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

Phenylketonurias Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03167697
• Other study ID #: PKUSyn.2017
• Status: Completed
• Phase: N/A
• Start date: May 1, 2017
• Est. completion date: December 17, 2018

Study information

• Verified date: March 2019
• Source: Nutricia UK Ltd
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 14
• Est. completion date: December 17, 2018
• Est. primary completion date: September 20, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 16 Years to 100 Years

Eligibility

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

Related Conditions & MeSH terms

• Hyperphenylalaninaemia, Type I
• Phenylketonurias

Intervention

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

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

Locations

Country	Name	City	State
United Kingdom	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

Sponsors (1)

Lead Sponsor	Collaborator
Nutricia UK Ltd

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Nutritional Status (objective measure)	Blood micro nutrient levels; active Vitamin B12 (holotranscobalamin)	2 days
Secondary	Nutritional Status (Objective measure)	Blood micro nutrient levels; Folate (Folic Acid)	2 days
Secondary	Nutritional Status (Objective measure)	Blood micro nutrient levels; Iron (Ferritin)	2 days
Secondary	Nutritional Status (Objective measure)	Blood micro nutrient levels; Zinc	2 days
Secondary	Nutritional Status (Objective measure)	Blood micro nutrient levels; Vitamin D (25-hydroxyvitamin D)	2 days
Secondary	Nutritional Status (subjective measure)	3 day weighed food diary	6 days
Secondary	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.	31 days
Secondary	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.	2 days
Secondary	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.	12 days
Secondary	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.	2 days
Secondary	Subjective Mood	Profile of mood states questionnaire	3 days
Secondary	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.	2 days