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.
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
SUPPORTIVE_CARE
Masking
NONE
Enrollment
14
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
Nutritional Status (objective measure)
Blood micro nutrient levels; active Vitamin B12 (holotranscobalamin)
Time frame: 2 days
Nutritional Status (Objective measure)
Blood micro nutrient levels; Folate (Folic Acid)
Time frame: 2 days
Nutritional Status (Objective measure)
Blood micro nutrient levels; Iron (Ferritin)
Time frame: 2 days
Nutritional Status (Objective measure)
Blood micro nutrient levels; Zinc
Time frame: 2 days
Nutritional Status (Objective measure)
Blood micro nutrient levels; Vitamin D (25-hydroxyvitamin D)
Time frame: 2 days
Nutritional Status (subjective measure)
3 day weighed food diary
Time frame: 6 days
Daily compliance with prescribed protein substitute as assessed by standarised questionnaire
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.
Time frame: 31 days
Metabolic Control
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.
Time frame: 2 days
Gastrointestinal tolerance as assessed by standarised questionnaire
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.
Time frame: 12 days
Acceptability as assessed by standarised questionnaire
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.
Time frame: 2 days
Subjective Mood
Profile of mood states questionnaire
Time frame: 3 days
Anthropometry
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.
Time frame: 2 days
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