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

Nutricia

Status: Completed

Conditions: Phenylketonurias; Hyperphenylalaninaemia, Type I

Treatments: Other: Routine; Dietary Supplement: Synergy

Study type: Interventional

Funder types: Industry

Identifiers

NCT03167697

PKUSyn.2017

Details and patient eligibility

About

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.

Full 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.

Enrollment: 14 patients

Sex: All

Ages: 16 to 100 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Supportive Care

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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14 participants in 2 patient groups

Synergy

Experimental group

Description:

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.

Treatment:

Dietary Supplement: Synergy

Routine

Active Comparator group

Description:

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.

Treatment:

Other: Routine