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Trial record 28 of 80 for:    "phenylketonuria"

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

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ClinicalTrials.gov Identifier: NCT03167697
Recruitment Status : Enrolling by invitation
First Posted : May 30, 2017
Last Update Posted : November 1, 2017
Sponsor:
Information provided by (Responsible Party):
Nutricia UK Ltd

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.

Condition or disease Intervention/treatment Phase
Phenylketonurias Hyperphenylalaninaemia, Type I Dietary Supplement: Synergy Other: Routine Not Applicable

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  (Clinical Trial)
Estimated Enrollment : 50 participants
Allocation: Randomized
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 : May 2018
Estimated Study Completion Date : May 2018


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




Primary Outcome Measures :
  1. Nutritional Status (objective measure) [ Time Frame: 2 days ]
    Blood micro nutrient levels; active Vitamin B12 (holotranscobalamin)


Secondary Outcome Measures :
  1. Nutritional Status (Objective measure) [ Time Frame: 2 days ]
    Blood micro nutrient levels; Folate (Folic Acid)

  2. Nutritional Status (Objective measure) [ Time Frame: 2 days ]
    Blood micro nutrient levels; Iron (Ferritin)

  3. Nutritional Status (Objective measure) [ Time Frame: 2 days ]
    Blood micro nutrient levels; Zinc

  4. Nutritional Status (Objective measure) [ Time Frame: 2 days ]
    Blood micro nutrient levels; Vitamin D (25-hydroxyvitamin D)

  5. Nutritional Status (subjective measure) [ Time Frame: 6 days ]
    3 day weighed food diary

  6. 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.

  7. 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.

  8. 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.

  9. 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.

  10. Subjective Mood [ Time Frame: 3 days ]
    Profile of mood states questionnaire

  11. 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.



Information from the National Library of Medicine

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Ages Eligible for Study:   16 Years to 100 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

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


Locations
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, United Kingdom
Sponsors and Collaborators
Nutricia UK Ltd
Investigators
Principal Investigator: Rebecca Stratton, PhD Nutricia UK

Responsible Party: Nutricia UK Ltd
ClinicalTrials.gov Identifier: NCT03167697     History of Changes
Other Study ID Numbers: PKUSyn.2017
First Posted: May 30, 2017    Key Record Dates
Last Update Posted: November 1, 2017
Last Verified: October 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: No IPD will be shared with other researchers.

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Nutricia UK Ltd:
Phenylketonuria
Hyperphenylalaninaemia
Inherited Metabolic Disorders

Additional relevant MeSH terms:
Phenylketonurias
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases