Evaluating the Efficacy of PKU Synergy in Patients Expressing Phenylketonuria or Hyperphenylalaninemia
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03167697|
Recruitment Status : Enrolling by invitation
First Posted : May 30, 2017
Last Update Posted : August 31, 2018
|Condition or disease||Intervention/treatment||Phase|
|Phenylketonurias Hyperphenylalaninaemia, Type I||Dietary Supplement: Synergy Other: Routine||Not Applicable|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||50 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Primary Purpose:||Supportive Care|
|Official Title:||Evaluating the Efficacy of PKU Synergy in Patients Expressing Phenylketonuria or Hyperphenylalaninemia|
|Actual Study Start Date :||May 1, 2017|
|Estimated Primary Completion Date :||December 30, 2018|
|Estimated Study Completion Date :||December 31, 2018|
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.
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).
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).
- Nutritional Status (objective measure) [ Time Frame: 2 days ]Blood micro nutrient levels; active Vitamin B12 (holotranscobalamin)
- Nutritional Status (Objective measure) [ Time Frame: 2 days ]Blood micro nutrient levels; Folate (Folic Acid)
- Nutritional Status (Objective measure) [ Time Frame: 2 days ]Blood micro nutrient levels; Iron (Ferritin)
- Nutritional Status (Objective measure) [ Time Frame: 2 days ]Blood micro nutrient levels; Zinc
- Nutritional Status (Objective measure) [ Time Frame: 2 days ]Blood micro nutrient levels; Vitamin D (25-hydroxyvitamin D)
- Nutritional Status (subjective measure) [ Time Frame: 6 days ]3 day weighed food diary
- 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.
- 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.
- 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.
- 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.
- Subjective Mood [ Time Frame: 3 days ]Profile of mood states questionnaire
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03167697
|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|
|Principal Investigator:||Rebecca Stratton, PhD||Nutricia UK|