaining to tolerance, compliance, safety and acceptability. Designed for poorly compliant adult patients with proven phenylketonuria or hyperphenylalaninemia, this new protein substitute is composed of an adapted mixture of other essential and non-essential amino acids, carbohydrates, vitamins and selected minerals and trace elements and enriched with docosahexaenoic acid (DHA). Fifty eligible adults (≥ 16 years) will be recruited and randomly allocated to one of two intervention arms (n = 25 per arm). Following a 3-day baseline period, and in addition to routine nutritional management, patients will receive either one sachet of the new protein substitute daily (intervention) or continue their usual dietary and/or protein substitute regimen (maximum of 1 protein substitute per day (equal to 20g protein equivalent) control) for 28 days.

Conditions

Phenylketonurias; Hyperphenylalaninaemia, Type I

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: SUPPORTIVE_CARE
• Blinding Strategy: NONE

Study Groups

Synergy

This group will receive the new phenylalanine-free protein substitute daily for 28 days. Patients in this group intervention will be directed to consume one powder sachet (33 g) of daily made up with 100mL of water. The new substitute delivers 414 kJ, 20g protein equivalent and a combination of essential and non-essential amino acids as well a combination of vitamins and minerals.

Group Type: EXPERIMENTAL

Synergy

Intervention Type: DIETARY_SUPPLEMENT

Small serving (x1 33 g serving daily) of the new phenylalanine-free protein substitute made up with 100mL of water daily (28 days).

Routine

This group will continue their usual dietary and/or protein substitute regimen (maximum of 1 protein substitute per day (equal to 20g protein equivalent) control) for 28 days.

Group Type: ACTIVE_COMPARATOR

Routine

Intervention Type: OTHER

Patients will continue their usual dietary and/or protein substitute regimen (28 days).

Interventions

Synergy

Small serving (x1 33 g serving daily) of the new phenylalanine-free protein substitute made up with 100mL of water daily (28 days).

Intervention Type: DIETARY_SUPPLEMENT

Routine

Patients will continue their usual dietary and/or protein substitute regimen (28 days).

Intervention Type: OTHER

Eligibility Criteria

Inclusion Criteria

• Male or female
• Over 16 years of age
• Diagnosed with proven PKU or hyperphenylalaninemia with an increased phenylalanine-tolerance/intake
• Currently taking a maximum of 1 protein substitute per day (equal to 20g protein equivalent)
• Have a minimum blood phenylalanine level of ≥ 600 umol/L (for PKU patients)
• Have relaxed (if not stopped) their dietary and protein substitute regimen for at least 1 month prior to trial commencement
• Have Written informed consent from patient

Exclusion Criteria

• Pregnant or lactating
• Requiring nutritional support (including enteral and parenteral nutrition)
• Major hepatic or renal dysfunction
• Participation in other studies within 1 month prior to entry of this study
• Allergy to any of the study product ingredients, including milk protein or soya
• Investigator concern around willingness/ability of patient to comply with protocol requirements

• Minimum Eligible Age: 16 Years
• Maximum Eligible Age: 100 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Nutricia UK Ltd

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Rebecca Stratton, PhD

Role: PRINCIPAL_INVESTIGATOR

Nutricia UK

Locations

Queen Elizabeth Hospital

Birmingham, , United Kingdom

Royal Hospital for Sick Children

Edinburgh, , United Kingdom

Queen Elizabeth University Hospital

Glasgow, , United Kingdom

Guys & St Thomas' Hospital

London, , United Kingdom

Royal Victoria Infirmary

Newcastle upon Tyne, , United Kingdom

Southampton General Hospital

Southampton, , United Kingdom

Countries

United Kingdom

Other Identifiers

PKUSyn.2017

Identifier Type: -

Identifier Source: org_study_id