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Completed

NCT Number: NCT03167697

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

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.

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Key information

Conditions

Phenylketonurias Amino Acid Metabolism, Inborn Errors Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hyperphenylalaninaemia, Type I Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases

Age range

16 year–100 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Queen Elizabeth Hospital, Birmingham, United Kingdom

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Synergy

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

Other

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

Primary outcomes

  1. Nutritional Status (objective measure)

    Time frame: 2 days

    Blood micro nutrient levels; active Vitamin B12 (holotranscobalamin)

Secondary outcomes

  1. Nutritional Status (Objective measure)

    Time frame: 2 days

    Blood micro nutrient levels; Folate (Folic Acid)

  2. Nutritional Status (Objective measure)

    Time frame: 2 days

    Blood micro nutrient levels; Iron (Ferritin)

  3. Nutritional Status (Objective measure)

    Time frame: 2 days

    Blood micro nutrient levels; Zinc

  4. Nutritional Status (Objective measure)

    Time frame: 2 days

    Blood micro nutrient levels; Vitamin D (25-hydroxyvitamin D)

  5. Nutritional Status (subjective measure)

    Time frame: 6 days

    3 day weighed food diary

  6. Daily compliance with prescribed protein substitute as assessed by standarised questionnaire

    Time frame: 31 days

    Compliance with the recommended intake of the patients previously prescribed protein substitute (during baseline in both groups, and during the study period in the control group) and with the study product during the intervention period will be assessed daily throughout the study. Patients will be asked to record how much of the protein substitute is taken compared to that recommended by their Health Care Professional. The daily amount prescribed by the Health Care Professional managing the patients care will be recorded at the start of the study and any changes to this prescription during the study will be noted. During the intervention period, protein substitute consumption patterns (e.g. timing and amount consumed) will be recorded daily and assessed b y a standardised questionnaire.

  7. Metabolic Control

    Time frame: 2 days

    Blood amino acid levels; Collected samples will be analysed for blood phenylalanine, tyrosine and 16 other proteinogenic amino acids, 2 non-proteinogenic amino acids and 1 amino sulfonic acid.

  8. Gastrointestinal tolerance as assessed by standarised questionnaire

    Time frame: 12 days

    Gastrointestinal tolerance (including diarrhoea, constipation, nausea, vomiting, abdominal pain, bloating, flatulence and burping) will be assessed using a standardised gastrointestinal tolerance questionnaire completed by the patient.

  9. Acceptability as assessed by standarised questionnaire

    Time frame: 2 days

    Acceptability (ease of use and liking) of the patients previously prescribed protein substitute and the study product will be assessed at the end of baseline (day 4) and the end of the intervention period (day 31) using a standardised questionnaire completed by the patient.

  10. Subjective Mood

    Time frame: 3 days

    Profile of mood states questionnaire

  11. Anthropometry

    Time frame: 2 days

    Measures of weight and height will be made during baseline observations (day 1) and at the end of the intervention period (day 31). Weight will be determined to the nearest 0.1 kg, using portable scales shoeless and wearing light weight clothing.

Sponsors and collaborators

Lead sponsor

Nutricia UK Ltd

Industry

Registry information

Important dates

Study start
2017
Primary completion
2018
Study completion
2018
First posted
May 30, 2017
Registry last updated
Mar 13, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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