Clinical trial NCT03167697: Evaluating the Efficacy of PKU Synergy in Patients Expressing Phenylketonuria or Hyperphenylalaninemia
Official title: Evaluating the Efficacy of PKU Synergy in Patients Expressing Phenylketonuria or Hyperphenylalaninemia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Phenylketonurias
Hyperphenylalaninaemia, Type I
Keywords: Phenylketonuria
Hyperphenylalaninaemia
Inherited Metabolic Disorders
Lead sponsor: Nutricia UK Ltd
Sponsor class: INDUSTRY
First posted: 2017-05-30
Enrollment: 14
Sex: ALL
Minimum age (years): 16
Maximum age (years): 100
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: SUPPORTIVE_CARE

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.

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

Primary outcomes:
measure: Nutritional Status (objective measure)
timeFrame: 2 days
Secondary outcomes:
measure: Nutritional Status (Objective measure)
timeFrame: 2 days
measure: Nutritional Status (Objective measure)
timeFrame: 2 days
measure: Nutritional Status (Objective measure)
timeFrame: 2 days
measure: Nutritional Status (Objective measure)
timeFrame: 2 days
measure: Nutritional Status (subjective measure)
timeFrame: 6 days
measure: Daily compliance with prescribed protein substitute as assessed by standarised questionnaire
timeFrame: 31 days
measure: Metabolic Control
timeFrame: 2 days
measure: Gastrointestinal tolerance as assessed by standarised questionnaire
timeFrame: 12 days
measure: Acceptability as assessed by standarised questionnaire
timeFrame: 2 days
measure: Subjective Mood
timeFrame: 3 days
measure: Anthropometry
timeFrame: 2 days
Other outcomes:
Locations:
city: Birmingham
country: United Kingdom
facility: Queen Elizabeth Hospital
city: Edinburgh
country: United Kingdom
facility: Royal Hospital for Sick Children
city: Glasgow
country: United Kingdom
facility: Queen Elizabeth University Hospital
city: London
country: United Kingdom
facility: Guys & St Thomas' Hospital
city: Newcastle upon Tyne
country: United Kingdom
facility: Royal Victoria Infirmary
city: Southampton
country: United Kingdom
facility: Southampton General Hospital
Study officials:
affiliation: Nutricia UK
name: Rebecca Stratton, PhD
role: PRINCIPAL_INVESTIGATOR
References: