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

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Disease Information

Descriptive Information
Brief Title † 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
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.
Study Phase N/A
Study Type † Interventional
Study Design †
Primary Outcome Measure † Nutritional Status (objective measure)
Secondary Outcome Measure † Nutritional Status (Objective measure)
Condition † Phenylketonurias Hyperphenylalaninaemia, Type I
Intervention † Dietary SupplementSynergy
Study Arms / Comparison Groups 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. 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.
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status † Dietary Supplement
Estimated Enrollment † 50
Start Date † May 1, 2017
Completion Date May 2018
Primary Completion Date May 2018
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
Gender All
Ages 16 Years - 100 Years
Accepts Healthy Volunteers No
Contacts ††
Location Countries † United Kingdom
Administrative Information
NCT ID † NCT03167697
Organization ID PKUSyn.2017
Secondary IDs ††
Responsible Party Sponsor
Study Sponsor † Nutricia UK Ltd
Collaborators ††
Investigators † Principal Investigator: Rebecca Stratton, PhD, Nutricia UK
Information Provided By
Verification Date May 2017
First Received Date † May 22, 2017
Last Updated Date May 24, 2017
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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