- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03167697
Evaluating the Efficacy of PKU Synergy in Patients Expressing Phenylketonuria or Hyperphenylalaninemia
March 11, 2019 updated by: Nutricia UK Ltd
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.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
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.
Study Type
Interventional
Enrollment (Actual)
14
Phase
- Not Applicable
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
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Birmingham, United Kingdom
- Queen Elizabeth Hospital
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Edinburgh, United Kingdom
- Royal Hospital for Sick Children
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Glasgow, United Kingdom
- Queen Elizabeth University Hospital
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London, United Kingdom
- Guys & St Thomas' Hospital
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Newcastle upon Tyne, United Kingdom
- Royal Victoria Infirmary
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Southampton, United Kingdom
- Southampton General Hospital
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-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
16 years to 100 years (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Supportive Care
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Synergy
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.
|
Small serving (x1 33 g serving daily) of the new phenylalanine-free protein substitute made up with 100mL of water daily (28 days).
|
Active Comparator: Routine
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.
|
Patients will continue their usual dietary and/or protein substitute regimen (28 days).
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Nutritional Status (objective measure)
Time Frame: 2 days
|
Blood micro nutrient levels; active Vitamin B12 (holotranscobalamin)
|
2 days
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Nutritional Status (Objective measure)
Time Frame: 2 days
|
Blood micro nutrient levels; Folate (Folic Acid)
|
2 days
|
Nutritional Status (Objective measure)
Time Frame: 2 days
|
Blood micro nutrient levels; Iron (Ferritin)
|
2 days
|
Nutritional Status (Objective measure)
Time Frame: 2 days
|
Blood micro nutrient levels; Zinc
|
2 days
|
Nutritional Status (Objective measure)
Time Frame: 2 days
|
Blood micro nutrient levels; Vitamin D (25-hydroxyvitamin D)
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2 days
|
Nutritional Status (subjective measure)
Time Frame: 6 days
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3 day weighed food diary
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6 days
|
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.
|
31 days
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Metabolic Control
Time Frame: 2 days
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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
|
Gastrointestinal tolerance as assessed by standarised questionnaire
Time Frame: 12 days
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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.
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12 days
|
Acceptability as assessed by standarised questionnaire
Time Frame: 2 days
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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.
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2 days
|
Subjective Mood
Time Frame: 3 days
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Profile of mood states questionnaire
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3 days
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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.
|
2 days
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Principal Investigator: Rebecca Stratton, PhD, Nutricia UK
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
May 1, 2017
Primary Completion (Actual)
September 20, 2018
Study Completion (Actual)
December 17, 2018
Study Registration Dates
First Submitted
May 22, 2017
First Submitted That Met QC Criteria
May 24, 2017
First Posted (Actual)
May 30, 2017
Study Record Updates
Last Update Posted (Actual)
March 13, 2019
Last Update Submitted That Met QC Criteria
March 11, 2019
Last Verified
March 1, 2019
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- PKUSyn.2017
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
IPD Plan Description
No IPD will be shared with other researchers.
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
No
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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