NCT03167697

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
14

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started May 2017

Geographic Reach
1 country

6 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

May 1, 2017

Completed
21 days until next milestone

First Submitted

Initial submission to the registry

May 22, 2017

Completed
8 days until next milestone

First Posted

Study publicly available on registry

May 30, 2017

Completed
1.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 20, 2018

Completed
3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

December 17, 2018

Completed
Last Updated

March 13, 2019

Status Verified

March 1, 2019

Enrollment Period

1.4 years

First QC Date

May 22, 2017

Last Update Submit

March 11, 2019

Conditions

PhenylketonuriasHyperphenylalaninaemia, Type I

Keywords

PhenylketonuriaHyperphenylalaninaemiaInherited Metabolic Disorders

Outcome Measures

Primary Outcomes (1)

  • Nutritional Status (objective measure)

    Blood micro nutrient levels; active Vitamin B12 (holotranscobalamin)

    2 days

Secondary Outcomes (11)

  • Nutritional Status (Objective measure)

    2 days

  • Nutritional Status (Objective measure)

    2 days

  • Nutritional Status (Objective measure)

    2 days

  • Nutritional Status (Objective measure)

    2 days

  • Nutritional Status (subjective measure)

    6 days

  • +6 more secondary outcomes

Study Arms (2)

Synergy

EXPERIMENTAL

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.

Dietary Supplement: Synergy

Routine

ACTIVE COMPARATOR

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.

Other: Routine

Interventions

SynergyDIETARY_SUPPLEMENT

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

Synergy
RoutineOTHER

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

Routine

Eligibility Criteria

Age16 Years - 100 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (6)

Queen Elizabeth Hospital

Birmingham, United Kingdom

Location

Royal Hospital for Sick Children

Edinburgh, United Kingdom

Location

Queen Elizabeth University Hospital

Glasgow, United Kingdom

Location

Guys & St Thomas' Hospital

London, United Kingdom

Location

Royal Victoria Infirmary

Newcastle upon Tyne, United Kingdom

Location

Southampton General Hospital

Southampton, United Kingdom

Location

MeSH Terms

Conditions

Phenylketonurias

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesAmino Acid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Rebecca Stratton, PhD

    Nutricia UK

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
not applicable
Allocation
RANDOMIZED
Masking
NONE
Purpose
SUPPORTIVE CARE
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 22, 2017

First Posted

May 30, 2017

Study Start

May 1, 2017

Primary Completion

September 20, 2018

Study Completion

December 17, 2018

Last Updated

March 13, 2019

Record last verified: 2019-03

Data Sharing

IPD Sharing
Will not share

No IPD will be shared with other researchers.

Locations

GB(6)