NCT03167697

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

• Status: Completed
• Phase: N/A
• Study type: Interventional
• Enrollment: 14 (actual)
• Sponsor: Nutricia UK Ltd · Industry
• Sex: All
• Age: 16 Years – 100 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Phenylketonurias
• Hyperphenylalaninaemia, Type I

Interventions

Type	Name	Description
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).
OTHER	Routine	Patients will continue their usual dietary and/or protein substitute regimen (28 days).

Timeline

• Start date: 2017-05-01
• Primary completion: 2018-09-20
• Completion: 2018-12-17
• First posted: 2017-05-30
• Last updated: 2019-03-13

Locations

6 sites across 1 country: United Kingdom